The University of Manchester;Centre Hospitalier Universitaire Sainte-Justine;Alexey , Pchejetsky
发明人:
Alexey Pchejetsky,Brian Bigger,Claire O'Leary
申请号：
US16317126
公开号：
US20190314522A1
申请日:
2017.07.12
申请国别(地区):
US
年份:
2019
代理人:
摘要：
The present invention relates to compositions and adeno associated viral vectors comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof. Uses of such compositions and vectors are also contemplated along with kits of parts for their administration.
