September 27, 2020

The Niche

Knoepfler lab stem cell blog

Just freakin do it: patients voice impatience on CRISPR for genetic diseases

IMG_4221Both before and particularly now after the big human gene editing summit in Washington, D.C. at the National Academy of Sciences, I’ve talked with patients about their views on this new technology including at last week’s World Stem Cell Summit.

One of the most striking moments of the DC summit was when the mother of a pediatric patient made the comment after a talk about human gene editing that if it could be helpful with genetic diseases, “just freakin do it!” A lot of pain was evident in her voice and tears.

Some patients at the World Stem Cell Summit indicated very much the same perspectives. I’ve heard from patients on this via email over the past year as well.

There is a need for rapid, new approaches to genetic diseases.

Patients and their loved ones often have a higher degree of risk tolerance than scientists. I have seen this in the stem cell field too, where many patients ask me about unapproved stem cell treatments for themselves (“I have MS, ALS, etc.”) or their children (“he has autism, etc.”). It’s not that they want to take risks, but understandably they are willing because they are in an urgent, health-related situation in their family.

Antonio Regalado over at MIT Tech Review has a powerful recent piece on a case of Huntington’s Disease patients including Jeff Carroll who are in favor of human embryo editing:

“I have no compunctions about it,” says Carroll, who is a neuroscientist at Western Washington University, in Bellingham. “I am saying, please, please do mess with our DNA.”

What risks are acceptable when we are talking about making genetically modified human beings?

There isn’t going to be one “right” answer to such questions, but it is clear that patient voices need to be part of the discussion. At future meetings and discussions on germline modification more patient voice need to be heard.

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