CRISPR/Cas9-mediated Cxcr4disease allele inactivation for gene therapy in a mouse model of WHIM syndrome
- Resource Type
- Article
- Authors
- Gao, Ji-Liang; Owusu-Ansah, Albert; Yang, Alexander; Yim, Erin; McDermott, David H.; Jacobs, Paejonette; Majumdar, Shamik; Choi, Uimook; Sweeney, Colin L.; Malech, Harry L.; Murphy, Philip M.
- Source
- Blood; July 2023, Vol. 142 Issue: 1 p23-32, 10p
- Subject
- Language
- ISSN
- 00064971; 15280020
•A 2-step gene therapy protocol of autologous HSPC transplantation for WHIM syndrome by CRISPR/Cas9-mediated disease allele inactivation.•WHIM disease allele–inactivated HSPCs have a strong selective advantage for durable hematopoietic reconstitution over WHIM cells.