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

Potirat, Ponthip; Wattanapanitch, Methichit; Viprakasit, Vip; Kheolamai, Pakpoom; Issaragrisil, Surapol

doi:10.1016/j.scr.2018.09.021

Cited by 1 publication

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“…Moreover, this study suggested the underlying mechanisms of the p.E325K mutation, which involve the disruption of cell cycle regulators, the cell membrane, and enzyme abnormalities [104]. Recently, our group has generated an hiPSC line from a pediatric patient with severe hemolytic anemia carrying compound heterozygote mutations in KLF1 (G176RfsX179 and A298P) [145]. Subsequently, the hiPSCs were differentiated toward the erythroid cells.…”

Section: Hereditary Hematologic Disordersmentioning

confidence: 84%

Induced Pluripotent Stem Cells as a Tool for Modeling Hematologic Disorders and as a Potential Source for Cell-Based Therapies

Pratumkaew

Issaragrisil

Luanpitpong

2021

Cells

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The breakthrough in human induced pluripotent stem cells (hiPSCs) has revolutionized the field of biomedical and pharmaceutical research and opened up vast opportunities for drug discovery and regenerative medicine, especially when combined with gene-editing technology. Numerous healthy and patient-derived hiPSCs for human disease modeling have been established, enabling mechanistic studies of pathogenesis, platforms for preclinical drug screening, and the development of novel therapeutic targets/approaches. Additionally, hiPSCs hold great promise for cell-based therapy, serving as an attractive cell source for generating stem/progenitor cells or functional differentiated cells for degenerative diseases, due to their unlimited proliferative capacity, pluripotency, and ethical acceptability. In this review, we provide an overview of hiPSCs and their utility in the study of hematologic disorders through hematopoietic differentiation. We highlight recent hereditary and acquired genetic hematologic disease modeling with patient-specific iPSCs, and discuss their applications as instrumental drug screening tools. The clinical applications of hiPSCs in cell-based therapy, including the next-generation cancer immunotherapy, are provided. Lastly, we discuss the current challenges that need to be addressed to fulfill the validity of hiPSC-based disease modeling and future perspectives of hiPSCs in the field of hematology.

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Section: Hereditary Hematologic Disordersmentioning

confidence: 84%