ADENO-ASSOCIATED VIRUS VECTOR FOR THERAPEUTIC DELIVERY INTO THE CENTRAL NERVOUS SYSTEM

摘要

FIELD: biotechnology.;SUBSTANCE: invention relates to methods for preventing, inhibiting or treating one or more symptoms associated with a central nervous system disease, by intranasal, intrathecal, intracerebrovascular or intravenous introduction of rAAV encoding a gene product associated with a disease, e.g. a mammal, in which a gene product is absent or present at a lower level compared to a mammal not having the disease, in an amount effective, e.g. to provide cross-correction.;EFFECT: disclosed is an adeno-associated virus vector for therapeutic delivery into the central nervous system.;21 cl, 42 dwg, 8 ex