Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
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.
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Nature Portfolio
2021
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oai:doaj.org-article:81e517a4261d442a884395258d491bfe2021-11-08T11:06:08ZSelf-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo10.1038/s41467-021-26518-y2041-1723https://doaj.org/article/81e517a4261d442a884395258d491bfe2021-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-26518-yhttps://doaj.org/toc/2041-1723Long-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.Raed IbraheimPhillip W. L. TaiAamir MirNida JaveedJiaming WangTomás C. RodríguezSuk NamkungSamantha NelsonEraj Shafiq KhokharEsther MintzerStacy MaitlandZexiang ChenYueying CaoEmmanouela TsagkarakiScot A. WolfeDan WangAthma A. PaiWen XueGuangping GaoErik J. SontheimerNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-17 (2021) |
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DOAJ |
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DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q 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 Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| description |
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. |
| format |
article |
| author |
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 |
| author_facet |
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 |
| author_sort |
Raed Ibraheim |
| title |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| title_short |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| title_full |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| title_fullStr |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| title_full_unstemmed |
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo |
| title_sort |
self-inactivating, all-in-one aav vectors for precision cas9 genome editing via homology-directed repair in vivo |
| publisher |
Nature Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/81e517a4261d442a884395258d491bfe |
| work_keys_str_mv |
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