A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR) can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy because of their ability to achieve sustained expres...

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Bibliographic Details
Main Authors: Ang Li, Ciaran M. Lee, Ayrea E. Hurley, Kelsey E. Jarrett, Marco De Giorgi, Weiqi Lu, Karol S. Balderrama, Alexandria M. Doerfler, Harshavardhan Deshmukh, Anirban Ray, Gang Bao, William R. Lagor
Format: Article
Language:English
Published: Elsevier 2019-03-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118301219

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http://www.sciencedirect.com/science/article/pii/S2329050118301219

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