RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING DISEASES INVOLVING DOMINANT-NEGATIVE OR GAIN OF FUNCTION MUTATIONS
- 专利权人:
- 发明人:
- 申请号：
- EP17201358.3
- 公开号：
- EP3318634A1
- 申请日:
- 2012.04.20
- 申请国别(地区):
- EP
- 年份:
- 2018
- 代理人:
- 摘要：
- The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases caused by dominant-negative or gain of function mutations
- 来源网站：
- 中国工程科技知识中心
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