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Article

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

27 Apr 2016

Abstract excerpt

Adeno-associated viral (AAV) vectors have shown promise as a platform for gene therapy of neurological disorders. Achieving global gene delivery to the central nervous system (CNS) is key for development of effective therapies for many of these diseases. Here we report the isolation of a novel CNS tropic AAV capsid, AAV-B1, after a single round of in vivo selection from an AAV capsid library. Systemic injection...

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PMID: 27117222
DOI: 10.1038/mt.2016.84

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