Nature Communications (Mar 2017)

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

  • Wenhan Yu,
  • Suddhasil Mookherjee,
  • Vijender Chaitankar,
  • Suja Hiriyanna,
  • Jung-Woong Kim,
  • Matthew Brooks,
  • Yasaman Ataeijannati,
  • Xun Sun,
  • Lijin Dong,
  • Tiansen Li,
  • Anand Swaroop,
  • Zhijian Wu

DOI
https://doi.org/10.1038/ncomms14716
Journal volume & issue
Vol. 8, no. 1
pp. 1 – 15

Abstract

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Retinitis pigmentosa is mainly caused by mutations that initially affect survival of rod photoreceptors, leading to secondary loss of cones. Here the authors use gene editing to prevent rod degeneration, leading to survival of cones and improved vision in mice.