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Translational Neuroscience: Fundamental Approaches for Neurological Disorders

Gene therapy of CNS disorders using recombinant AAV vectors

Publication ,  Chapter
Murlidharan, G; Samulski, RJ; Asokan, A
January 1, 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

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ISBN

9781489976529

Publication Date

January 1, 2016

Start / End Page

9 / 24
 

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Murlidharan, G., Samulski, R. J., & Asokan, A. (2016). Gene therapy of CNS disorders using recombinant AAV vectors. In Translational Neuroscience: Fundamental Approaches for Neurological Disorders (pp. 9–24). https://doi.org/10.1007/978-1-4899-7654-3_2
Murlidharan, G., R. J. Samulski, and A. Asokan. “Gene therapy of CNS disorders using recombinant AAV vectors.” In Translational Neuroscience: Fundamental Approaches for Neurological Disorders, 9–24, 2016. https://doi.org/10.1007/978-1-4899-7654-3_2.
Murlidharan G, Samulski RJ, Asokan A. Gene therapy of CNS disorders using recombinant AAV vectors. In: Translational Neuroscience: Fundamental Approaches for Neurological Disorders. 2016. p. 9–24.
Murlidharan, G., et al. “Gene therapy of CNS disorders using recombinant AAV vectors.” Translational Neuroscience: Fundamental Approaches for Neurological Disorders, 2016, pp. 9–24. Scopus, doi:10.1007/978-1-4899-7654-3_2.
Murlidharan G, Samulski RJ, Asokan A. Gene therapy of CNS disorders using recombinant AAV vectors. Translational Neuroscience: Fundamental Approaches for Neurological Disorders. 2016. p. 9–24.
Journal cover image

DOI

ISBN

9781489976529

Publication Date

January 1, 2016

Start / End Page

9 / 24