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Gene Therapy of CNS Disorders Using Recombinant AAV Vectors

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Translational Neuroscience

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.

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Authors and Affiliations

  1. Curriculum in Genetics and Molecular Biology, Gene Therapy Center, University of North Carolina at Chapel Hill, CB7352, Thurston Building, Rm 5125, Chapel Hill, NC, 27599, USA

    Giridhar Murlidharan BTech, MS

  2. Department of Pharmacology, School of Medicine, Gene Therapy Center, University of North Carolina at Chapel Hill, 104 Manning Drive, Chapel Hill, NC, 27599, USA

    R. Jude Samulski PhD

  3. Departments of Genetics, Biochemistry and Biophysics, Gene Therapy Center, University of North Carolina at Chapel Hill, CB7352, Thurston Building, Rm 5123, Chapel Hill, NC, 27599, USA

    Aravind Asokan PhD

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  1. Giridhar Murlidharan BTech, MS
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  2. R. Jude Samulski PhD
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  3. Aravind Asokan PhD
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Correspondence to Aravind Asokan PhD .

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  1. UnivofCA-SanDiego,VeteransAdmintnMedCntr, Translational Neuroscience Institute, La Jolla, California, USA

    Mark H. Tuszynski

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Murlidharan, G., Samulski, R.J., Asokan, A. (2016). Gene Therapy of CNS Disorders Using Recombinant AAV Vectors. In: Tuszynski, M. (eds) Translational Neuroscience. Springer, Boston, MA. https://doi.org/10.1007/978-1-4899-7654-3_2

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