METHODES ET COMPOSITIONS POUR LE TRAITEMENT DE LA MALADIE DE FABRY

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专利权人:

SANGAMO THERAPEUTICS; INC.

发明人:

HUSTON, MARSHALL W.

申请号：

CA3039673

公开号：

CA3039673A1

申请日:

2017.10.19

申请国别(地区):

CA

年份:

2018

代理人:

摘要：

The disclosure provides nucleases and methods of using these nucleases for inserting a sequence encoding a therapeutic alpha galactosidase A (alpha-Gal A) protein such as an enzyme into a cell, thereby providing proteins or cell therapeutics for treatment and/or prevention of Fabry disease. Further provided are compositions for expressing at least one alpha-Gal A protein.

来源网站：

中国工程科技知识中心

来源网址：

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