Jiliang Gao,Philip M. Murphy,David H. McDermott,Marie Siwicki,Harry L. Malech,Joy Liu,Paejonette Jacobs
申请号：
US15324219
公开号：
US20170196911A1
申请日:
2015.07.17
申请国别(地区):
US
年份:
2017
代理人:
摘要：
Provided herein are methods of enhancing engraftment of hematopoietic stem cells (HSC) in recipient subjects by reducing expression (or activity) of CXC chemokine receptor 4 (CXCR4). Such methods can be used in gene therapy protocols and in HSC transplantation, for example allowing this to occur without radiation or chemotherapy conditioning as is typically done in non-myeloablative HSC transplant. In some examples, gene editing methods are used to delete one copy of the CXCR4 gene before HSC or bone marrow transplantation, enhancing the efficiency and durability of donor cell repopulation.
