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
Affiliations
- Raed Ibraheim
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Phillip W. L. Tai
- Horae Gene Therapy Center, University of Massachusetts Medical School
- Aamir Mir
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Nida Javeed
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Jiaming Wang
- Horae Gene Therapy Center, University of Massachusetts Medical School
- Tomás C. Rodríguez
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Suk Namkung
- Horae Gene Therapy Center, University of Massachusetts Medical School
- Samantha Nelson
- Department of Microbiology and Physiological Systems, University of Massachusetts Medical School
- Eraj Shafiq Khokhar
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Esther Mintzer
- Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School
- Stacy Maitland
- Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School
- Zexiang Chen
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Yueying Cao
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Emmanouela Tsagkaraki
- Program in Molecular Medicine, University of Massachusetts Medical School
- Scot A. Wolfe
- Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School
- Dan Wang
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Athma A. Pai
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Wen Xue
- RNA Therapeutics Institute, University of Massachusetts Medical School
- Guangping Gao
- Horae Gene Therapy Center, University of Massachusetts Medical School
- Erik J. Sontheimer
- RNA Therapeutics Institute, University of Massachusetts Medical School
- DOI
- https://doi.org/10.1038/s41467-021-26518-y
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 17
Abstract
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.
