The Significance of Induced Pluripotent Stem Cells in Translational Medicine

Induced pluripotent stem cells (iPSCs) are generated from human somatic cells (e.g. skin or blood cells) by reprogramming technology to regain an embryonic ‘stemness’, which not only enables their differentiation into any cell type but also bypasses any ethical considerations since they no longer derive from human embryos. iPSCs represent a groundbreaking development, holding tremendous potential to revolutionize medicine and improve our understanding of human biology. Specifically, iPSCs use is of substantial importance in the
a) study and modeling human diseases, especially those that are poorly recapitulated in animal models (e.g. autism, schizophrenia, etc.) or require hard to access and age-related cell types (e.g. neurons in Alzheimer’s Disease)
b) validation of disease-causing role for inherited gene mutations (e.g. isogenic model)
c) drug development and screening of its efficacy, toxicity and mechanism(s) of action,
d) regenerative medicine and cell-based therapy, autologous or allogeneic, that can be genetically engineered or clonally expanded.

iPSCs hold great promise to restore tissue homeostasis and function. Despite the advantages in translational precision medicine, iPSC-based applications pose major challenges regarding safety, quality assurance and logistics. For instance, a major concern is the propensity of iPSCs for teratoma formation and genomic alterations acquired during reprogramming, prolonged culture or gene editing. It is critical, therefore, to ensure that undifferentiated iPSCs and stem cell-like intermediates are completely absent from the cellular product that will be transplanted into the patient and to screen for potentially harmful genomic alterations to prevent tumour formation. Also, incomplete maturation of iPSCs-derived cells remains a major hurdle in developing efficacious cell therapies. A potential action to counterbalance such an effect is to use organoids and in vivo niche mimicking to improve iPSC differentiation and maturation through cell-cell interaction and networking. Finally, challenges related to reproducibility, consistency and logistics may require a) sharing of optimization protocols related to iPSC derivation, maintenance, and differentiation, b) decentralization of manufacturing processes and c) automation. This Research Topic aims to outline the significance of IPSC-based disease modelling and cell therapy approaches by exploring the challenges and providing the counteracting measures to improve the efficacy, effectiveness and efficiency of iPSCs usage.

To explore the constantly increasing use of iPSCs-based applications, we welcome manuscripts focusing but not limited to the following sub-topics:
• Modeling human diseases using IPS-derived cellular models (e.g. co-cultures, organoids)
• IPSC-based drug screening and development or repurposing
• Clinical trials using iPS-derived cellular products
• Optimization protocols for the efficient and efficacious use of iPS-derived cells
• Framework profiling the design, quality control, clinical testing and regulatory validity of iPSCs use
• Strategies to reduce graft-versus-host disease using allogeneic iPS-derived cellular products
• Potential measures to overcome hurdles in iPSC-based therapy safety, efficiency and logistics, e.g.
o Propensity for teratoma formation
o Genomic stability
o Limited therapeutic response
o Reproducibility
o Manufacturing process

This Research Topic accepts Original Research, Systematic Review, Methods, Review and Mini-Review, Hypothesis & Theory, Clinical Trial, Classification, Technology and Code, Study Protocol, Perspective, Case Report, Conceptual Analysis, Brief Research Report, Data Report, General Commentary.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Classification
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Classification
• Clinical Trial
• Editorial
• FAIR² Data
• General Commentary
• Hypothesis and Theory
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Review
• Systematic Review
• Technology and Code

Keywords: induced pluripotency, precision medicine, cell therapy, challenges, measures

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.