articleNature MedicineJan 21, 2019Closed access

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

MLMorgan L. MaederMSMichael StefanidakisCJChristopher J. WilsonRBReshica BaralLBLuis Barrera

Editas Medicine (United States) · Fulcrum Therapeutics (United States)

PubMed
Indexed incrossrefpubmed

Abstract

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698
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34.69
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100%
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33
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Authors

37

Topics & keywords

Topics
Keywords
  • Genome editing
  • CRISPR
  • Gene
  • Biology
  • Cas9
  • Exon
  • Genetics
  • Retinal degeneration
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