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Gene therapies for rare diseases are under threat. Scientists hope to save them

As industry steps aside, scientists seek innovative ways to make sure expensive treatments can reach people who need them.
  1. Heidi Ledford
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It was one of gene therapy’s greatest successes — and greatest disappointments.

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doi: https://doi.org/10.1038/d41586-023-03109-z

Updates & Corrections

  • Correction 06 October 2023: An earlier version of this article misstated P. J. Brooks’s title.

  • Correction 09 October 2023: The original title given for P. J. Brooks was, in fact, correct. The story has been updated accordingly.

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