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Article

AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice

1 Jan 2016

Abstract excerpt

Current barriers to the use of adeno-associated virus serotype 9 (AAV9) in clinical trials for treating neurological disorders are its high expression in many off-target tissues such as liver and heart, and lack of cell specificity within the central nervous system (CNS) when using ubiquitous promoters such as human cytomegalovirus (CMV) or chicken-β-actin hybrid (CAG). To enhance targeting the transgene...

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PMID: 26942208
DOI: 10.1038/mtm.2015.55

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