Category Archives: Transhuman News

Scientists should be allowed to altera persons DNA in ways that will be passed on to future generations, but only to prevent serious and strongly heritable diseases, according to a new report from the National Academy of Sciences and the National Academy of Medicine.

However, tinkering with these genes in orderto enhance or alter traits such as strength, intelligence or beauty should remain off-limits, the report authors concluded.

Changing theso-called germline effectively, editing humanitys future by altering genes in human reproductive cells is illegal in the United States. It has largely been considered ethically off-limits here as well, at least whilebioethicists and scientists pondered the unforeseen effects and unexamined moral dilemmas of using new gene-editing technologies.

However, scientists have moved forward aggressively to explore the feasibility of altering disease genes in other adult human cells with a revolutionarytechnique known asCRISPR-Cas9. It is widely believed that gene editing of this sort could treat patients with metabolic disorders, certain cancers, anda range of otherdiseases that arise from genetic mutationswithout altering the germline.

Last year, Chinese scientists launched a trial that uses CRISPR-Cas9 in a treatment for lung cancer. While the trials outcome is awaited with high anticipation, scientists outside of China have expressed concern that ethical reservations in the United States and Europe will put themat a disadvantage.

CRISPR-Cas9 makesgene editing more straightforward, more precise and far more widespread. As such, the National Academies report acknowledgesthat changingheritable DNA ineggs, sperm and early embryosis fast becoming a realistic possibility that deserves serious consideration.

The 22-memberpanel of scientists and bioethicists who produced the report completed a comprehensive review of the issues raised by that prospect.

Clinical trials involving germline editing should onlybe pursuedto treat diseases that cannot be improvedwith reasonable alternatives, the committee said. In addition, they added,scientists should convincingly demonstratethey are targeting a gene that eithercauses or strongly predisposes a carrier to a serious disease or condition, and that they have weighed the likely risks and benefits of altering that gene.

These clinical trials should be conducted under public scrutiny that takes into account issues ofsocietal fairness, personal dignityand scientific integrity, the panel said.

Finally, scientists should conduct long-term follow-up studies to discern how gene editing affects subsequent generations.Public debate and discussion about the technologyshould continue, the panel added.

Genome editing research is very much an international endeavor, and all nations should ensure that any potential clinical applications reflect societal values and be subject to appropriate oversight and regulation, saidMIT cancer researcher Richard O.Hynes, who co-chaired the panel with University of Wisconsin-Madison bioethicist R. Alta Charo. These overarching principles and the responsibilities that flow from them should be reflected in each nations scientific community and regulatory processes.

Dr. J. Patrick Whelan, an immunologist and bioethicist who was not on the panel, said the grouphas asked the compelling questions, sparkinga conversation that must keep up with a rapid pace of scientific discovery in this field. He called the reportsrelease a fantastic development.

What theyre saying is, lets start the conversation, maintain ethical structures along the way, and hopefully do this the right way, said Whelan, who serves on the advisory board of USCs Institute for Advanced Catholic Studies.

The international panel included members from the U.S., China, France, Israel andItaly.Their report was underwritten in part by the Department of Defenses Advanced Research Projects Agency and the U.S. Food and Drug Administration.

melissa.healy@latimes.com

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To prevent serious medical conditions, scientists should be able to edit people's DNA, panel says - Los Angeles Times

A study from Massachusetts General Hospital (MGH) researchers has found that a pattern of gene variants associated with an "apple-shaped" body type, in which weight is deposited around the abdomen, rather than in the hips and thighs, increases the risk for type 2 diabetes and coronary heart disease, as well as the incidence of several cardiovascular risk factors. The report appears in the February 14 issue of JAMA.

"People vary in their distribution of body fat -- some put fat in their belly, which we call abdominal adiposity, and some in their hips and thighs," says Sekar Kathiresan, MD, director of the MGH Center for Genomic Medicine, associate professor of Medicine at Harvard Medical School, and senior author of the JAMA report. "Abdominal adiposity has been correlated with cardiometabolic disease, but whether it actually has a role in causing those conditions was unknown. We tested whether genetic predisposition to abdominal adiposity was associated with the risk for type 2 diabetes and coronary heart disease and found that the answer was a firm 'yes'."

While several observational studies have reported greater incidence of type 2 diabetes and heart disease among individuals with abdominal adiposity, they could not rule out the possibility that lifestyle factors -- such as diet, smoking and a lack of exercise -- were the actual causes of increased disease risk. It also could have been possible that individuals in the early stages of heart disease might develop abdominal adiposity because of a limited ability to exercise. The current study was designed to determine whether body type really could increase cardiometabolic risk.

To answer that question, the research team applied a genetic approach called mendelian randomization, which measures whether inherited gene variants actually cause outcomes such as the development of a disease. Using data from a previous study that identified 48 gene variants associated with waist-to-hip ratio adjusted for body mass index -- an established measure for abdominal adiposity -- they developed a genetic risk score. They then applied that score to data from six major genome-wide association studies and to individual data from the U.K. Biobank -- a total research group of more than 400,000 individuals -- to determine any association between a genetic predisposition to abdominal adiposity and cardiometabolic disease and its risk factors.

The results clearly indicated that genetic predisposition to abdominal adiposity is associated with significant increases in the incidence of type 2 diabetes and coronary heart disease, along with increases in blood lipids, blood glucose and systolic blood pressure. No association was found between the genetic risk score and lifestyle factors, and testing confirmed that only the abdominal adiposity effects of the identified gene variants were associated with cardiometabolic risk.

"These results illustrate the power of using genetics as a method of determining the effects of a characteristic like abdominal adiposity on cardiometabolic outcomes," says lead author Connor Emdin, DPhil, of the MGH Center for Genomic Medicine and the Cardiology Division. "The lack of association between the body type genetic risk score and confounding factors such as diet and smoking provides strong evidence that abdominal adiposity itself contributes to causing type 2 diabetes and heart disease."

Emdin continues, "Not only do these results allow us to use body shape as a marker for increased cardiometabolic risk, they also suggest that developing drugs that modify fat distribution may help prevent these diseases. Future research also could identify individual genes that could be targeted to improve body fat distribution to reduce these risks."

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Gene variants associated with body shape increase risk of heart disease, type 2 diabetes - Science Daily

Editing of human cells to alter traits handed down to future generations may one day be ethically permissible, said a committee co-chaired by bioethicist Alta Charo, a professor at the University of Wisconsin School of Law.

"It is not ready now, but it might be safe enough to try in the future," Charo told National Public Radio. "And if certain conditions are met, it might be permissible to try it."

Charo, along with UW-Madison science communication professor Dietram Scheufele, was part of a committee appointed by the National Academies of Sciences and Medicine that on Tuesday released a groundbreaking report on the ethics of human gene editing.

Dietram Scheufele

Despite the consensus at a 2015 international summit in Washington, D.C., where scientists concluded they should not perform gene editing on reproductive cells intended for establishing a pregnancy, the new report advises that such research might be permitted after further work to develop appropriate risk/benefit standards.

The panel recommended alteration of genes in human reproductive cells, sometimes called germ lines, be allowed only to prevent serious disease or disability for which there is no treatment. On the other hand, the potential benefits of human germ line editing to enhance desired inherited traits like beauty, intelligence or strength are at this time unlikely to outweigh risks like the inheritance of undesired mutations, panel members found.

Up until now, weve been talking only hypothetically and most people assumed we simply wouldnt ever do this, Charo told Nature. We are not saying that you have to or you should, but we are saying that if you can meet these criteria it is permissible.

In the United States, the use of federal funds for research involving the creation of a human embryo to include an inheritable modification is prohibited. But the committee defined a set of criteria for use in other countries, or in the U.S. if restrictions were allowed to expire. The criteria include: absence of reasonable alternatives, data on the likely risks and potential health benefits; rigorous oversight; and comprehensive plans for long-term, multi-generational follow-up.

The committee also called for broad public education and engagement in applying societal values to the risks and benefits of gene editing technologies.

In the months since the Washington summit, the development of a gene-editing tool called Crispr-Cas9 has made addressing the ethical issues surrounding human gene editing urgent.

As human genome editing becomes more effective, clinicians working in countries with few regulations will likely begin modifying embryos and implanting them in patients, raising the potential for inheritance of unintended mutations along with targeted changes, Charo said.

We are very much aware that medical tourism is a fact of global life now, she said. We certainly don't want to see the same thing, and a prohibition might exacerbate the problem.

Scheufele, a social scientist who studies communication and public understanding of science, spoke to the importance of public debate on the ethical issues involved.

We want to bring in people even if they know little about the topic and get them to the point where they can engage in meaningful debate, Scheufele said. We want to move forward in a responsible fashion with the best available science and a meaningful understanding of the risks and benefits.

The report also established a set of broad governance principles with respect to human genome editing in the U.S. and elsewhere: promote well-being, transparency, due care, responsible science, respect for persons, fairness and transnational cooperation.

Every country struggles with the same set of complexities but has a different regulatory system. So, we established larger principles that are applicable across different political or cultural contexts, Scheufele said.

While the National Academies panel drew a bright line between gene modification for treatment of disease and gene modification for enhancement, the boundary often is not clear, say opponents of human germ line editing.

This opens the door to advertisements from fertility clinics of giving your child the best start in life with a gene-editing packet, Marcy Darnovsky of the Center for Genetics and Society, a public interest group in Berkeley, Calif., told the New York Times. And whether these are real advantages or perceived advantages, they would accrue disproportionately to people who are already advantaged.

The new guidelines, Darnovsky noted, also set the United States apart from many European countries that have signed a treaty to refrain from human germ line editing.

George Church, co-founder of the gene-editing company Editas Medicine, told Wired that the line between treatment and enhancement will be hard to hit.

In the process of fixing something that is broken, you can move it to the middle of the bell curve or beyond, said Church, a geneticist at Harvard and MIT. If you are aiming at the middle, you will fall on the low or the high side.

Church isnt worried about athletes using Crispr-based therapies, he said the big market is aging adults who want to put off the effects of old age. If you want to be useful longer or do age reversal, that could be preventive medicine, he said. But if a therapy was sufficiently good, it would be enhancement.

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UW's Alta Charo: Gene editing for inherited human traits 'not ready now, might be in future' - The Courier Life News

The Atlantic

Harvard and MIT Scientists Win Gene-Editing Patent Fight New York Times The Broad Institute in Cambridge, Mass., will retain potentially lucrative rights to a powerful gene-editing technique that could lead to major advances in medicine and agriculture, the federal Patent and Trademark Office ruled on Wednesday. The ... What the CRISPR Patent Decision Means for Gene EditingThe Atlantic Broad Institute wins case over gene-editing patents, boosting Editas sharesBoston Business Journal Broad Institute Wins Big Battle Over CRISPR Gene-Editing PatentNPR 24/7 Wall St. -Motley Fool -Lexington Herald Leader all 52 news articles »

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Harvard and MIT Scientists Win Gene-Editing Patent Fight - New York Times

February 15, 2017 Dr. Madhavan Nair oversees work in his lab at Herbert Wertheim College of Medicine. Credit: Florida International University

Opiate abuse is a significant risk factor for HIV infection, and in combination they can have a devastating effect on the brain. Scientists at FIU Herbert Wertheim College of Medicine (HWCOM) are studying new therapies that can short-circuit HIV infection and mitigate the damaging effects that opiate addiction has on the central nervous system.

The ambitious $3.5 million five-year study, funded by the National Institutes for Health is now underway and will be completed b7 2021. Researchers hope the work will lead to lead to a multi-purpose platform for drugs targeting a variety of other difficult to treat diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer's, Parkinson's and Huntington's diseases.

The Institute of Neuro-Immune Pharmacology at HWCOM, led by Chair and Associate Dean of the Department of Immunology Madhavan Nair, is teaming up with Kamel Khalili, chair of the Department of Neuroscience at Temple University, and the Comprehensive NeuroAIDS Center at Temple University's Lewis Katz School on a new study that will combine Khalil's gene editing strategy using nanotechnology with Nair's work to help opiate users with HIV.

Despite significant advances in anti-retroviral therapy (ART), which is used to treat HIV patients, ART is unable to penetrate the blood brain barrier (BBB) after systemic administration. In addition, the elimination of HIV from the central nervous system and peripheral reservoirs remains challenging due to the HIV genome's ability to integrate itself into the host genome.

But advances in nanotechnology have expanded the possibilities for novel drug delivery systems that can cross the BBB to recognize and eradicate HIV in the brain. Nair and other scientists from the Institute of Neuro-Immune Pharmacology at HWCOM have combined nanotechnology with magneto electro nanoparticles (MENPs) as externally field triggered/controlled drug carriers that offer the unique capability of low energy and dissipation free on-demand drug release across the BBB.

Nair's MENP drug-based delivery system is now the basis for the partnership with Khalili, who developed the Cas9/gRNA system; a genetic engineering tool that has shown great promise in finding and destroying copies of HIV that have burrowed into the host's genome.

The partnership will use Cas9/gRNA to eliminate entire integrated copies of the HIV genome from the host chromosome with the MENP drug-based delivery system.

"This is the first time that we are sending medicine to the brain that will eliminate latent HIV as well as deliver a morphine antagonist (methylnaltrexone) across the BBB in a non-invasive manner to protect neurons from morphine induced neurodegenerative effects," Nair says. MENP is non-invasive and fast-acting, and this newly created multi-disciplinary approach will also introduce unprecedented 3-D diagnostic views and allow clearance of the nanoparticles from the brain to the periphery by reverse external magnetic force once the cargo has been delivered.

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A breakthrough by CSIRO-led scientists has made the world's strongest material more commercially viable, thanks to the humble soybean.

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Nanotechnology based gene editing to eradicate HIV brain reservoir in drug abusers - Phys.Org