Abstract
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.
Highlights
Over a decade ago, human cellular reprogramming from adult somatic cells to pluripotent state, so-called human induced pluripotent stem cells was successfully discovered [1,2]
This study demonstrated the use of a clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system to induce both disease progression and reversal via genetic correction or introduction of mutations, which may aid in the mechanistic understanding of myeloid transformation and drug testing for stage-specific therapeutic interventions [135]
Several concerns still limit the validity of human induced pluripotent stem cells (hiPSCs)-based disease modeling, most of these limitations can be overcome by following some general guidelines or by mindfully interpreting the data
Summary
Human cellular reprogramming from adult somatic cells to pluripotent state, so-called human induced pluripotent stem cells (hiPSCs) was successfully discovered [1,2]. Due to their unique properties of unlimited self-renewal and the ability to differentiate into all cell types of three primordial germ layers, termed ‘pluripotency’, hiPSCs offer numerous opportunities for a wide range of research and clinical applications. We summarize the uses of hiPSCs as a research tool for hematologic disorders and highlight recent hereditary and acquired hematologic disease modeling with patient-specific iPSCs. We provide current applications of hiPSCs as instrumental drug screening and potential therapeutic candidates for hematologic disorders, with or without further genetic corrections or modifications. We highlight and analyze current challenges, considerations, and future research and clinical directions
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
