Category Archives: Genetic Engineering

HENRIETTA, N.Y. (WROC) A company and project called Colossal has received $15 million dollars in funding, according to CNN, to do one thing with a big goal: Resurrect the woolly mammoth.

The project hopes that the reintroduction of the animal that has been extinct for more than 4,000 years can reform the Artic, tundra, and the Steppes to help combat climate change.

Colossals website has this to offer: It is a science that has been developed and mastered by George Church, Ph.D. and his lab. With a 99.6% genetic match in the Asian elephant, intact Mammoth DNA, and modern genetic engineering, the task is well underway.

This comes on the heels of a full genetic sequence taken from a discovered mammoth fossil, and the project says that they can use CRISPR technology to essentially pick which genes were present in the mammoth and then splice them into the Asian elephant genome.

Our goal is to have our first calves in the next four to six years, said tech entrepreneur Ben Lamm, a backer of Colossal also said to CNN.

To offer some perspective and insight on this project, Robert Rothman, RIT Professor Emeritus of Life Sciences who is both familiar with the project and the technology used in this project discussed with News 8 his reaction to the feasibility of the project, its mission, practicality, the amount of funding, and more.

To get funding quickly out of the way:

I would say seems pretty slim, right? he said. Just to sequence it and and the cloning is probably going to require a lot of manpower I would guess thats barely scratching the surface of what it would cost.

And as for the mission itself, Rothman gives an overview for us.

Its an idea called rewilding, Rothman said. The goal is to replace a missing keystone organism to re-introduce a modern population [and] if thats not possible to introduce either a closely related species or a species that fills a similar ecological niche.

Rothman equates it to the re-introduction of gray wolves to Yellowstone Park animals that were there, but the project increased the number or in his area of expertise, tortoises on the Galapagos. While both of these projects invovled extant, rather than extinct, animals, Rothman draws parallels to the captive breeding programs, as well as their timelines and difficulty.

He discusses the Espanola tortoise, which at one point only had 15 remaining animals. They were all brought back to a station in 1971, and over 30 years later, the population he says is over 2,000. But that was a decades-long process, unlike the 4-5 timeline the Colossal project laid out.

So thats kind of the comparison that I look at now, if you put this in the context of the elephants, it takes about two years for a fertilized egg to mature into a baby elephant, Rothman said.

He credits the Colossal team for being well-researched and having exceptional ability to do the actual gene splicing work with CRISPR, but says that birthing these mammoth-esque animals has other complications, namely how the animal will develop, specifically how it will be rasied and socialized.

If its born in a tube, how will it grow up and socialize? If the fertilized egg with the mammoth analogue, would the Asian elephant be willing to raise its alien offspring? Rothman says theres no way to answer these questions until the project begins.

He also adds that in the case of the tortoises, since they were a product of the captive breeding program and thanks to an industrious tortoise named Diego, who according to the Galapagos National Parks may have sired around 40% of the new population the current population is fairly homogenous, and may lead to inbreeding issues down the line.

As for the overall goal itself, Rothman has his doubts. While he cited two examples that have worked, rewilding doesnt always work, saying that often solutions to one problem create others.

The classic example are cane toads in Australia, he said. I think its a huge problem (They) were introduced about a hundred years ago to deal with pest insect pests that attacking sugar cane, but the frog population exploded. And theyre poisonous. If you touch them, I think you you get a bad reaction, but if a dog (eats) one (it can get) sick or die.

But with these examples, these creatures are extant; they currently exist on Earth, and are moved someplace else, or their numbers are increased. In this case The woolly mammoth has not trundled across the Steppes in 4,000 years. Rothman says mammoths arent around for a reason, saying that they were animals who thrived in cold weather, and with a warming planet, their odds of survival dont intuitively feel promising.

Finally, Rothman addresses the ethical concerns of recreating species, and extensively editing the genes of current ones. He points out that some say that this technology is so powerful that it could be possibly be used to help current elephants populations, but instead being used to recreate old ones. He does add one more philosophical concern of many:

We shouldnt play God,' he said, echoing those concerns.

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RIT professor on $15M effort to resurrect the wooly mammoth by Harvard scientists - RochesterFirst

EAGAN, Minn., Sept. 15, 2021 /PRNewswire/ -- Makana Therapeutics today received a $650,000 KidneyX Artificial Kidney Prize for their work to increase the supply of kidney donor organs through pig-to-human xenotransplants. Xenotransplantation is the process of transplanting organs or tissues between members of different species into humans, thus addressing the crisis of organ shortage we are facing today.

"We see a future where xenotransplantation will allow end-stage renal disease patients who need a kidney transplant to have one immediately, without the years of waiting and uncertainty they currently face," said Matt Tector, chief scientific officer of Makana Therapeutics.

Every year more than 100,000 new patients are deemed to need some form of renal replacement, yet fewer than 25,000 kidney transplants are performed annually due to a shortage of suitable donors.

To meet this increasing demand, Makana has developed a "triple knockout" pig with kidneys viable for human transplant by inactivating three separate genes to reduce expression of pig molecules on the transplanted kidney that are targeted by human antibodies. By eliminating these three genes from pigs, it is possible to reduce, and in some cases, eliminate human antibody binding to the pig cells which typically lead to organ rejection or other complications. Makana estimates the "triple knockout" pig will be a suitable kidney donor for as many as 70 percent of kidney failure patients.

Makana, a subsidiary of Minneapolis-based life-sciences company Recombinetics, is strategically placed to bring this innovation to market leveraging both Makana's strong intellectual property and pre-clinical success as well as Recombinetics leadership in large animal genetic engineering and animal husbandry expertise.

"We believe there are no remaining scientific unknowns for us to get to our clinical trial. Our remaining efforts will be geared towards gaining FDA approval for the trial and continuing to improve our technology so we can meet our goal of providing a transplant to every patient in need," said Dr. Joe Tector, founder of Makana.

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As a pre-clinical stage company, Makana expects its first-in-human trial to commence as early as 2022 and could be to market as soon as 2025.

About The Artificial Kidney Prize

The Kidney Innovation Accelerator (KidneyX), a public-private partnership between the U.S. Department of Health and Human Services (HHS) and the American Society of Nephrology (ASN), is accelerating innovation in the prevention, diagnosis, and treatment of kidney diseases.

The Artificial Kidney Prize is a competition to accelerate the development of continuous kidney replacement therapies that provide transformational treatment options beyond current dialysis methods. For this competition, artificial kidneys may be wearable, implantable, bioengineered, developed as a xenotransplant or chimera organ, or other approaches not yet conceived.

About Makana Therapeutics

Founded in 2009, Makana Therapeutics is focused on developing swine with reduced xenoantigen expression, making human transplantation of cells, tissues and organs from these animals possible. Makana's focus on simplified genetics, optimized pig cloning techniques and careful patient selection is expected to streamline product development and result in safer more efficacious products.

About Recombinetics

Founded in 2008, Recombinetics Inc. is a recognized global leader in the development, deployment, and commercialization of genetically engineered animals. Its three subsidiaries, Regenevida, Surrogen, and Acceligen, have delivered hundreds of animals to: enable drug, device, and therapeutic discovery; generate transplantable cells, tissues, and organs; and provide improved health, well-being, and productivity in agricultural animals.

PR/Media ContactContact: Nikki Rockstroh, 318939@email4pr.com 612.727.2000

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View original content:https://www.prnewswire.com/news-releases/makana-therapeutics-awarded-kidneyx-artificial-kidney-prize-for-innovation-in-xenotransplantation-301376910.html

SOURCE Recombinetics Inc

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Makana Therapeutics Awarded KidneyX Artificial Kidney Prize for Innovation in Xenotransplantation - Yahoo Finance

Scientists at Ohio State University provide evidence that members of a class of proteins called serine incorporator proteins, best known for curbing HIV infection also promote other antiviral activities that increase the production of type I interferons and proinflammatory cytokines. By promoting these signaling pathways of innate immunity, the SERINC restriction factors SERINC3 and SERINC5, protect cells from infection by HIV-1, vesicular stomatitis virus (VSV), and Zika virus.

The authors show SERINC5, usually present in the cell membrane, moves to the outer mitochondrial membrane protein where it forms a protein complex with an adapter protein, MAVS (mitochondrial antiviral signaling), and TRAF6, an E3 ubiquitin ligasean enzyme that degrades proteins.

Reducing the expression of SERINC5 in target cells, the authors note, increases cellular infection by HIV-1, VSV and an endemic Asian strain of Zika virus. This demonstrates SERINC5 mediates direct antiviral activities in host cells in addition to the indirect inhibition of HIV-1 reported in earlier studies. Probing further into the mechanism, the authors show SERINC5s antiviral activity depends on its ability to stimulate the expression of type I interferons and NF-kB inflammatory signaling.

These findings are reported in the Science Signaling article, SERINC proteins potentiate antiviral type I IFN production and proinflammatory signaling pathways.

Senior author of the paperShan-Lu Liu, PhD, professor of virology in theDepartment of Veterinary Biosciences at The Ohio State University says, Viruses can get around direct antiviral effects, but if this protein can also modulate key pathways without acting directly on the virus, then a virus may have limited capacity to counteract it.

Restriction factorsproteins produced by host cells to restrict viral infectioninterfere either with the entry of virus particles into healthy cells or viral replication mechanisms in the host cell. Earlier studies have shown the restriction factor SERINC5, found in the host cell membrane incorporates into HIV-1 virus particles, blocking their entry into the host cell.

The current study notes SERINC5 also inhibits viral infections by promoting intracellular innate signaling pathways that involves its binding to the adaptor protein MAVS at the mitochondrial membrane. This binding results in MAVS oligomerization and activation of downstream transcriptional regulators that promote the expression of genes that encode antiviral type I IFNs and proinflammatory cytokines like NF-kB.

The aggregation of these proteins means they need each other, says Liu. A big complex like this can recruit additional molecules, enhancing the efficiency of the signal transduction pathway.

The researchers are currently testing whether this function is also effective against SARS-CoV-2, the virus that causes COVID-19.

If this family of molecules can do this in animals and humans, then you may think about whether it could be used in a broad antiviral therapy, says Liu. He is optimistic that the efficacy of SERINC proteins in inhibiting viral infection will extend to suppressing the COVID-19 virus.

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HIV, VSV, and Zika Virus Suppression in Just One Protein - Genetic Engineering & Biotechnology News

Scientists are a cautious bunch, fond of a caveat even when describing their own research. Our favourite expressions are Yes, but and On the other hand and It remains unclear says gene editor Dr Fyodor Urnov. So please add all of that to what Im about to say.

If CRISPR realises 10 per cent of what we practitioners of gene editing dream it to be able to do, it will rival the greatest advances in the history of biomedicine as a technology to impact public health.

Urnov is talking via a crackly Zoom link from his office at the Innovative Genomics Institute (IGI) in Berkeley, California, which is at the forefront of what may prove to be the defining scientific breakthrough of the 21st Century.

CRISPR is a genome editing technology that allows scientists to cut DNA with incredible precision and insert or delete DNA to correct unwanted mutations. To oversimplify the technology, its the power to edit the building blocks of life, just like text on a computer screen. Not only could it enable scientists to switch off genes that lead to a broad spectrum of disease, but it will unshackle all of us from the genetics were born with.

The knowledge gained is amazing and its just really accelerated basic research. That in itself is already transformative, says Dr Robin Lovell-Badge, from the Crick Institute. And the notion that we can actually treat people with genetic diseases in a way that was never possible before is mind boggling.

For much of the past decade, the technology has been confined to the lab. Now, though, the first CRISPR therapies are changing the fate of people born with debilitating inherited conditions. Most of us havent realised it yet, but were in the foothills of a profound medical and technological revolution that raises not only the prospect of new treatments and cures, but also huge questions about ethics, equality and health justice.

The incoming wave of gene-editing applications has been compared to the Industrial Revolution or the birth of the internet in terms of the game-changing impact it will have on society.

Jennifer Doudna, who collaborated with Emmanuelle Charpentier on the development of CRISPR and founded the Innovative Genomics Institute IGI

Biochemist Dr Jennifer Doudna established the IGI to tackle all of that and more. As a non-profit organisation, the institute exists not just to research gene editing, but also make it affordable and accessible to everyone. Its a big claim, given the breadth of CRISPRs potential, so the IGIs leadership team agreed to give BBC Science Focus an exclusive overview of its efforts.

It was there on the Berkeley campus where Doudna and Dr Emmanuelle Charpentier changed the world nearly a decade ago. Their collaboration led to the development of CRISPR-Cas9, the gene-editing tool described in a landmark 2012 paper that won a Nobel Prize.

I think we both had a little sense of, you know, kind of a chill, she says. I still remember that feeling of hairs standing up on my neck, thinking theres something really interesting here. And I would wager that neither of us at the time had any idea where it would end up, because I dont think one ever does.

CRISPR isnt the only gene-editing technology, and the IGI is not the only institute pioneering the field of study. But CRISPR is more versatile, easier to use and cheaper than a lot of other technologies. And whats astonishing is the speed of progress. In less than a decade its gone from discovery to human trials and potential cures, something thats practically unheard of in biomedicine.

Its a little bit of whiplash, says Dr Brad Ringeisen, executive director of the IGI. Think about nanoparticles, the nano revolution. Pfizer has nanoparticles in their COVID mRNA vaccine, but that [technology] has taken 30 or 40 years.

Read more about CRISPR:

People born with sickle cell disease will be among the first to benefit from advances in gene editing. In 2019, a woman called Victoria Gray became one of the first people in the world to be treated for a genetically inherited disease with a CRISPR-based therapy.

Born with sickle cell anaemia, she required strong pain medication and regular blood transfusions to stave off the effects of the mutated gene that makes red blood cells warp and block the flow of blood and oxygen around the body. Aside from bouts of excruciating pain, sickle cell anaemia can lead to stroke, hypertension, organ damage and more.

The treatment was not simple. Doctors removed bone marrow cells from Gray and the other patients in the trial, then used CRISPR to edit a gene that activates production of foetal haemoglobin, a protein that can alleviate the symptoms of sickle cell disease. The patients then underwent chemotherapy to destroy most of their bone marrow, after which billions of edited cells were infused back into their bodies.

Victoria Gray, who volunteered to have her sickle cell anaemia treated with CRISPR-based therapy Sarah Cannon Research Institute

Gray no longer requires medication or transfusions, and neither do the other people in her trial, which included patients with a related blood disorder. It seems that a one-time CRISPR treatment has cured them.

Sickle cell disease is probably the easiest target that affects the most people, says Ringeisen. Its about as simple as you can possibly get: you go in and either turn on an alternative gene or you try to correct the one thing. One hundred thousand people in United States have sickle cell disease and I think its over a million people worldwide. So theres a huge impact that can be had.

The IGI is preparing its own sickle cell trial, but the institutes scope also includes many other conditions. As well as blood disorders, it has active research projects on autoimmune disease, neurological disease, cancer and COVID-19. One promising avenue of research is with T-cells, sometimes described as the troops on the ground of our immune system.

Dr Alex Marson, the IGIs director of human health, is running a lab that is working to engineer T-cells using CRISPR to treat different kinds of disease. He is currently planning a clinical trial with a family where a strong genetic mutation has caused different manifestations of autoimmune disease in the younger generation.

In the lab weve corrected cells from these children, he says. Now were working towards doing a clinical trial to take those gene-corrected T-cells and infuse them into at least one of these young adults to try to restore balance in the system and treat the autoimmune disease.

Whereas current T-cell therapies are wildly expensive, Marson envisions a future where off-the-shelf T-cells are manufactured to treat different kinds of disease, their production industrialised to a scale that makes them accessible to anybody who needs them.

Sickle cell disease causes the body to produce unusually shaped red blood cells. These cells do not live as long as other red blood cells and can block blood vessels, leading to problems Getty Images

We can treat infectious diseases by designing immune cells that recognise infections, he says. I think were going to have this sort of flexible ability to actually write the language of the DNA in the immune cell and use it in a drug platform.

This is where things start getting really interesting, because it showcases just how broad the medical applications of CRISPR will be. Its potential lies not just in those conditions caused by a single genetic mutation like sickle cell disease, but any disease that has a genetic component, either in terms of susceptibility or protection.

That includes many of the major killers, including cancer, cardiovascular disease and neurodegenerative disease, plus chronic conditions like inflammatory bowel disease and rheumatoid arthritis.

All disease is on the map, says Ringeisen.

Part of what gives scientists optimism is that gene editing can be used to bestow protective DNA on a person as well as correcting unwanted mutations.

We know there is a genetic change you can make that will dramatically lower your risk of heart disease, says Urnov. How do we know that? Because of very rare individuals who have those genetic changes. And when you study lots of them, its kind of jaw-dropping. Im not going to say theyre immune to heart disease, but theyre close to it.

Doudna believes that CRISPR could even be used more to prevent disease than to treat it. Imagine a time when people get their genome sequenced and be told that you have a gene that makes you have a higher likelihood of getting cardiovascular disease, she says. But you have the option of editing your cells so that you dont have to wait to find out if youre one of the unlucky folks that is susceptible.

Today, lots of us take preventative action to protect our future health. It could be anything from eating a high-fibre diet to keep heart disease at bay, to having a double mastectomy because breast cancer runs in the family. Would you be comfortable editing your DNA to achieve the same results?

Its harder to do the cost-benefit analysis when youre talking about such experimental therapies. There are some who will say that any form of gene editing is playing God and conspiracy theories around COVID-19 vaccines show how mistrust in new technologies can spread.

There can be misrepresentations or just misconceptions embedded in the public mindset that can have a negative effect on what I think should be positive advances, Doudna says. Another example of that is the whole anti-GMO movement.

In November 2018, twin girls were born in China, the so-called CRISPR babies. Biophysicist Dr He Jiankui announced that he had created the worlds first genome-edited babies to widespread condemnation.

Chinese scientist He Jiankui hit the headlines in 2018 for using CRISPR to create the first genetically edited human babies. The press coverage soon turned sour when it emerged that the experiments had been carried out in an unethical, irresponsible manner. Jiankui is now serving a three-year prison sentence Shutterstock

He engineered mutations in human embryos that were later implanted into a woman, crossing an ethical boundary by altering the human germline, meaning the edits he made would also be passed on to future generations. Additionally, he was criticised for flouting normal safety procedures.

Jiankui claimed that he had disabled a gene called CCR5, offering protection against HIV. His critics pointed out that he could have also inadvertently caused mutations in other parts of the genome. Jiankui was jailed in China for three years at the beginning of 2020 and ordered to pay a three million yuan fine (240,000 approx), his work a stark warning to everyone in the gene-editing world.

This field is experimental and we are one severe adverse event from the entire effort being frozen, Urnov says. The painful thing for me and for tens of thousands of folks like me who have spent 40 years building human genetic engineering to treat disease, you know, this technologys now tainted with the concept of designer babies.

We have 250 million people on planet Earth with genetic disease. We should not be talking about designing anyone. We should be putting all of our attention to the fact that there are hundreds of millions of our fellow human beings that have had their fate handed to them on genetic platter, he adds.

There are other challenges to overcome, too. One of the major criticisms of Jiankuis work stems from the fact that gene editing is not yet so precise that scientists haveabsolute faith that any edit only affects the part of the genome being targeted. There can be so-called off-target effects: unintended genetic modification that occurs elsewhere on the target genome. A worst-case scenario in clinical terms might be genotoxicity, where an off-target effect causes DNA damage that could lead to cancer.

For this reason, whats known as the delivery challenge of CRISPR is a major focus of research, both at the IGI and beyond.

Emmanuelle Charpentier, who worked with Jennifer Doudna on the development of CRISPR Alamy

We can only gain confidence in the safety of these procedures by performing clinical trials, says Dr Ross Wilson, the IGIs director of therapeutic delivery. We are not so hubristic to think that we have the ability to forecast every possible outcome when a new therapeutic procedure is attempted, which is why these trials are being performed methodically and without haste.

Once we have confidence that peoples lives are being saved or transformed for the better, without incurring unwanted outcomes, the technology can be moved into other applications, reducing risks of heart disease, for example.

There are ways to offset this risk of unintended consequences of gene editing. A patients own cells can be sent to the lab for trial editing, giving an informed look at what is likely to happen inside that patients body when they are dosed.

CRISPRoff technology is also under development. This tool allows researchers to target the epigenome, rather than the genome itself. That is, scientists can turn off a particular gene without cutting a strand of DNA by instead targeting the proteins and other molecules that attach themselves to DNA and control when that gene is switched on or off. Because the genome itself is untouched, researchers expect the risk of unwanted effects to be lower.

The pandemic taught us that the public has a major craving for things to be declared safe or unsafe, but in reality its all about the balance between risk and benefit, says Wilson. That ratio looks extremely promising for CRISPR technology, and it improves each year, so the future is bright for therapeutic genome editing.

Doudnas dream for CRISPR is to make it the standard of medical care. For that to happen, one more thing needs to be addressed, and its arguably more complex than any technical hurdle: the cost of treatment.

Gene editing is, or could be, a great leveller in healthcare. But like all experimental treatments, it is research-heavy, labour-intensive and expensive. One of Doudnas fears is that it will become a boutique technology, available only to those who can afford it. This, she says, would not only exacerbate the health gap that already exists between rich and poor, but also create a new kind of health inequality: a gene gap.

The IGI is a non-profit, funded publicly and via philanthropy. Its staff talk of their work not just in technological terms but societal ones too, focused on how to maximise the reach of the technology.

Its a major problem, Urnov says. In the US, [some of] these medicines are sold for $2m, but European countries have refused to even licence them. You have a surreal situation where parents in Europe, whose children have these severe diseases, start GoFundMe campaigns to be able to pay American prices.

We have this gap between the fact that this technology is rapidly expanding in its utility and we are struggling, frankly, with how to make it equitable and affordable.

Add to that the issue of health justice, the fact that if youre born with an inherited condition, an affordable cure may be possible if enough people have the same illness. That would make it economically viable for pharmaceutical companies to invest in the research to develop new treatments.

T-cells (orange) are an important part of the immune system. Scientists could use CRISPR to modify the cells so they could potentially lead to treatments for cancer (blue), cardiovascular disease and arthritis Science Photo Library

If youre born with a rare genetic mutation, that may not be possible. There is this horrific gap between our ability to read that persons DNA and say, Yes, this is the mutation that killed your mom and will kill you, Im sorry to say, and actually developing a treatment that would help that person. Developing such personalised cures has essentially no commercial value. Nobody will ever make money, says Urnov.

Lovell-Badge agrees. The cost is a problem for it becoming really useful for public health, he says. [We need to] start off from the very beginning thinking, How can we do this in a way thats going to be more affordable? Then you approach the problem in a different way.

Unlike so much of the biotech industry, addressing this issue is a cornerstone that the IGI was built on not just developing novel treatments, but creating scalable pipelines for discovery, testing and rollout.

Our mission really has to be ensuring that technology benefits everyone, Doudna says. That was the impetus for the Innovative Genomics Institute in the first place. Many institutes, many companies, many academic labs are now developing gene editing, but not with an eye towards controlling cost and doing the science with a focus on public access to that technology. Thats really our purpose.

The genetic revolution is coming. After pioneering the technology, Doudna is adamant that when it arrives, its available to everybody. As more than one of her colleagues tells us, thats just in her DNA.

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CRISPR: A guide to the health revolution that will define the 21st century - BBC Science Focus Magazine

The film Jurassic Park (1993) made human beings dream of the possibility of resurrecting species. Almost three decades later, synthetic biology is nearly here not to rescue extinct dinosaurs, but to exterminate harmful animals.

Steven Spielbergs blockbuster film set the action on an imaginary island in Costa Rica (though it was inspired by Cocos Island).

And it is precisely on an island where the first scientific experiments could one day occur, perhaps in the next decade, according to experts and activists at the World Conservation Congress.

80% of the islands in the world share the same problem: mice. They infest crops, eat bird eggs and endanger the fragile local ecosystem.

For more than 25 years, the organization Island Conservation has been dedicated to eradicating invasive species, explains Royden Saah, representative of the organization at the Marseille congress, to AFP.

One of its latest successes was in two of the Galapagos Islands, North Seymour and the islet of Mosquera, using drones and baits. But it is an expensive and uncertain task, and the use of rodenticides can cause collateral damage.

Should we create a genetically modified mouse so that its future generations are exclusively male (or female)? asks Island Conservation on its website.

Saah coordinates a team of researchers, GBIRd, with institutions in the United States, Australia and New Zealand.

We dont have the mouse yet the conservation scientist says. But if we dont investigate, we wont be able to know the potential of this technology.

Saah points out that scientists will only perform experiments in countries interested in the technology.

With synthetic biology coming ever-closer to reality, the more than 1,400 members of the International Union for Conservation of Nature (IUCN) four years ago created a working group on the issue.

The result is a Charter of Principles on the use of synthetic biology (which includes genetic engineering) that must be voted on this week in Marseille.

The draft of the Charter reaffirms the right of any country to prohibit these activities by appealing to the precautionary principle.

Participants in the debates at the Marseille congress agreed the questions regarding synthetic biology are considerable.

I am also afraid of the potential applications of synthetic biology declared the head of the working group, Kent Redford, when presenting the groups conclusions in Marseille.

There are obvious ecological risks and concerns about genetic modifications of wild species, warns Ricarda Steinbrecher, geneticist and scientific advisor of the NGO ProNatura.

ProNatura and Friends of the Earth are some of the NGOs that have sounded the alarm in Marseille. The Charter of Principles has not been sufficiently debated, they believe.

Among other reasons, scientists dont even agree on the exact frontiers of synthetic biology.

Does a modified mouse still belong to its original species, or does it create a new one?

One of the examples proposed by scientists in favor of experimentation is to recreate the material of a rhinoceros horn, so that this animal can escape extinction.

I have not found anything that prevents further investigation, Saah says about synthetic biology.

The debate is intense, but the situation in some places is just as pressing.

Samuel Gon, science advisor for the Nature Conservancy in Hawaii, says he cant wait.

Synthetic biology is not an option. It will not arrive in time to save the birds of the islands, he explained to AFP.

Of the more than 50 endemic species of honeybirds that were known in Hawaii, only about 15 remain, five in a critical state of extinction.

Historically, Hawaii didnt have mosquitoes. When they were introduced, beginning in the 19th century, some were carrying malaria a disease that has devastated local bird populations.

Hawaiian conservation authorities are preparing to use a known technique to sterilize mosquitoes by inoculating them with a bacterium, Wolbachia.

Beyond the ecological urgency, some scientists seem irresistibly drawn to bigger dreams.

A few months ago a group of researchers claimed that they had achieved the complete sequence of the genome of a million-year-old mammoth.

The technical challenges to achieve the reliable sequence of the genome of extinct species are immense, warns the report of the IUCN experts.

Steinbrecher is even more emphatic. We have to accept that some species have gone extinct, however disappointing it may be. The main objective is to preserve what we already have.

The featured photo shows Costa Ricas Cocos Island. Photo is used for illustrative purposes.

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Synthetic biology makes ecologists dream ... and tremble : - The Tico Times

People want to know that all genetically engineered foods have been rigorously assessed for safety by Health Canada, including any new gene-edited foods

Dear Editor,

Many Canadians may not be aware that Health Canada is proposing to remove regulation from most new genetically engineered foods. Local candidates in the federal election may not even be aware!

Health Canada is proposing to let some new genetically engineered foods onto the market without any government safety checks. In fact, companies wouldnt even have to tell Health Canada that these new foods exist. Many of these unregulated foods would be produced by the new genetic engineering techniques called gene editing. But gene-edited foods need to be carefully assessed by our government, not just left to companies to evaluate safety.

Mandatory government safety assessments for all genetically engineered foods are necessary. People want to know that all genetically engineered foods have been rigorously assessed for safety by Health Canada, including any new gene-edited foods. Private companies cannot be trusted to test for safety properly!

We need transparency, independent science, and government oversight! Information and analysis by the Canadian Biotechnology Action Network are available here.

Sincerely,Julie Dupuis

Noelville

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Letter to the editor: No exemptions on genetically-engineered foods and plants - BayToday.ca

Zolgensmawhich treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decayis currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child costs $2.1 million.

While Zolgensmas exorbitant price is an outlier today, by the end of the decade therell be dozens of cell and gene therapies, costing hundreds of thousands to millions of dollars for a single dose. The Food and Drug Administration predicts that by 2025 it will be approving 10 to 20 cell and gene therapies every year.

Im a biotechnology and policy expert focused on improving access to cell and gene therapies. While these forthcoming treatments have the potential to save many lives and ease much suffering, healthcare systems around the world arent equipped to handle them. Creative new payment systems will be necessary to ensure everyone has equal access to these therapies.

Currently, only 5% of the roughly 7,000 rare diseases have an FDA-approved drug, leaving thousands of conditions without a cure.

But over the past few years, genetic engineering technology has made impressive strides toward the ultimate goal of curing disease by changing a cells genetic instructions.

The resulting gene therapies will be able to treat many diseases at the DNA level in a single dose.

Thousands of diseases are the result of DNA errors, which prevent cells from functioning normally. By directly correcting disease-causing mutations or altering a cells DNA to give the cell new tools to fight disease, gene therapy offers a powerful new approach to medicine.

There are 1,745 gene therapies in development around the world. A large fraction of this research focuses on rare genetic diseases, which affect 400 million people worldwide.

We may soon see cures for rare diseases like sickle cell disease, muscular dystrophy, and progeria, a rare and progressive genetic disorder that causes children to age rapidly.

Further into the future, gene therapies may help treat more common conditions, like heart disease and chronic pain.

The problem is these therapies will carry enormous price tags.

Gene therapies are the result of years of research and development totaling hundreds of millions to billions of dollars. Sophisticated manufacturing facilities, highly trained personnel and complex biological materials set gene therapies apart from other drugs.

Pharmaceutical companies say recouping costs, especially for drugs with small numbers of potential patients, means higher prices.

The toll of high prices on healthcare systems will not be trivial. Consider a gene therapy cure for sickle cell disease, which is expected to be available in the next few years. The estimated price of this treatment is $1.85 million per patient. As a result, economists predict that it could cost a single state Medicare program almost $30 million per year, even assuming only 7% of the eligible population received the treatment.

And thats just one drug. Introducing dozens of similar therapies into the market would strain healthcare systems and create difficult financial decisions for private insurers.

One solution for improving patient access to gene therapies would be to simply demand drugmakers charge less money, a tactic recently taken in Germany.

But this comes with a lot of challenges and may mean that companies simply refuse to offer the treatment in certain places.

I think a more balanced and sustainable approach is two-fold. In the short term, itll be important to develop new payment methods that entice insurance companies to cover high-cost therapies and distribute risks across patients, insurance companies, and drugmakers. In the long run, improved gene therapy technology will inevitably help lower costs.

For innovative payment models, one tested approach is tying coverage to patient health outcomes. Since these therapies are still experimental and relatively new, there isnt much data to help insurers make the risky decision of whether to cover them. If an insurance company is paying $1 million for a therapy, it had better work.

In outcomes-based models, insurers will either pay for some of the therapy upfront and the rest only if the patient improves, or cover the entire cost upfront and receive a reimbursement if the patient doesnt get better. These models help insurers share financial risk with the drug developers.

Another model is known as the Netflix model and would act as a subscription-based service. Under this model, a state Medicaid program would pay a pharmaceutical company a flat fee for access to unlimited treatments. This would allow a state to provide the treatment to residents who qualify, helping governments balance their budget books while giving drugmakers money up front.

This model has worked well for improving access to hepatitis C drugs in Louisiana.

On the cost front, the key to improving access will be investing in new technologies that simplify medical procedures. For example, the costly sickle cell gene therapies currently in clinical trials require a series of expensive steps, including a stem cell transplant.

The Bill & Melinda Gates Foundation, the National Institute of Health and Novartis are partnering to develop an alternative approach that would involve a simple injection of gene therapy molecules. The goal of their collaboration is to help bring an affordable sickle cell treatment to patients in Africa and other low-resource settings.

Improving access to gene therapies requires collaboration and compromise across governments, nonprofits, pharmaceutical companies, and insurers. Taking proactive steps now to develop innovative payment models and invest in new technologies will help ensure that healthcare systems are ready to deliver on the promise of gene therapies.

This article is republished from The Conversation under a Creative Commons license. Read the original article.

The Bill & Melinda Gates Foundation has provided funding for The Conversation US and provides funding for The Conversation internationally.

Image Credit: nobeastsofierce/Shutterstock.com

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Gene Therapies Are Almost Here, But Healthcare Isn't Ready for Sky-High Prices - Singularity Hub

We have problems coming to terms with our bodies.

This is true of secularists who now insist that sex and gender have nothing to do with the body. And it is true of Christians, who tend to be squeamish in talking about the body.

And yet many of todays most important issues have to do with the body: abortion, COVID policies, health care, genetic engineering, transgenderism, sex, pornography, homosexuality, marriage, parenting, race, virtual reality, virtual communities, the metaverse. . . .

Both Christians and non-Christians seem to be caught in a web of Gnosticism, that ancient heresy that taught that the body doesnt exist or, at most, doesnt matter. This worldview manifested itself in the two opposite, but related, extremes of hyperspirituality (pursuing the spiritual while suppressing and trying to escape from the physical) and moral permissiveness (indulging all physical desires, since only the spiritual counts, it doesnt matter what the body does). And so it is today.

Christianity counters Gnosticism with its doctrines of creation, incarnation, sacraments, and vocation. But those teachings do not carry the weight they used to. In order to deal with the issues it now faces and to help Christians navigate through the increasingly Gnostic culture, the church needs to cultivate a theology of the body.

This has become very influential in conservative Catholic circles. I have dipped into it found it well-worth reading, but it is, of course Catholic, both in its philosophical approach to theology and in its doctrinal presuppositions. That book has launched a myriad of other theological treatments of the body, including some from Protestants.

But now we have by the Australian theologian John W. Kleinig. Dr. Kleinig is well-known in confessional Lutheran circles. (Im currently working with him on his monumental translation of J. G. Hamanns London Writings, soon to be released. ) But he is a resource that all Christians can draw on, and, beginning with this book, published by the evangelical publisher Lexham Press, Im sure he will be.

I can think of no other author who can take on this subject in a more Biblically-rich, Gospel-centered, scholarly, readable, engaging, and devotional way than John Kleinig.

Here are his chapters:

I have bought my copy and will give the book a proper review once I read it thoroughly.

In the meantime, here is the publishers summary and endorsements (my bolds) from Amazon:

Read this article:
A Protestant Theology of the Body | Gene Veith - Patheos

A molecular genetic technique used for the direct manipulation, alteration or modification of genes or genome of organisms in order to manipulate the phenotypes is called genetic engineering.

Or in other words, we can say,

Genetic engineering is a technique using which the genetic composition of an organism can be altered.

The technique is often known as genetic manipulation, genetic modification or genetic alterations, broadly it is categorized as genetic engineering.

In this technique, a recombinant DNA is constructed and inserted into the host genome using a vector. Or we can delete some mutant sequences from a genome.The first recombinant DNA was constructed by Paul Berg in 1972.

Using the genetic engineering technique genetically modified organisms can be constructed which are economically very important for us.

It is employed for the production of improved plant species, therapeutic drugs or proteins, prevention of inherited genetic disorders and construction of a genetically modified organism.

In the present article, we will our major talk is genetic engineering and its applications. The content of the article is,

Humans are manipulating the genetic material of many organisms for long. Using selective breeding and cross-hybridization, economically important plant species were created by us.

The purpose of developing the genetic engineering or genetic manipulating technique is to produce organisms or phenotypes which are useful to us. Genetic engineering techniques are used for,

In genetic engineering, two different cells DNA are combined and inserted into the host genome via vector. Important components of the gene manipulation experiments are explained here.

Gene of interest: A DNA sequence which we want to insert in our target cells.

Vector: using the plasmid DNA like vectors the gene of interest is inserted into the host genome. Vectors are kind of vehicles which transfer the genetic material.

Target cells: target cells are the population of cells whose genome we wish to manipulate or change.The general process of gene therapy.

A technique used to insert or delete a mutant gene or to manipulate a genome of an organism is known as genetic engineering.

The term genetic engineering was first used by the science-fiction novelist, not by any scientist.In the year, 1951, Jack Williamson used the term genetic engineering for the first time in his novel Dragons island.

Soon after that, the molecular structure of the DNA was discovered by Watson and Crick, although the genetic experiments were popular since the time of Mendel.

The first recombinant DNA was constructed by Paul Berg in 1972. In the same year, Herbert Boyer and Stanley Cohen performed gene transfer experiments.In 1974, Rudolf Jaenisch had created genetically modified mice, the first time in the history of genetics.

After the success of Rudolf, the genetically modified or genetically engineered tobacco plant species was developed in 1976.

During this period (between 1960 to 1990) restriction digestion, ligation and PCR like techniques were discovered which gave wings to genetic engineering technology.

Related article: What is a genome?

Recombinant DNA- A recombinant DNA technology is a type of genetic engineering technology in which an artificial DNA molecule is constructed by ligating two different DNAs using physical methods.For that, the gene of interest is inserted into the plasmid vector and used for gene transfer experiments.

Gene delivering-Gene delivering technique is employed for the insertion of a gene of interest into the host genome.

Electrophoration, solicitation and viral vector-mediated gene transfer, liposome-mediated gene transfer, transposon-mediated gene transfer are some of the methods used for that.

Gene editing- A gene-editing technique is used to edit the genome in which an undesired DNA sequence is removed or a new gene can be inserted into the host genome. CRISPR-CAS9, TALEN and ZFN are some known gene-editing tools used in gene therapy experiments.

Read more:What is gene editing and CRISPR-CAS9?

The genetic engineering technique is used for many different purposes thus we must have to decide first the purpose of the experiment.The entire process of genetic engineering can be divided into 5 broader steps:

The gene must contain a sequence of DNA that we want to study and for that, a gene has some special characteristics. A candidate gene should have high GC content and a lower repetitive DNA sequence.

In addition to this, the gene of interest must not be too long- only a few kb genes can be successfully inserted.Longer the gene higher the chance of failure. The candidate gene must have a start and stop codon in it. Related article:What is The Genetic Code?

Now, the gene of interest can be isolated from the rest of the DNA using either restriction digestion or polymerase chain reaction.

The restriction endonucleases are the bacterial enzyme having the power to digest DNA sequence at a specific location.Using a specific type of restriction endonuclease we can cut and isolated our gene of interest.

The restriction digestion method is explained in our previous article: What is restriction digestion?

In the polymerase chain reaction, using the information of the gene sequence, the gene of interest or the candidate gene is amplified in the thermocycler.

The machine, using the polymerase chain reaction makes millions of copies of a gene of our interest. Through the process of agarose gel electrophoresis, the amplified gene is isolated.

If the gene of interest is well studied, previously, then the information of a gene is accessible in the genetic library and we can use it for the artificial synthesis of a gene of our interest. (using the genetic library information, the gene can also be artificially synthesized)

In the next step, perform DNA purification, if required. Now our DNA is ready to insert in a plasmid.

Selecting plasmid for the genetic engineering experiment is one of the crucial steps in the entire experiment.Before selecting the plasmid, we must understand why the plasmid is used in the gene transfer experiments.

The plasmid DNA is a circular, double-stranded cytoplasmic DNA of the bacteria that replicate independently.

Scientists are using it as a vehicle for transferring the gene of interest to the target location in the genome.It can efficiently transfer the gene at the target location. The structure of plasmid is explained in the figure below,The general structure of the plasmid DNA used in recombinant DNA technology.

Related article: What is a plasmid?

Preparation of plasmid:

Select the plasmid which suits your experiment.

The plasmid must have the origin of replication, promoter region, antibiotic resistance gene and other important sequences.Using the restriction digestion method, an insertion site is introduced in the plasmid at which our gene of interest is ligated.

Utilizing the T4 DNA ligase like power sealer, the DNA of our interest in inserted and ligated in the plasmid.Along with the plasmid, a selectable marker is also introduced in the plasmid DNA to identify the recombinant DNA.

In addition to this, a promoter region and terminator sequences are also included in the plasmid for the effective expression of a gene of our interest. A plasmid with our gene of interest and some other important sequences is now referred to as a recombinant DNA molecule.

Now our recombinant DNA is ready for for the expression.

If we are performing gene cloning than the plasmid is inserted in the bacterial host, for that generally E.Coli are commonly used.Once the bacteria starts dividing, our recombinant plasmid DNA is also replicated along with it.

Now we have the multiple copies of our plasmid DNA which are extracted using the plasmid DNA extraction kit and used for the transformation experiments.The process of Genetic engineering.

Transporting the recombinant DNA into the recipient cell or the host genome is yet another tedious and difficult task.Various methods for recombinant DNA insertion is used for various cell types because a single method cant used for all cell types.

Using stress- bacteria easily uptake the plasmid DNA using some stress factors such as heat or electrical sock.

Microinjection- a sharp needle is used for insertion of DNA directly into the nucleus of a cell, however, the method is less effective and required a higher level of expertise for that.

Electroporation- one of the best methods having a great success rate is the electrophoration method in which the recombinant DNA is inserted into the host genome by permeabilizing the cell with electrical current.

We have covered a whole article on it. Read it here:Electroporation- A Modern Gene Transfer Technique.

Sonication- sonication is yet another good method sometimes used in the gene transfer experiment in which the recombinant DNA is inserted into the target cell using ultrasonic waves. The ultrasonic waves also increase the permeability of cells.

Liposome mediated gene transfer- Using an artificial cell-like outer coat known as a liposome- recombinant DNA can be inserted in the host genome.

Gene transfer using bacterial infection-This method is one of the popular methods and routinely used in plant genetic engineering experiments. Here, the plant species is infected with the transformed bacteria for inserting a gene of interest.

Agrobacterium tumifecian is utilized to insert recombinant DNA into the plant cell. A gene of interest is inserted into the Ti- plasmid of the Agrobacterium. The plant cells are infected by this bacteria cell culture and the transformed cells are regenerated using the plant tissue culture methods.

Chemical in gene transfer- Some metal ions, chemicals, and solutions of different chemicals are also employed in the gene transfer experiments, however, the success rate is too low as compared with the other methods.

Our work is still not completed.

Now we have to conform, whether the recombinant DNA is inserted in our target cell or not. Various molecular genetic technologies are used for that.In the traditional culturing method, the presence or absence of a selectable marker is used to differentiate transformed cells from the untransformed cells.

Although, it is not necessary for the PCR based detection method.The polymerase chain reaction-based detection method is widely accepted more trusted than other methods.

DNA is extracted from the transformed cell and amplified using the primers complementary to our gene of interest or our recombinant DNA.

If the recombinant DNA is present it surely amplified otherwise no amplification obtained. For the two factor conformation, one primer set complementary to recombinant DNA specific and one set of primer complementary to the selectable marker sequence are taken and multiplex PCR is performed.

For validating results, amplification must be obtained in both the reaction.

But wait a minute!

What happened if any mutation occurred during the experiment in our gene of interest? Because the PCR can only amplify the DNA.We must need sequence information to detect the mutation.

For that, the DNA sequencing method is used.

DNA is extracted from the transformed cells and the gene of interest is amplified using the PCR. Now the PCR amplicons are used for DNA sequencing in which using the fluorescent chemistry the sequence of our gene of interest is orderly determined.

Once all the parameters for determining the gene of interest fulfilled, our cells are now ready to inject in the host organism or for tissue culture experiments.

Now coming to the important point of this topic, What is genetic engineering used for?

Genetic engineering has great industrial and agricultural value. It is practiced in medicine, genetic research, agriculture, crop improvement, and for production of therapeutic drugs.

It is also used in the development of genetically modified organisms.Here we are discussing some of the important applications of genetic engineering.

The recombinant DNA technology is used in the crop improvement and development of new economically important traits. Some of them are:

A classical example of it is the BT cotton- one of the types of genetically modified species provides resistance to the plant against bacillus thuringiensis.

Process of developing genetically modified plant species:

A gene of interest is isolated from the organism using restriction digestion or amplified by the polymerase chain reaction.Recombinant DNA is constructed by inserting a gene of interest into the plasmid, here the T- plasmid is used.

In the next step, the T- plasmid is inserted into the agrobacterium.In the last step, the plant species is infected with the transformed bacterial cells and cultured.The entire process of it is shown in the figure below,Agrobacterium-mediated gene transfer in plant species.

GMF- genetically modified food is another best application of genetic engineering in which economically important food products are constructed using recombinant DNA technology.

The classical example of it is Flavr Savr tomato, a genetically modified tomato species made up of the antisense RNA technology.It has great economic values as the GM- tomato can easily be transported from one place to another place.

Another important application of genetic engineering is genetically modified or genetically engineered food.

The quality of some of the food products such as cotton, corn, and soybeans are improved using the present recombinant DNA technology.The aim of developing genetically modified crops or plant species is to make them economical important, nutritious, protein-rich, disease, and stress resistance.

Even, using genetic engineering and tissue culture techniques insecticides resistance plant species in tobacco, potato, corn, and cotton are developed.

In addition to this, some modified plants capable of generating their own fertilizers can also be created using the present genetic modification technique.

Transgenic model organisms are developed to test different parameters- the function of certain genes can be determined by designing the transgenic microorganism and animal models.

Harmful pathogens and insecticidal pasts can be destroyed using genetically modified microorganisms which are capable of degrading toxics.

Medicinal applications:

Low-cost drugs, hormones, enzymes, and vaccines are created using genetic engineering tools.

The anti-blood-clotting factor is the best example of it in which the plasminogen activating enzyme which is capable of dissolving the blood clot is artificially designed and used in the patients with coronary artery disease or heart attack.

Other examples are two other therapeutic proteins somatostatin and lymphokines which are worked against several disease conditions and can be synthesized artificially.Insulin is yet a classic example of a therapeutic protein designed using genetic engineering technology.

A gene for insulin is isolated by restriction digestion or through PCR and inserted int the plasmid.The recombinant plasmid DNA is immediately inserted into the bacterial or yeast cell in which the plasmid is multiplying.As the microorganism starts dividing it starts making artificial insulin.

A large amount of insulin produced using the same technique at an industrial scale. The detailed outline of insulin production is shown in the figure below,Production of insulin using genetic engineering technology.

The commercial production of insulin started after the FDA approval in 1982.

Recombinant vaccines:

Vaccines against smallpox, herpes simplex virus and hepatitis are produced using the genetic engineering technique.The vaccines are the inactivated viral particles used to induce an immune response against that pathogen, however, the chance of contamination is high in it.

Using the recombinant DNA technology scientists has created a unique type of vaccines that only contains the DNA for viral coat protein thus the pathogen can never be activated again.The main advantage of it is that it is safer, contamination-free and more reactive.

Genetic engineering in gene therapy:

Using the gene therapy or gene transfer technique, inherited genetic disorders can be cured. Cystic fibrosis, Duchenne muscular dystrophy and sickle cell anemia like gene therapies are now under the final clinical trial phase and ready to use on patients.

In the gene therapy, a faulty, non-function or mutated gene is replaced with the wild type one using the same technique as explained above.

We have covered amazing articles on gene therapy, read it here:

More:
What Is Genetic Engineering?- Definition, Types, Process And ...

Genetic engineering has been a topic of varying contention for years. Recently, though, there was new fuel thrown on the fire with a series of experiments done with Clustered Regularly Interspaced Short Palindromic Repeats or CRISPR. CRISPER is commonly used to refer to a variety of systems that can target specific stretches of DNA allowing scientists to delete particular portions of the genetic code or insert new genetic material into a previously existing genome. The precision of CRISPR allows geneticists to permanently modify an organisms genetic code with previously unheard of accuracy. This technology is based on the naturally occurring abilities of some bacteria.

Even though debate has surrounded genetically engineered crops and genetic experiments in animals, for most people, the controversy surrounding genetic experimentation has been largely ignored. The ethics of genetic engineering, however, are back in the spotlight.

Early this year, a team of scientists successfully performed genetic modification on a fertilized human embryo using CRISPR. In vitro fertilization and gene therapy have involved elements of genetic engineering nearly since their conception, but the CRISPR experiments are the first time humanity has been confronted with human germline genetic modification. Germline modification is used to refer to genetic changes that would be passed down to an organisms offspring. Any genetic alterations done to a parent would appear in children and grandchildren. Naturally, this has once again raised the question of whether genetic engineering is ethical.

Books have been written on the ethics of all sorts of genetic engineering, but the controversy reignited by the CRISPR studies focuses on genetic modification of humans. For decades, accurate and feasible human genetic engineering was something out of a science fiction novel. Depending on a persons opinion on genetic modification, genetically engineered humans were a distant fantasy or specter that loomed centuries down the road.

The CRISPR experiments did not use viable embryos and so no child has resulted from the study, but the CRISPR team proved that genetically modified humans were possible. The ethics of human genetic engineering is no longer a question to be dealt with in some remote future, but a debate that is very relevant now. So, what are the benefits and dangers of human genetic engineering?

Genetic testing is not terribly new. Amniocentesis has been a staple of modern pregnancies for many years, and many at-risk people choose to be tested for genetic diseases such as Huntingtons disease. Improved genetic testing would lead to earlier diagnosis of such diseases. Earlier diagnoses would allow people destined to develop genetic diseases to make the most of their healthy years. Those who did not carry a genetic disease would be able to set their minds at ease.

Human genetic engineering has the potential to do more than identify a faulty gene. Improvements in technologies such as those used in CRISPR have the potential to correct the genetic errors that cause genetic diseases in the first place. Furthermore, germline genetic engineering could lead to the eradication of certain genetic diseases all-together.

Opponents of human genetic engineering argue that some faulty genes actually serve important purposes. The classic example of a useful genetic defect is sickle cell disease. Sickle cell disease, also known as sickle cell anemia, is caused by a genetic flaw that causes some red blood cells to be sickle shaped. The sickle shaped cells are prone to causing blockages in the circulatory system resulting in pain, stroke, cardiac arrest and death. Sickle cell disease, though, only presents if a person carries two copies of the sickle cell gene. If a person only has one copy, they have normal red blood cells and some protection against malaria. Were the sickle cell gene to be universally corrected, malaria-related deaths would increase dramatically.

Critics of genetic modification in humans also point out that genetic engineering is still relatively new. The potential long-term consequences of altering the human genome are still unknown. Changes to the human genetic code could potentially create new genetic diseases or genetic defects that, in the case of germline engineering, would persist for generations.

The specter of designer babies is commonly raised by opponents of human genetic engineering. Advancement in genetic modification techniques could allow parents to influence their childs eye color, hair color, height, intelligence and athleticism. It sounds like something out of a dystopian sci-fi story, but the possibility of designer babies is not as far-fetched as it sounds. Researchers have isolated genes that influence a persons ability to gain muscle mass, and professional athletic associations have struggled to control gene-doping, the non-therapeutic use of cells, genes or genetic elements to enhance performance. Parents can already select the sex of their child in certain areas of the world and, while the genetics of intelligence have not yet been determined, they have long been a topic of interest in the scientific community.

This ability to design a child, genetic engineering critics argue, would lead to a generation of children whose very make-up was shaped by parental whims, market forces, constantly shifting standards of beauty and societal preferences. It could lead to a constantly deepening divide between those who were genetically enhanced or improved and those who were not. This divide might follow current class lines depending on the monetary cost of genetic engineering. This incorporation of a genetic component to the haves and have nots could also lead to a new form of eugenics or even the split of humanity into two distinct species.

Proponents of genetic engineering, however, argue that such claims have little basis in fact. Sex is based entirely on the presence or absence of the Y chromosome while traits such as hair and eye color are controlled by many different genes. Furthermore, the genetics of intelligence are still something of a mystery.

Some genetic diseases have a very high potential of being inherited. A person with Huntingtons disease, for example, has a 50 percent chance of passing the faulty gene on to their child. In such situations, parents may decide not to have children due to a fear of passing on the genetic disorder regardless of how much they wish to have a child. Human genetic engineering has the potential to lower the risks for such couples. Improvements in technology such as CRISPR could allow scientists to correct a faulty gene. Genetic engineering could also be used to lower the dangers of high-risk pregnancies by insuring the genetic health of the fetus.

Those who are against human genetic engineering argue that alternatives exist for parents with a highly inheritable genetic disease. Surrogacy and adoption are options that do not involve invasive changes to an embryos genome.

Opponents of human genetic engineering claim that genetic modification could eventually become a tool of discrimination and prejudice. Researchers have long been curious what genetic predispositions, if any, influence a persons tendency toward anger, violence, hatred and addiction. Genetic tests for such undesirable, but non-medical, traits could lead to discrimination against a person who carried a violence gene, regardless of whether or not the person has ever acted in a violent manner. Furthermore, if genes linked to such social undesirables were found in higher concentrations in certain ethnic groups, racial prejudice would suddenly have a genetic rationalization.

Proponents of human genetic modification argue that genetic testing could be kept confidential to avoid discrimination against individuals. Genetic information would be part of a persons medical record and therefore privileged information.

Despite the potential abuses, those who favor genetic engineering argue that research into genetic influences on violence and addiction should continue. Identifying genetic predispositions towards addiction could help people with a high likelihood of developing a substance abuse problem manage their risks more effectively. Studying genetic links to violence could also lead to the identification of the gene pattern responsible for psychopathy as current research points to the disorder having a hereditary component.

Human genetic engineering has the potential to lead to a longer average lifespan. Researchers have identified the portion of human chromosomes responsible for determining how many times a cell can divide and, thus, how long an organism will live. Human genetic modification could alter this portion of the chromosomes, extending a persons lifespan.

Opponents of human genetic modification point out that the earth is already struggling to support a population of 7.2 billion people. Lengthening the average human lifespan would place even greater stress on an already overburdened planet.

This is one of the most expected controversies in human genetic research. Human genetic experimentation requires the use of human DNA. As with stem cell research, that DNA is usually found in donated eggs, sperm and embryos. This, naturally, runs headlong into the explosive question that has kept the debate over abortion raging for years: when does human life begin?

People who believe that human life begins at conception see the use of fertilized human embryos in medical research, such as the CRISPR study, as abhorrent. To those who hold that life begins at conception, experimentation on a fertilized human embryo is nothing short of sickening violation if not torture.

The use of human embryos in genetic experiments is not universally supported by those who believe that an embryo cannot be considered human until later in development. As of now, embryos used in genetic research are destroyed when the study is complete. This is in part because the scientists working on such research recognize that the long-term consequences of genetic modification are not yet understood. The knowledge required for a woman to safely carry a genetically engineered child to term simply does not exist yet. Still, the waste of human embryos or donated eggs grates on people, especially those who struggle to conceive. Some who rely on fertility treatments or in vitro fertilization see the use of embryos in medical research as a waste of viable eggs.

Proponents of genetic research are quick to point out that the embryos used in the CRISPR experiments were not truly viable. Had any one of the embryos been implanted in a womans womb, the embryo would not have survived to term. Some scientists argue that healthy, viable embryos would not be involved in such genetic modification research until closer to clinical trials. The waste of some viable embryos would be inevitable but would not seriously begin until science was preparing to implant a genetically modified embryo in a woman.

This comes up in nearly every argument involving genetic engineering, regardless of whether it is corn or cows or children being modified. Some people who believe that human beings especially have a right to be unmodified, maintain that altering the human genome is equivalent to playing God. Playing God has a different meaning to every individual with some people claiming than any genetic modification involves a moral and spiritual trespass. On the other side of the spectrum are religious authorities who claim that genetic experimentation is within Gods gift to mankind of dominion over the earth. So far, few religious authorities see the question of genetic engineering as black-and-white. Most allow for genetic engineering that would preserve human life but frown upon the use of genetic modification for non-medically necessary uses such as sex selection.

The ability to select for or against specific traits could affect the genetic diversity of the human species. Opponents of genetic modification argue that germline human genetic engineering would decrease the genetic diversity of the human species as certain traits would be seen as more desirable than others. This decrease in biodiversity would leave the population as a whole more vulnerable to diseases and changes in the environment.

Supporters of human genetic modification argue that genetic engineering could be used to increase genetic diversity. Geneticists could select for traits that would normally be lost in the random shuffle of genes. Human genetic engineering could also theoretically be used to create entirely new traits thus increasing genetic diversity beyond its original starting point.

Regardless of whether human genetic engineering is a marvel or an abomination, the technology to achieve it exists. Human genetic modification is possible and the world knows it. Proponents of human genetic engineering argue that human genetic modification is now inevitable. Someone, somewhere will improve and use the technology. Banning further research, testing and eventual usage would keep the technology from being done in a safe environment. Genetic modification would be driven underground and sold on the black market. Permitting human genetic engineering would also allow organizations to regulate the technologys usage rather than leaving it to become part of the medical tourism industry. Men and women already travel internationally to receive risky surgeries, cheaper pharmaceuticals or procedures illegal in their home countries. The same thing would happen to human genetic modification.

Experiments involving the human genetic modification have revealed information about the human genome that would not have otherwise been discovered. The CRISPR studies, for example, revealed that a human embryo can sometimes repair its own faulty DNA without medical intervention. This phenomenon had never been observed before and scientists had not imagined it was possible. Such discoveries increase geneticists understanding of the human species and genetics as a whole. Further studies of the phenomenon of self-repaired DNA alone could lead to revolutionary treatments for diseases such as Huntingtons, Tay-Sachs and dozens of types of cancer. For proponents of genetic engineering, the information gained through human genetic research is invaluable. Opponents of human genetic modification, however, argue that the ends do not always justify the means.

Both opponents and proponents of human genetic engineering have valid points and strong arguments defending their position. There is a great deal of good to be gained from research into human genetic engineering, but there is also enormous potential for abuse. A genetically engineered human being is not yet safely possible, but the CRISPR studies have taken the concept out of science fiction and planted it squarely in todays reality. What society will decide to do with the potential to modify the human species at its fundamental level has yet to be determined, but the debate over genetic engineering has been reignited, and it suddenly has far more personal consequences for mankind.

The rest is here:
Is Genetic Engineering Ethical | Genetic Engineering Debate ...