Biology of adeno-associated viral vectors in the central nervous system.

Published online

Journal Article (Review)

Gene therapy is a promising approach for treating a spectrum of neurological and neurodegenerative disorders by delivering corrective genes to the central nervous system (CNS). In particular, adeno-associated viruses (AAVs) have emerged as promising tools for clinical gene transfer in a broad range of genetic disorders with neurological manifestations. In the current review, we have attempted to bridge our understanding of the biology of different AAV strains with their transduction profiles, cellular tropisms, and transport mechanisms within the CNS. Continued efforts to dissect AAV-host interactions within the brain are likely to aid in the development of improved vectors for CNS-directed gene transfer applications in the clinic.

Full Text

Duke Authors

Cited Authors

  • Murlidharan, G; Samulski, RJ; Asokan, A

Published Date

  • 2014

Published In

Volume / Issue

  • 7 /

Start / End Page

  • 76 -

PubMed ID

  • 25285067

Pubmed Central ID

  • 25285067

International Standard Serial Number (ISSN)

  • 1662-5099

Digital Object Identifier (DOI)

  • 10.3389/fnmol.2014.00076


  • eng

Conference Location

  • Switzerland