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Ethical challenges for a new generation of early-phase pediatric gene therapy trials.

Alexander A IyerDimah SaadeDiana Bharucha-GoebelA Reghan FoleyGilberto 'Mike' AverionEduardo ParedesSteven GrayCarsten G BönnemannChristine GradySaskia HendriksPatrick Ober
Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2021)
After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.
Keyphrases
  • gene therapy
  • clinical trial
  • endothelial cells
  • decision making
  • human health
  • phase ii
  • transcription factor
  • study protocol
  • induced pluripotent stem cells
  • climate change