Publication:
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

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Issued Date

2019-01-01

Resource Type

Article

ISSN

18767753
18735061

DOI

10.1016/j.scr.2018.09.021

Other identifier(s)

2-s2.0-85059233299

Rights

Mahidol University

Rights Holder(s)

SCOPUS

Bibliographic Citation

Stem Cell Research. Vol.34, (2019)

Suggested Citation

Ponthip Potirat, Methichit Wattanapanitch, Vip Viprakasit, Pakpoom Kheolamai, Surapol Issaragrisil 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. Stem Cell Research. Vol.34, (2019). doi:10.1016/j.scr.2018.09.021 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/50364

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Title

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

Author(s)

Ponthip Potirat
 Methichit Wattanapanitch
 Vip Viprakasit
 Pakpoom Kheolamai
 Surapol Issaragrisil

Other Contributor(s)

Faculty of Medicine, Thammasat University
 Faculty of Medicine, Siriraj Hospital, Mahidol University

Abstract

© 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.

Keyword(s)

Biochemistry, Genetics and Molecular Biology

Availability

URI

https://repository.li.mahidol.ac.th/handle/123456789/50364

Collections

Scopus 2019