Genzyme Corporation;Samsung Life Public Welfare Foundation
发明人:
Nelson YEW,Dong-Kyu JIN
申请号：
US16061579
公开号：
US20200261598A1
申请日:
2016.12.14
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising nucleic acid encoding N-acetyl-glucosamine-1-phosphate transferase, alpha and beta subunits (GNPTAB) and at least one AAV inverted terminal repeat (ITR). In some embodiments, the rAAV vectors may be included in a rAAV particle, which may be contained in pharmaceutical compositions and kits. These vectors, particles, compositions, and kits may find use, inter alia, in methods and uses related to treating mucolipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal, or related to increasing body size, bone mineral content, and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).
