Alexander FEDERATION,Li CHAI,Hiro TATETSU,Daniel Geoffrey TENEN,James Elliot BRADNER
申请号:
US15129142
公开号:
US20170173083A1
申请日:
2015.03.25
申请国别(地区):
US
年份:
2017
代理人:
摘要:
Described herein are methods and compositions which lead to the efficient ex vivo expansion of hematopoietic cells, such as hematopoietic stem cells (HSCs) and hematopoietic stem and progenitor cells HSPCs. Using combinations of small molecule drugs and cytokines/growth factors/grown factors targeting epigenetic status in cells, significant improvements in the expansion of cells was observed, including cells isolated from human cord blood or peripheral mobilized stem/progenitor cells. Multiple genes implicated in HSPC function were unperturbed, and efficiency of genomic editing using lentivirus was greatly enhanced following treatment. These novel approaches could be used therapeutically in a variety of hematopoietic transplantation settings, in addition to benefiting gene therapy techniques.