An integration-free iPSC line (MUSIi008-A) derived from a patient with severe hemolytic anemia carrying compound heterozygote mutations in KLF1 gene for disease modeling

Ponthip PotiratMethichit WattanapanitchVip ViprakasitPakpoom KheolamaiSurapol IssaragrisilFaculty of Medicine, Thammasat UniversityFaculty of Medicine, Siriraj Hospital, Mahidol University2020-01-272020-01-272019-01-01Stem Cell Research. Vol.34, (2019)18767753187350612-s2.0-85059233299https://repository.li.mahidol.ac.th/handle/123456789/50364© 2018 The Authors We generated an induced pluripotent stem cell (iPSC) line from peripheral blood mononuclear cells (PBMNCs) isolated from a 1-year old female carrying compound heterozygote for KLF1 mutations (G176RfsX179 and A298P mutations). Non-integrating Sendai viral (SeV) vectors containing KOS, hc-MYC and hKLF4 were used for reprogramming. The established MUSIi008-A cell line contained the same mutations found in the patient, expressed pluripotent markers, differentiated into cells of three embryonic germ layers both in vitro and in vivo, and exhibited normal karyotype. This cell line may provide an alternative renewable source of cells for in vitro disease modeling of severe transfusion-dependent hemolytic anemia.Mahidol UniversityBiochemistry, Genetics and Molecular BiologyAn integration-free iPSC line (MUSIi008-A) derived from a patient with severe hemolytic anemia carrying compound heterozygote mutations in KLF1 gene for disease modelingArticleSCOPUS10.1016/j.scr.2018.09.021