GENE THERAPY FOR NEUROMETABOLIC DISORDERS

摘要

PROBLEM TO BE SOLVED: To provide methods for treating neurometabolic disorders including lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease.SOLUTION: Provided compositions comprise adeno-associated virus (AAV) vectors including transgenes encoding lysosomal hydrolases and metalloendopeptidases. The methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and that transgene product is expressed distally to the administration site.