Viral vectors and delivery strategies for CNS gene therapy

Steven J. Gray, Kenton T. Woodard, R. Jude Samulski

Research output: Contribution to journalReview articlepeer-review

93 Scopus citations


This review aims to provide a broad overview of the targets, challenges and potential for gene therapy in the CNS, citing specific examples. There are a broad range of therapeutic targets, with very different requirements for a suitable viral vector. By utilizing different vector tropisms, novel routes of administration and engineered promoter control, transgenes can be targeted to specific therapeutic applications. Viral vectors have proven efficacious in preclinical models for several disease applications, spurring several clinical trials. While the field has pushed the limits of existing adeno-associated virus-based vectors, a next generation of vectors based on rational engineering of viral capsids should expand the application of gene therapy to be more effective in specific therapeutic applications.

Original languageEnglish (US)
Pages (from-to)517-534
Number of pages18
JournalTherapeutic Delivery
Issue number4
StatePublished - Oct 2010

ASJC Scopus subject areas

  • Pharmaceutical Science


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