GOSS, KENDRICK A.,PARSONS, GEOFFREY B.,GINIATULLINA, ASIYA
申请号：
CA3058735
公开号：
CA3058735A1
申请日:
2017.06.13
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
The invention provides, in part, compositions and methods for treating neuronal ceroid lipofuscinoses (NCL). The invention further provides, in part, gene therapy compositions and methods for the treatment, prevention, or amelioration of at least one symptom of NCL. Particular embodiments provide a pharmaceutical composition comprising a pharmaceutically acceptable carrier and a lentiviral vector, or a mammalian cell transduced with a lentiviral vector. Further embodiments provide a method of treating NCL comprising administering a lentiviral vector or a mammalian cell transduced with a lentiviral vector.
