Nature Communications (Jan 2020)

Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice

  • Koji M. Nishiguchi,
  • Kosuke Fujita,
  • Fuyuki Miya,
  • Shota Katayama,
  • Toru Nakazawa

DOI
https://doi.org/10.1038/s41467-019-14181-3
Journal volume & issue
Vol. 11, no. 1
pp. 1 – 9

Abstract

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Replacing mutant genes with wildtype copies using adeno-associated virus (AAV) has been explored for the treatment of inherited retinopathies, but the low cargo limit restricts its use. Here the authors describe a single AAV platform that allows local replacement of a mutated sequence with its wildtype counterpart, based on combined CRISPR-Cas9 and micro-homology-mediated end joining.