INTRANASAL DELIVERY OF AAV ENCODING THERAPEUTIC ENZYMES TO THE CENTRAL NERVOUS SYSTEM FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES

摘要

The invention provides a method to prevent, inhibit or treat one or more neurological symptoms associated with a lysosomal storage disease in a mammal in need thereof, which includes intranasally administering to the mammal a composition comprising an effective amount of a lysosomal storage enzyme or a recombinant adeno-associated virus vector comprising an open reading frame encoding a lysosomal storage enzyme. Also provided are compositions and devices useful in the methods.