METHODS AND COMPOSITIONS FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES

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专利权人:

Sangamo Therapeutics; Inc.

发明人:

Edward J. Rebar

申请号：

US16842139

公开号：

US20200231989A1

申请日:

2020.04.07

申请国别(地区):

US

年份:

2020

代理人:

摘要：

Nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of a lysosomal storage disease.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/