Protocol

Viral Vectors for Gene Therapy

Volume 76 of the series Methods in Molecular Medicine™ pp 221-236

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

  • Matthew J. DuringAffiliated withCNS Gene Therapy Center, Department of Neurosurgery, Jefferson Medical CollegeDepartment of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland
  • , Deborah YoungAffiliated withDepartment 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
  • , Kristin BaerAffiliated withDepartment of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland
  • , Patricia LawlorAffiliated withDepartment of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland
  • , Matthias KlugmannAffiliated withDepartment of Molecular Medicine & Pathology, Faculty of Medical and Health Sciences, The University of Auckland

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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].