STEWART, MORAG HELEN,VALLASTER, MARKUS PARZIVAL,MONTI, ANTHONY,NARENDRA, POOJA KYATSANDRA,HO, QUAN
申请号:
CA3077391
公开号:
CA3077391A1
申请日:
2018.09.28
申请国别(地区):
CA
年份:
2019
代理人:
摘要:
The present disclosure relates to compositions and methods for introducing an mRNA into stem cells, such asHSPCs, and for delivering gene editing components to such cells in vitro. For example, the disclosure relates to modifying a gene sequence using a CR1SPR-Cas9 complex in HSPCs, and methods and delivery systems for achieving such gene modification in HSPCs.