Nature Communications (Nov 2021)

Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

  • Raed Ibraheim,
  • Phillip W. L. Tai,
  • Aamir Mir,
  • Nida Javeed,
  • Jiaming Wang,
  • Tomás C. Rodríguez,
  • Suk Namkung,
  • Samantha Nelson,
  • Eraj Shafiq Khokhar,
  • Esther Mintzer,
  • Stacy Maitland,
  • Zexiang Chen,
  • Yueying Cao,
  • Emmanouela Tsagkaraki,
  • Scot A. Wolfe,
  • Dan Wang,
  • Athma A. Pai,
  • Wen Xue,
  • Guangping Gao,
  • Erik J. Sontheimer

DOI
https://doi.org/10.1038/s41467-021-26518-y
Journal volume & issue
Vol. 12, no. 1
pp. 1 – 17

Abstract

Read online

Long-term expression of Cas9 following precision genome editing in vivo may lead to undesirable consequences. Here we show that a single-vector, self-inactivating AAV system containing Cas9 nuclease, guide, and DNA donor can use homology-directed repair to correct disease mutations in vivo.