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Published 14 days ago

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

  • A clinical trial of CRISPR gene editing in 14 individuals with inherited blindness proved safe and yielded measurable improvements in 11 participants.
  • All 14 participants, aged 10 to 63, had Leber Congenital Amaurosis due to mutations in the CEP290 gene.
  • Eleven participants saw improvements in various outcomes, with four having improved visual acuity and six showing enhanced cone-mediated vision.
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