Gene Therapy Approaches to Mucolipidosis IV (MLIV)
- 专利权人:
- The General Hospital Corporation
- 发明人:
- Yulia Grishchuk,Susan A. Slaugenhaupt
- 申请号：
- US17772017
- 公开号：
- US20220372520A1
- 申请日:
- 2020.10.29
- 申请国别(地区):
- US
- 年份:
- 2022
- 代理人:
- 摘要：
- Described herein are compositions comprising AAV vectors comprising a sequence encoding mucolipin 1, and methods of use thereof for gene therapy of Mucolipidosis IV (MLIV).
- 来源网站：
- 中国工程科技知识中心
