PROCEDES DE TRAITEMENT DE MALADIES NEURODEGENERATIVES UTILISANT LA THERAPIE GENIQUE POUR RETARDER LE DECLENCHEMENT ET L'EVOLUTION DE LA MALADIE TOUT EN CONFERANT UNE PROTECTION COGNITIVE
DAVIDSON, BEVERLY L.,CHEN, YONG HONG,TECEDOR, LUIS
申请号:
CA3002654
公开号:
CA3002654A1
申请日:
2016.10.24
申请国别(地区):
CA
年份:
2017
代理人:
摘要:
The present disclosure provides methods of treating a lysosomal storage disorder in a mammal which method comprises administering AAV particles encoding a polypeptide directly to the central nervous system of the mammal in conjunction with administering at least two immunosuppressive agents. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof.