The safety and effectiveness of genetically corrected iPSCs derived from β-thalassaemia patients in nonmyeloablative β-thalassaemic mice.

Stem Cell Research & Therapy
Yexing XianXiaofang Sun

Abstract

β-Thalassaemia is a clinically common cause of hereditary haemolytic anaemia stemming from mutations in important functional regions of the β-globin gene. The rapid development of gene editing technology and induced pluripotent stem cell (iPSC)-derived haematopoietic stem cell (HSC) transplantation has provided new methods for curing this disease. Genetically corrected β-thalassaemia (homozygous 41/42 deletion) iPSCs that were previously established in our laboratory were induced to differentiate into HSCs, which were transplanted into a mouse model of IVS2-654 β-thalassaemia (B6;129P2-Hbbtm2Unc/J mice) after administration of an appropriate nonmyeloablative conditioning regimen. We also investigated the safety of this method by detecting the incidence of tumour formation in these mice after transplantation. The combination of 25 mg/kg busulfan and 50 mg/(kg day) cyclophosphamide is an ideal nonmyeloablative protocol before transplantation. Genetically corrected β-thalassaemic HSCs survived and differentiated in nonmyeloablated thalassaemia mice. No tumour formation was observed in the mice for 10 weeks after transplantation. Our study provides evidence that the transplantation of genetically corrected, patient-specific iPSCs c...Continue Reading

References

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Citations

Jul 3, 2021·International Journal of Molecular Sciences·Dito AnurogoJeanne Adiwinata Pawitan

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Methods Mentioned

BETA
FACS
flow cytometry
Flow
genetic modification

Software Mentioned

SPSS

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