Viral Vectors for Gene Therapy pp 221-236

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

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

  • Matthew J. During
  • Deborah Young
  • Kristin Baer
  • Patricia Lawlor
  • Matthias Klugmann

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].

Copyright information

© Humana Press Inc. 2003

Authors and Affiliations

  • Matthew J. During
    • 1
    • 2
  • Deborah Young
    • 3
  • Kristin Baer
    • 4
  • Patricia Lawlor
    • 4
  • Matthias Klugmann
    • 4
  1. 1.CNS Gene Therapy Center, Department of NeurosurgeryJefferson Medical CollegePhiladelphia
  2. 2.Department of Molecular Medicine & Pathology, Faculty of Medical and Health SciencesThe University of AucklandAucklandNew Zealand
  3. 3.Department of Pathobiology and Center for Comparative Medical Genetics, School of Veterinary MedicineUniversity of Pennsylvania and Department of Neurology and Neuroscience Research, Children’s Hospital of PhiladelphiaPhiladelphia
  4. 4.Department of Molecular Medicine & Pathology, Faculty of Medical and Health SciencesThe University of AucklandAucklandNew Zealand

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