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

Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of multiple vectors can limit dose and efficacy and increase safety risks. Here, we describe single-vector, ~4.8-kb AAV platforms t...

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Detalles Bibliográficos
Autores principales: Ibraheim, Raed, Tai, Phillip W. L., Mir, Aamir, Javeed, Nida, Wang, Jiaming, Rodríguez, Tomás C., Namkung, Suk, Nelson, Samantha, Khokhar, Eraj Shafiq, Mintzer, Esther, Maitland, Stacy, Chen, Zexiang, Cao, Yueying, Tsagkaraki, Emmanouela, Wolfe, Scot A., Wang, Dan, Pai, Athma A., Xue, Wen, Gao, Guangping, Sontheimer, Erik J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8560862/
https://www.ncbi.nlm.nih.gov/pubmed/34725353
http://dx.doi.org/10.1038/s41467-021-26518-y

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