Use of a recombinant adeno-associated virus (aav) aav4 or aav5 vector comprising a transgene encoding sphingomyelinase in the manufacture of a medicament formulated for intraventricular administration for distribution throughout the ependymal cell layer of the ventricular system for treating niemann-pick a disease or niemann-pick b disease

摘要

This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.