Skip to main content
Fig. 8 | Stem Cell Research & Therapy

Fig. 8

From: Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies

Fig. 8

Graphical abstract of the work. The HSPCs were electroporated with Cas9-RNP and ssODN to introduce two naturally occurring beneficial mutations (-175T > C and − 158 C > T) and a PAM shield mutation (-153G > C). The electroporated HSPCs were then co-cultured with BM-MSCs and AZD-7648 small molecule. This process resulted in HBG promoter activation, with over 70% of HSPCs containing all three conversions, minimal indels, and no compromise on cell viability. The erythroblasts from the edited HSPCs had high HbF levels

Back to article page

Stem Cell Research & Therapy

ISSN: 1757-6512