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Development and Optimization of Adeno-Associated Virus Vector Transfer into the Central Nervous System

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Viral Vectors for Gene Therapy

Part of the book series: Methods in Molecular Medicine™ ((MIMM,volume 76))

Abstract

The postgenomic era has been ushered in making promises for cures and functional analysis of previously unknown or uncharacterized genes. However, realization of the potential of gene therapy for neurological disorders and functional genomic analysis in the brain remains a significant challenge and is largely limited by current technology. Efficient gene transfer to neurons remains a bottleneck for widespread molecular genetic studies of the nervous system, and has also limited the effective translation of gene therapy for various human neurological disorders, which currently have inadequate or no treatment [reviewed in refs. 1 and 2].

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Author information

Authors and Affiliations

  1. CNS Gene Therapy Center, Department of Neurosurgery, Jefferson Medical College, Philadelphia, PA

    Matthew J. During

  2. Department of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland, Auckland, New Zealand

    Matthew J. During

  3. Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania and Department of Neurology and Neuroscience Research, Children’s Hospital of Philadelphia, Philadelphia, PA

    Deborah Young

  4. Department of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland, Auckland, New Zealand

    Kristin Baer, Patricia Lawlor & Matthias Klugmann

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  1. Matthew J. During
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  2. Deborah Young
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  3. Kristin Baer
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Editor information

Editors and Affiliations

  1. Department of Oral Molecular Biology, School of Dentistry Oregon Health & Science University, Portland, OR

    Curtis A. Machida

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During, M.J., Young, D., Baer, K., Lawlor, P., Klugmann, M. (2003). Development and Optimization of Adeno-Associated Virus Vector Transfer into the Central Nervous System. In: Machida, C.A. (eds) Viral Vectors for Gene Therapy. Methods in Molecular Medicine™, vol 76. Humana Press. https://doi.org/10.1385/1-59259-304-6:221

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  • DOI: https://doi.org/10.1385/1-59259-304-6:221

  • Publisher Name: Humana Press

  • Print ISBN: 978-1-58829-019-9

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