METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY DISEASE
- 专利权人:
- 发明人:
- Marshall W. Huston
- 申请号：
- US15788059
- 公开号：
- US20180117181A1
- 申请日:
- 2017.10.19
- 申请国别(地区):
- US
- 年份:
- 2018
- 代理人:
- 摘要：
- Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic α-Gal A protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of Fabry disease.
- 来源网站：
- 中国工程科技知识中心
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