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Evalytics 13 May at 09.05 PM

Participants of pioneering CRISPR gene editing trial see vision improve


  • EDIT-101, a CRISPR-based treatment, demonstrated improvement in 79% of participants with inherited retinal degeneration.
  • The treatment targeted a mutation in the CEP290 gene associated with Leber Congenital Amaurosis Type 10.
  • Safety was confirmed, with no serious adverse events reported among the 14 participants.
  • Visual acuity and quality of life showed clinically meaningful improvement.
  • Further research is needed to optimize dosing and explore treatment effects across different age groups.

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