Miguel Sena-Esteves,Timothy M. Cox,Maria Begona Cachon-Gonzalez,Thomas N. Seyfried
申请号：
US13452339
公开号：
US20130090374A1
申请日:
2012.04.20
申请国别(地区):
US
年份:
2013
代理人:
摘要：
The present disclosure provides methods for the treatment of lysosomal storage disorders using gene replacement therapy. In particular, methods are provided for the treatment of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis using enzyme replacement therapy. Expression constructs encoding enzymes required for ganglioside metabolism are delivered to the brain of subjects with an enzyme deficiency. Methods are also provided for delaying the onset of, reducing the likelihood of onset of, or reducing the severity of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis.
