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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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Detalles Bibliográficos
Autores principales:	Murlidharan, Giridhar, Sakamoto, Kensuke, Rao, Lavanya, Corriher, Travis, Wang, Dan, Gao, Guangping, Sullivan, Patrick, Asokan, Aravind
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5330941/ https://www.ncbi.nlm.nih.gov/pubmed/27434683 http://dx.doi.org/10.1038/mtna.2016.49

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