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

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 ].