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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Bibliographische Detailangaben
Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: Murlidharan, Giridhar, Sakamoto, Kensuke, Rao, Lavanya, Corriher, Travis, Wang, Dan, Gao, Guangping, Sullivan, Patrick, Asokan, Aravind
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2016
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5330941/
https://ncbi.nlm.nih.gov/pubmed/27434683
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.49
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