CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

Gene therapy using recombinant adeno-associated viral (AAV) vectors is emerging as a promising approach to treat central nervous system disorders such as Spinal muscular atrophy, Batten, Parkinson and Alzheimer disease amongst others. A critical remaining challenge for central nervous system-targete...

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Bibliographic Details
Main Authors: Giridhar Murlidharan, Kensuke Sakamoto, Lavanya Rao, Travis Corriher, Dan Wang, Guangping Gao, Patrick Sullivan, Aravind Asokan
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117300628

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http://www.sciencedirect.com/science/article/pii/S2162253117300628

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