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COMPOSITIONS AND METHODS FOR STEM CELL TRANSPLANT
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:
SG11202008400S
公开号:
SG11202008400SA
申请日:
2019.04.11
申请国别(地区):
SG
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/
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