GENE THERAPY FOR NEUROMETABOLIC DISORDERS

摘要

PROBLEM TO BE SOLVED: To provide a method for treating disorders affecting the central nervous system including neurometabolic disorders such as lysosomal storage diseases, e.g. Niemann-Pick A disease.

SOLUTION: The method includes: a composition containing an adeno-associated virus (AAV) vector which contains a transgene encoding a lysosomal hydrolase and a metalloendopeptidase; and a step of contacting an axonal ending of the neuron with a composition containing high titer AAV having a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and the transgene product is expressed distally to the administration site.

COPYRIGHT: (C)2012,JPO&INPIT