Gene Editing for CEP290 -Associated Retinal Degeneration

  • Pierce E
  • Aleman T
  • Jayasundera K
  • et al.
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Abstract

CEP290-associated inherited retinal degeneration causes severe early-onset vision loss due to pathogenic variants in CEP290. EDIT-101 is a clustered regularly interspaced short palindromic repeats ...

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Pierce, E. A., Aleman, T. S., Jayasundera, K. T., Ashimatey, B. S., Kim, K., Rashid, A., … Pennesi, M. E. (2024). Gene Editing for CEP290 -Associated Retinal Degeneration. New England Journal of Medicine, 390(21), 1972–1984. https://doi.org/10.1056/nejmoa2309915

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