THE REGENTS OF THE UNIVERSITY OF MICHIGAN;UCL BUSINESS LTD
发明人:
THOMPSON, DEBRA A.,ALI, ROBIN R.,SMITH, ALEXANDER J.
申请号：
CA3082586
公开号：
CA3082586A1
申请日:
2018.11.15
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
Provided are materials, methods and uses for treating an ophthalmological condition such as Leber Congenital Amaurosis by administering an effective amount of an adeno-associated virus AAV2, serotype 5 (AAV 2/5) or AAV-5 comprising an expressible coding region for human RDH12.
