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Human gene editing therapies approved for treating diseases

By Chukwuma Muanya, Assistant Editor   |   22 February 2017   |   4:52 am

Stanford scientists say they can “trim” unwanted parts of the human genome, paving the way to a cure for the inherited blood disorder which causes anaemia, organ damage, episodic pain and hypertension in millions globally. PHOTO CREDIT: TRT World9

Human gene editing to prevent genetic diseases from being passed to future generations may be permissible under certain conditions, a panel of experts says.

A report issued February 14 by the National Academies of Sciences and Medicine recommends Altering Deoxy ribonucleic Acid (DNA)/genetic material in germline cells — embryos, eggs, and sperm, or cells that give rise to them — may be used to cure genetic diseases for future generations, provided it is done only to correct disease or disability, not to enhance people’s health or abilities. The decision first reported in ScienceNews contradicts earlier recommendations by organizers of a global summit on human gene editing, who concluded that gene editing with molecular scissors such as CRISPR/Cas9 should not be used to produce babies.

Heritable gene editing is not yet ready to be done in people, says Alta Charo, a bioethicist at the University of Wisconsin‒Madison Law School, United States (U.S.), who co-chaired the panel. “We are not trying to greenlight heritable germline editing. We’re trying to find that limited set of circumstances where its use is justified by a compelling need and its application is limited to that compelling need,” says Charo. “We’re giving it a yellow light.”

The National Academies reports carry no legislative weight, but do often influence policy decisions in the United States and abroad. It will be up to Congress, regulatory agencies such as the U.S. Food and Drug Administration, and state and local governments to implement the recommendations. Supporters of new genetic engineering technologies hailed the decision.

“It looks like the possibility of eliminating some genetic diseases is now more than a theoretical option,” says Sean Tipton, a spokesman for the American Society for Reproductive Medicine in Washington, D.C. “That’s what this sets up.” Diseases such as cystic fibrosis and Huntington’s, which are caused by mutations in single genes, could someday be corrected by gene editing. More complex diseases or disorders caused by changes in multiple genes, such as autism or schizophrenia, probably would not be the focus of genome editing.

Others worry that allowing any tinkering with the germline will inevitably lead to “designer babies” and other social ills. It raises fears of stigmatization of people with disabilities, exacerbation of inequalities between people who can afford such therapies and those who can’t, and even a new kind of eugenics, critics say.

“Once you approve any form of human germline modification you really open the door to all forms,” says Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley, Calif.

Panelist Jeffrey Kahn, a bioethicist at Johns Hopkins University, says the door to heritable gene therapy remains closed until stringent requirements can be met. “It’s frankly more of a knock on the door,” he said at the public presentation of the report. The report also changes the debate from whether to allow germline editing to instead focus on the line between therapy and enhancement, Darnovsky says. “I’m feeling very unsettled and disappointed by what they are recommending.” Several clinical trials in the United States, China and other countries are already under way to do gene editing in people who have cancer or other diseases. But those therapies do not involve altering germline cells; instead they fix defects or make alterations to DNA in other body, or “somatic,” cells. The panel recommended that such somatic cell therapies should also be restricted to treating diseases, not allowing enhancements.

Researchers in the United Kingdom, Sweden and China have already done gene editing on early human embryos in the lab. Recent clinical trials in Mexico and Ukraine to produce “three-parent babies” are also seen as altering the germline because such children carry a small amount of DNA from an egg donor. But those children don’t have modifications of their nuclear DNA, where the genetic instructions that determine traits are stored.

Currently, researchers in the United States are effectively banned from conducting clinical trials that would produce heritable changes in the human genome, either by gene editing or making three-parent babies. The new recommendations could pave the way to allow such experiments.

But the panel lays out a number of hurdles that must be cleared before germline editing could move forward, ones that may be impossible to overcome, says Nita Farahany, a bioethicist at Duke Law School in Durham, N.C. “Some people could read into the stringency of the requirements to think that the benefits could never outweigh the risks,” she says.

One hurdle is a requirement to follow multiple generations of children who have gotten gene editing to determine whether the therapy has consequences for future generations. Researchers would never be able to guarantee that they could conduct such long-term studies, Farahany says. “You can’t bind your children and grandchildren to agree to be tracked by such studies.”

Distinctions between therapies and enhancements are also vague. Researchers may not be able to convincingly draw lines between them, says George Church, a Harvard University geneticist who has developed CRISPR/Cas9 for a variety of purposes. Virtually everything medicine has accomplished could be considered as enhancing human life, he says. “Vaccines are advancements over our ancestors. If you could tell our ancestors they could walk into a smallpox ward and not even worry about it, that would be a superpower.”

But the new technology may make it harder to enhance humans than drugs do, says Charo. Gene-editing technologies are so precise and specific that someone who does not carry a disease-causing mutation would probably not benefit from the technology, she says.

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