Search International and National Patent Collections

1. (WO2018085688) GENE TRANSFER COMPOSITIONS, METHODS AND USES FOR TREATING NEURODEGENERATIVE DISEASES

Pub. No.:    WO/2018/085688    International Application No.:    PCT/US2017/059986
Publication Date: Sat May 12 01:59:59 CEST 2018 International Filing Date: Sat Nov 04 00:59:59 CET 2017
IPC: A61K 31/343
A61K 31/5377
A61K 35/76
Applicants: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Inventors: DAVIDSON, Beverly L.
CHEN, Yong Hong
TECEDOR, Luis
Title: GENE TRANSFER COMPOSITIONS, METHODS AND USES FOR TREATING NEURODEGENERATIVE DISEASES
Abstract:
Provided are methods of treating a lysosomal storage disorder in a mammal which method includes administering AAV particles encoding a polypeptide to the central nervous system of the mammal. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof for expression.