Sep 26, 2019

Patients issue new pleas for experimental gene editing

Human chromosome pairs

Photo: Sandra Baker/Getty Images

Patients and parents of children with life-threatening diseases may start asking for experimental gene editing as a form of treatment, STAT reports.

The big picture: Bioethicists, genetic engineers and lawmakers are now asking the same ethical questions that led to the 2018 law allowing "the right to try" experimental drugs prior to FDA approval: namely, do patients have a right to try gene editing technology?

What they're saying: "It's so intuitively simple: replace or correct a gene that's not functioning properly. There is so much hype, more and more people will think, I want that," bioethicist Alison Bateman-House of New York University told STAT.

  • In a potentially prescient move, lawmakers in California passed a law banning people from using unproven therapies amid concern that people would start biohacking themselves or using do-it-yourself CRISPR kits.
  • There are only 2 approved gene therapies in the U.S., one for a form of blindness and the other for spinal muscular atrophy.

The bottom line: "Parents are increasingly asking scientists for access to experimental compounds that have never even been tested in animals" to help save their sick children, per STAT, and more such pleas for genetic technologies are likely only a matter of time.

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