Gene Therapy of CNS Disorders Using Recombinant AAV Vectors

  • Giridhar Murlidharan
  • R. Jude Samulski
  • Aravind Asokan
Chapter
First Online:

Abstract

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.

Keywords

Adeno-associated virus (AAV) Viral vectors Gene therapy Neurological disorders Neurodegenerative diseases Central nervous system 
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Copyright information

© Springer Science+Business Media New York 2016

Authors and Affiliations

  • Giridhar Murlidharan
    • 1
  • R. Jude Samulski
    • 2
  • Aravind Asokan
    • 3
  1. 1.Curriculum in Genetics and Molecular Biology, Gene Therapy CenterUniversity of North Carolina at Chapel HillChapel HillUSA
  2. 2.Department of Pharmacology, School of Medicine, Gene Therapy CenterUniversity of North Carolina at Chapel HillChapel HillUSA
  3. 3.Departments of Genetics, Biochemistry and Biophysics, Gene Therapy CenterUniversity of North Carolina at Chapel HillChapel HillUSA

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