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

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

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 hKLF...

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Main Authors: Ponthip Potirat, Methichit Wattanapanitch, Vip Viprakasit, Pakpoom Kheolamai, Surapol Issaragrisil
Format: Article
Language:English
Published: Elsevier 2019-01-01
Series:Stem Cell Research
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506118302745
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spelling doaj-443b89e67a294b78aa0cef2631a8b4d22020-11-25T00:29:08ZengElsevierStem Cell Research1873-50612019-01-0134An integration-free iPSC line (MUSIi008-A) derived from a patient with severe hemolytic anemia carrying compound heterozygote mutations in KLF1 gene for disease modelingPonthip Potirat0Methichit Wattanapanitch1Vip Viprakasit2Pakpoom Kheolamai3Surapol Issaragrisil4Graduate Program in Immunology, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand; Siriraj Center of Excellence for Stem Cell Research, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, ThailandSiriraj Center for Regenerative Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, ThailandDivision of Pediatric Hematology/Oncology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, ThailandDivision of Cell Biology, Department of Pre-clinical Sciences, Faculty of Medicine, Thammasat University, Pathumthani, ThailandSiriraj Center of Excellence for Stem Cell Research, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand; Division of Hematology, Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand; Corresponding author at: Siriraj Center of Excellence for Stem Cell Research, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.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.http://www.sciencedirect.com/science/article/pii/S1873506118302745
collection DOAJ
language English
format Article
sources DOAJ
author Ponthip Potirat
Methichit Wattanapanitch
Vip Viprakasit
Pakpoom Kheolamai
Surapol Issaragrisil
spellingShingle 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
author_facet Ponthip Potirat
Methichit Wattanapanitch
Vip Viprakasit
Pakpoom Kheolamai
Surapol Issaragrisil
author_sort Ponthip Potirat
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
title_short 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_sort integration-free ipsc line (musii008-a) derived from a patient with severe hemolytic anemia carrying compound heterozygote mutations in klf1 gene for disease modeling
publisher Elsevier
series Stem Cell Research
issn 1873-5061
publishDate 2019-01-01
description 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.
url http://www.sciencedirect.com/science/article/pii/S1873506118302745
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