Eye on genome editing

J Exp Med. 2023 May 1;220(5):e20230146. doi: 10.1084/jem.20230146. Epub 2023 Mar 17.

Abstract

CRISPR/Cas9 genome editing techniques have the potential to treat previously untreatable inherited genetic disorders of vision by correcting mutations that cause these afflictions. Using a prime editor, Qin et al. (2023. J. Exp. Med.https://doi.org/10.1084/jem.20220776) restored visual functions in a mouse model (rd10) of retinitis pigmentosa.

Publication types

  • Comment

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics
  • Disease Models, Animal
  • Gene Editing* / methods
  • Mice
  • Mutation / genetics
  • Retinitis Pigmentosa* / genetics
  • Retinitis Pigmentosa* / therapy