Gene therapy for lysosomal storage diseases

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.

主权项

1. A method to deliver acid beta glucosidase to the central nervous system in a subject having Gaucher disease, comprising:
administering a recombinant AAV4 or AAV5 viral vector comprising a transgene encoding acid beta glucosidase to at least one ventricle of the brain selected from the group consisting of a lateral ventricle and the fourth ventricle by intraventricular administration, whereby said transgene is expressed and acid beta glucosidase is delivered to the central nervous system, wherein the subject is a mammal.