WILSON, JAMES M.,HINDERER, CHRISTIAN,HORDEAUX, JULIETTE
申请号：
CA3083761
公开号：
CA3083761A1
申请日:
2018.11.29
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5' inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional human N-acetyl-alpha-glucosaminidase (hNAGLU), a regulatory sequence which direct expression of hNAGLU in a target cell, and an AAV 3 ' ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIB.
