Category Archives: Transhuman News

For the first time in the United States, scientists have edited the genes of human embryos, a controversial step toward someday helping babies avoid inherited diseases.

The experiment was just an exercise in science the embryos were not allowed to develop for more than a few days and were never intended to be implanted into a womb, according to MIT Technology Review, which first reported the news.

Officials at Oregon Health & Science University confirmed Thursday that the work took place there and said results would be published in a journal soon. It is thought to be the first such work in the U.S.; previous experiments like this have been reported from China. How many embryos were created and edited in the experiments has not been revealed.

The Oregon scientists reportedly used a technique called CRISPR, which allows specific sections of DNA to be altered or replaced. It's like using a molecular scissors to cut and paste DNA, and is much more precise than some types of gene therapy that cannot ensure that desired changes will take place exactly where and as intended. With gene editing, these so-called "germline" changes are permanent and would be passed down to any offspring.

The approach holds great potential to avoid many genetic diseases, but has raised fears of "designer babies" if done for less lofty reasons, such as producing desirable traits.

Last year, Britain said some of its scientists could edit embryo genes to better understand human development.

And earlier this year in the U.S., the National Academy of Sciences and National Academy of Medicine said in a report that altering the genes of embryos might be OK if done under strict criteria and aimed at preventing serious disease.

"This is the kind of research that the report discussed," University of Wisconsin-Madison bioethicist R. Alta Charo said of the news of Oregon's work. She co-led the National Academies panel but was not commenting on its behalf Thursday.

"This was purely laboratory-based work that is incredibly valuable for helping us understand how one might make these germline changes in a way that is precise and safe. But it's only a first step," she said.

"We still have regulatory barriers in the United States to ever trying this to achieve a pregnancy. The public has plenty of time" to weigh in on whether that should occur, she said. "Any such experiment aimed at a pregnancy would need FDA approval, and the agency is currently not allowed to even consider such a request" because of limits set by Congress.

One prominent genetics expert, Dr. Eric Topol, director of the Scripps Translational Science Institute in La Jolla, California, said gene editing of embryos is "an unstoppable, inevitable science, and this is more proof it can be done."

Experiments are in the works now in the U.S. using gene-edited cells to try to treat people with various diseases, but "in order to really have a cure, you want to get this at the embryo stage," he said. "If it isn't done in this country, it will be done elsewhere."

There are other ways that some parents who know they carry a problem gene can avoid passing it to their children, he added. They can create embryos through in vitro fertilization, screen them in the lab and implant only ones free of the defect.

Dr. Robert C. Green, a medical geneticist at Harvard Medical School, said the prospect of editing embryos to avoid disease "is inevitable and exciting," and that "with proper controls in place, it's going to lead to huge advances in human health."

The need for it is clear, he added: "Our research has suggested that there are far more disease-associated mutations in the general public than was previously suspected."

Hank Greely, director of Stanford University's Center for Law and the Biosciences, called CRISPR "the most exciting thing I've seen in biology in the 25 years I've been watching it," with tremendous possibilities to aid human health.

"Everybody should calm down" because this is just one of many steps advancing the science, and there are regulatory safeguards already in place. "We've got time to do it carefully," he said.

Michael Watson, executive director of the American College of Medical Genetics and Genomics, said the college thinks that any work aimed at pregnancy is premature, but the lab work is a necessary first step.

"That's the only way we're going to learn" if it's safe or feasible, he said.

Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP

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In US first, scientists edit genes of human embryos - ABC News

On any bustling city street, in the middle of the afternoon, its probably the case that half or more people are wearing earbuds, while the rest are abiding the noise pollution all around them. No one thinks twice about it, either.

The reality is that from a very young age, our hearing is now under assault.Little wonder that one in eight people in the United States aged 12 years or older has hearing loss in both ears, based on standard hearing examinations. By age 65, one in three people has hearing loss.

The problem will only grow as more people flock to city centers. According to recent United Nations data, roughly 54 percent of the worlds population lives in urban areas right now, and that number is expected to hit 66 percent by 2050, meaning cities could take in another 2.5 billion people, accounting for population growth.

With any luck, in our lifetimes, potentially soon, even, some of this hearing loss will be fixable not with hearing aids or cochlear implants, which arent available to everyone and dont work for a high percentage of people anyway. Scientists think instead that the combination of human genetics and single cell expression profiling has brought us to the point where medicine can help fix hearing. In fact, there are right now a small number of outfits quietly racing to develop the first approved drug for hearing loss, and if, like us, you live with a playlist unspooling in your ears part of each day, you should be rooting for them to succeed.

Some are further along than others, as a recent Xconomy piece observed. San Diego-basedOtonomyhas a drug for swimmers ear that could be approved this year. Meanwhile,Auris Medical, a Swiss biotech whose tinnitus candidate last year failed to beat a so-called dummy therapy in a Phase 3 trial, is currently working on other hearing loss conditions.

Both Otonomy and Auris Medical are publicly traded, but they have peers (and rivals) in the still-private world. Two young startups to watch they have strong founders and top venture backing on their side areFrequency Therapeutics and Decibel Therapeutics, both based in Boston.

Decibel Therapeutics was incubated by the powerhouse investment firm and incubator Third Rock Ventures. Along with SROne (a venture fund that counts GlaxoSmithKline as its sole investor), Third Rock provided the company with $52 million to get started in 2015, and it more recently raised an undisclosed amount of funding from GV.

Anthony Philippakis, a venture partner at GV who led the deal, says one aspect of Decibel that excited him is its portfolio approach, with some of its focus on single cell genomics, some on human genetics, some on direct-to-patient clinical trials and some on generating phenotypic data about the hearing system. (Philippakis seems to have embraced a portfolio approach to his own work. In addition to working with GV, hes a cardiologist at Brigham and Womens Hospital, and the chief data officer at Broad Institute of Harvard and MIT.)

As Decibels CEO, Steve Holtzman explains of the companys modus operandi: If you make investments in a broad discovery and translational medicine platform for drug discovery not just take a shot on goal with a single drug or assay you have a better chance to dominate the space.

Indeed, Holtzman who is focused first on hearing loss in millennials but who has ambitions to tackle hearing loss across the age spectrum says Decibel is working on drugs to reduce against drug toxicity [which can cause hearing loss], drugs to repair hair cells [in the inner ear] , drugs that are looking at other aspects of hearing that may involve the [central nervous system], and drugs focused on regeneration [versus just cellular repair].

Our play is much broader than that of any other firm, adds Holtzman, who has worked in the biotech industry for roughly 30 yearsand helped co-found the company with Third Rock.

Holtzman doesnt mention Frequency Therapeutics specifically, but its probably no coincidence that Frequency which recently raised $32 million in Series A led by CoBro Ventures, an investment firm formed by tech entrepreneur Marc Cohen and his brother Alain is taking a rather different approach.

The vision for the company started three years ago, says CEO David Lucchino. Bob Langer, a renowned biomedical engineer at MIT, had teamed up with peer Jeffrey Karp of Harvard Medical school on research showing that cells in the inner ear theyre called progenitor cells, and each of us is born with a fixed number of them could potentially be manipulated to create new inner ear cells.

Why thats important: these inner ear hair cells absorb sound and convert it to electrical impulses.

Frequencys lead program is focused on treating chronic hearing loss by regenerating cochlear hair cells with combinations of easily made drug molecules. But one challenge, among many, is whether this growth can happen in vivo. Why no one yet knows: Langer and Karps earlier findings involved human cochlear tissue that had been removed from a 40-year-old, whod had to have it removed in order for surgeons to get to a tumor.

Though the researchers witnessed an encouraging response from the tissue after dosing it with drugs, shooting medicine directly into someones ear and getting it to grow new cells is a giant leap from that starting point. Lucchino acknowledges, too, that determiningwhat amount of medicine to inject, or how often to inject it, would present a whole new host of other obstacles to overcome.

Given the various unknowns, its perhaps no surprise that Lucchino who worked as a venture capitalist with Polaris Partners before founding an earlier biosciences company 10 years ago says Frequency plans to focus on more than hearing eventually.

Its our first focus, but we view ourselves as a next-generation regenerative medicine player. And hearing is a wonderful place for us to start.

Either way, Frequency might find encouragement in other initiatives that are making meaningful strides. For example, two projects similarly involving endogenous cells (meaning already present in the body), are now in clinical development at the Swiss company Novartis.

The programs which came out of the small-molecule regenerative program of Scripps Research Institute of La Jolla, Calif. are focused on other areas, including treating multiple sclerosis and gastrointestinal problems. Some academics see the approach as potentially very powerful, however. If only it works. Stay tuned.

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A race is underway to repair our hearing with medicine ... - TechCrunch

America reportedly has moved ahead in a controversial race to tinker with human DNA but the scientific feat is shrouded in unanswered questions.

The MIT Technology Review published on Wednesday a news report about the first-known experiment to create genetically modified human embryos in the United States using a gene-editing tool called CRISPR.

Shoukhrat Mitalipov, director of the Oregon Health & Science Universitys Center for Embryonic Cell and Gene Therapy, reportedly led the new research. Mitalipov and the university would not confirm details of the research to CNN.

Results of the peer-reviewed study are expected to be published soon in a scientific journal. No further information will be provided before then, according to an emailed statement from the universitys press office. Another researcher cited in the MIT report, the Salk Institutes Jun Wu, did not reply to CNNs request for comment.

Mitalipov also declined to comment in the MIT Technology Review report, referencing the research results have not been published yet in a peer-reviewed scientific journal, which is considered the gold standard for scientific research. The author of the MIT report would not confirm to CNN whether he had seen the paper.

Previously, Mitalipov and his colleagues reported the first success in cloning human stem cells in 2013, successfully reprogramming human skin cells back to their embryonic state. In 2007, a research team led by Mitalipov announced they created the first cloned monkey embryo and extracted stem cells from it.

The MIT Technology Review reported the researchers in Portland, Oregon edited the DNA of a large number of one-cell embryos, specifically targeting genes associated with inherited diseases in those embryos. The MIT Technology Review could not determine which disease genes had been chosen for editing in the new research.

Im not surprised that they were looking at genetic diseases to try and see if they could target them, because thats exactly where I think the future inevitably leads, said Arthur Caplan, a professor and founding head of the division of bioethics at New York University Langone Medical Center, who was not involved in the research.

CRISPR research and controversy

Previously, scientists in China were the first in the world to reveal attempts to modify genes in human embryos using CRISPR. Three separate papers were published in scientific journals describing various studies in China on gene editing in human embryos.

When it comes to the new research, my reaction was this is an interesting incremental step and, boy, I bet its going to get blown up as being more important than it is, said Hank Greely, professor of law and genetics at Stanford University, who was not involved in the research. Its not the first time anybody has CRISPR-ed human embryos. Its not the first time anybodys CRISPR-ed viable human embryos. Its certainly not the first time people have CRISPR-ed viable mammalian embryos. Its the first time its been done in the US, but the embryos dont care where they are.

Yet, the research has already generated attention and controversy.

This is pushing the research faster than I thought we would see, said Dana Carroll, professor of biochemistry at the University of Utah, if the MIT Technology Review report rings true. Carroll has used CRISPR in his own studies but was not involved in the new research.

He pointed out the new research reportedly involved earlier, more delicate embryos, and CRISPR reportedly was still demonstrated as efficient.

From the perspective of research that would ultimately make germline editing safer and more effective, the earlier embryos will provide more relevant information, he said.

CRISPR an acronym for clustered, regularly interspaced, short palindromic repeats allows scientists to cut and edit small pieces of DNA at precise areas along a DNA strand, essentially modifying DNA.

Once scientists discovered they could develop a system that modifies pieces of DNA, they tested the gene-editing technology in microbes, then non-human mammals, then non-human primates and then, by 2015, human embryos.

The controversy surrounding gene-editing in human embryos partly stems from concern the changes CRISPR makes in DNA can be passed down to the offspring of those embryos later in life, from generation to generation. Down the line, that could possibly impact the genetic makeup of humans in erratic ways.

There is also considerable concern about off-target effects, such as making mutations at sites in the genome other than the intended target, Carroll said.

In other words, an edit made in one area of DNA possibly could cause problems in another, as a ripple or domino effect, which could be concerning.

Some CRISPR critics also have argued gene-editing may give way to eugenics and to allowing embryos to be edited with certain features in order to develop so-called designer babies.

Though, not all experts are too concerned.

Treating diseases

Some people are worried about wheres this all going to head? Are we going to wind up with super babies and eugenics? And, to me, I dont find that an interesting objection. Its too soon for that objection, Caplan said. Clearly, if were going to let this research proceed, its going to be to treat diseases and prevent diseases.

The enthusiasm surrounding gene-editing in human embryos partly stems from the promise CRISPR has shown in editing away and treating devastating intractable diseases. Earlier this year, the National Academies of Sciences, Engineering and Medicine published a report on human genome editing, addressing potential applications of gene editing, including the possible prevention or treatment of disease.

I hope the applications will be for the treatment of serious diseases and in cases where a sensible alternative is not available, as the National Academies report proposes, Carroll said.

Greely said: The National Academy of Sciences came out with a big report on Valentines Day this year about genome editing in humans, and I thought they very usefully divided it into three categories: basic research, treating living people and making changes that will pass down from generation to generation.

As for the reported new research, this is category one. This is basic research, he said. Category three is the ethically crucial one; this isnt that. Were still a long way from that.

Whats next

Other strides have been made recently in CRISPR research. Scientists at the Memorial Sloan Kettering Cancer Center in New York used the technology to genetically engineer immune cells to target and kill tumor cells in mice.

The mouse study was published in the journal Nature in February. More research is needed to determine whether similar results would appear in humans.

Last year, scientists in the Netherlands published a study in the journal PLOS Pathogens demonstrating CRISPR could be used to edit the DNA of three types of herpes viruses in a petri dish. More research is needed to see whether this tool could be used to fight herpes in actual humans.

Other examples of diseases where CRISPR could show promise as a treatment or preventive approach in the future include cystic fibrosis, sickle cell, hemophilia and mitochondrial diseases, such as the rare degenerative condition that the terminally-ill British infant Charlie Gard has, Caplan said.

There are what are called point mutations where you can go in and fix one genetic error. The simpler the genetic error, the easier it might be to try to repair it using a CRISPR gene-insertion technique, Caplan said about genetic diseases. I think rather than trying to treat cystic fibrosis, or treat sickle cell, or treat hemophilia, it does make ethical sense to figure out ways to prevent it. Now, obviously if its too risky we wont do it. If its too dangerous or maybe it wont work, we still dont know. Were in the early, early days (of research), but I dont think its fear of eugenics that should stop us.

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Report: Scientists edit human embryos for first time in US - kfor.com