Compositions and methods for stem cell transplant

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专利权人:

Rocket Pharmaceuticals, Ltd.;Fundacion para la Investigacion Biomedica del Hospital Infantil Universitario Nino Jesus

发明人:

BEARD, Brian,SEVILLA NAVARRO, Julian,SHAH, Gaurav,PATEL, Kinnari,PRABHAKAR, Raj

申请号：

AU2019252662

公开号：

AU2019252662A1

申请日:

2019.04.11

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

The present invention provides systems and methods for manufacturing and using gene corrected stem cells for gene therapy. In particular, herein provided are methods for treating Fanconi anemia in which a subject's stem cells are selected by a combination of high stringency CD34+ selection and low stringency CD34+ selection, genetically modified using a gene therapy vector encoding a FANC protein or a gene editing system, and administered to the subject.

来源网站：

中国工程科技知识中心

来源网址：

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