USE OF APOPTOSIS INHIBITING COMPOUNDS IN DEGENERATIVE NEUROLOGICAL DISORDERS

摘要

The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain associated with neurodegenerative diseases that are characterized by an excess buildup of intracellular protein aggregates. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding an inhibitor of apoptosis protein (IAP) to cells in the region.