A 251-page, long-awaited consensus report from the National Academies of Sciences and Medicine on genome editing was released on February 14, 2017. These two prestigious American science institutions have cautiously endorsed future use of powerful gene editing techniques to modify human embryos.

A concept that was once viewed as ultra-futuristic, seen only in science-fiction movies, may soon become a common medical treatment. The field of genome editing is rapidly advancing, accelerated by new technologies like zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and most recently, and probably most importantly, the CRISPR/Cas9 system. Genome editing is faster, cheaper, easier, and more accurate than ever before.

All these techniques utilize protein recognition of specific DNA sequences to selectively modify sections of DNA, where as little as an adjustment of one nucleotide could prevent fatal diseases from appearing in patients. Cystic fibrosis, Tay-Sachs, and sickle-cell anemia, are all diseases caused by a single incorrect nucleotide, for example.

Scientists at the University of Washington in Seattle have used these techniques to fix faulty genes causing Duchenne muscular dystrophy in mice, with promising results. But what if this muscle-boosting treatment was used on healthy people to give them superhuman strength?

This represents a slippery slope to bioethicists. Fears of a society with genetic haves and have-nots is a real possibility, but so is the ability to eliminate devastating genetic diseases from being passed on to children from their parents. The report itself calls the procedure “highly contentious,” since any changes to the genome will continue to be passed on to future generations without their consent, and “would therefore cross a line many have viewed as ethically inviolable.” Designer babies are a prominent issue; while super-intelligence is unlikely, as it is likely controlled by hundreds to thousands of genes, other cosmetic changes like muscle mass, height, or hair color, are possibilities.

Therefore, the group of scientists, lawyers, clinicians, and ethicists, endorsed only alterations that prevent “serious diseases and disability” for which no other treatment exists, and only if gene editing is strictly regulated and limited. They also call for international cooperation, strict regulatory and oversight framework, public input, and long-term follow-ups for all patients. As Richard Hynes, report chair and cancer researcher at MIT states, “The slope is not very slippery. Friction is introduced by the regulatory system.”

The US Food and Drug Administration is currently prohibited from considering applications for human germline (sex cell) therapy clinical trials, but that restriction is only in place until April this year – this month. Far more work is needed to perfect the process before trials can begin. However, the report states that if the technology is found to be safe and effective in the future, there is no reason that it shouldn’t be considered.


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