Gene therapy of CNS disorders using recombinant AAV vectors


Book Section

© Springer Science+Business Media New York 2016. Corrective intervention for CNS disorders typically requires replenishment of depleted biomolecules (e.g., catabolic enzymes), protection of neurons and glia from premature death, or utilization of CNS cells as bio-factories for production of neurotransmitters or their biological precursors/cofactors. Gene therapy offers the ability to treat disorders in various organs by delivering therapeutic transgenes for regaining lost functionality. Adeno-associated viruses (AAV) have emerged as the vector of choice for CNS gene therapy. This chapter summarizes key observations made during preclinical and clinical evaluations of AAV vectors toward gene therapy of two broad categories of CNS disorders, namely metabolic storage disorders and movement disorders

Full Text

Duke Authors

Cited Authors

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

Published Date

  • January 1, 2016

Book Title

  • Translational Neuroscience: Fundamental Approaches for Neurological Disorders

Start / End Page

  • 9 - 24

International Standard Book Number 13 (ISBN-13)

  • 9781489976529

Digital Object Identifier (DOI)

  • 10.1007/978-1-4899-7654-3_2

Citation Source

  • Scopus