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Peer-reviewedCC BY-NC-NDStem cell research & therapy

Therapeutic applications of stem cells in human diseases.

  • Cheng Hao1
  • Mansoor Bolideei2,3
  • Jamal Alshorman4
  • Kingsley Miyanda Tembo3
  • Mahdi Morshedi Yekta5
  • Mohammad Javad Mehran6
  • Zhe Zhang7
7 affiliations
  1. 1National Demonstration Center for Experimental General Medicine Education, Xianning Medical College, Hubei University of Science and Technology, Xianning 437100, China.
  2. 2Department of Medical Laboratory Sciences, Chabahar University of Medical Sciences, Chabahar, Iran.
  3. 3Department of Respiratory and Critical Care Medicine, The Center for Biomedical Research, NHC Key Laboratory of Respiratory Diseases, Tongji Hospital, Tongji Medical College, Huazhong University of Sciences and Technology, Wuhan, China.
  4. 4Department of Orthopedics, The Second Affiliated Hospital of Hainan Medical University, Haikou, 570311, China.
  5. 5Department of Medical Biotechnology, Faculty of Medicine, Kermanshah University of Medical Sciences, Kermanshah, 6714415185, Iran.
  6. 6Department of Medical Laboratory Sciences, Chabahar University of Medical Sciences, Chabahar, Iran. [email protected].
  7. 7National Demonstration Center for Experimental General Medicine Education, Xianning Medical College, Hubei University of Science and Technology, Xianning 437100, China. [email protected].
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Abstract

Stem-cell-based interventions encompass biologically distinct therapeutic strategies with markedly different levels of clinical maturity. Hematopoietic stem cell transplantation is an established treatment for selected hematologic disorders, whereas many other approaches remain investigational and require more rigorous evidence of durable efficacy and long-term safety. This review critically examines the major cellular platforms used in regenerative medicine, including embryonic stem cells (ESCs), tissue-specific adult stem cells, hematopoietic stem cells (HSCs), mesenchymal stromal cells (MSCs), and induced pluripotent stem cells (iPSCs). We compare their biological properties, therapeutic rationale, translational suitability, and principal limitations across cardiovascular, ocular, hepatic, oncologic, neurodegenerative, gastrointestinal, and autoimmune diseases. Particular attention is given to the distinction between direct cell replacement and indirect reparative effects mediated by immunomodulation, trophic signaling, angiogenesis, extracellular vesicles, and remodeling of the tissue microenvironment. We also discuss enabling technologies, including genome editing, organoid systems, and stem-cell-derived extracellular vesicles, as well as the major barriers to clinical translation. These include donor- and source-dependent heterogeneity, limited engraftment and persistence, incomplete differentiation or maturation, tumorigenic and immunologic risks, manufacturing reproducibility, quality-control requirements, and regulatory complexity. Rather than functioning as interchangeable therapeutic products, different stem-cell platforms are likely to have disease-specific roles that depend on their mechanism of action and the biological requirements of the target tissue. Greater mechanistic precision, standardized product characterization, and carefully designed clinical studies will be essential for broader and more reliable implementation.

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Cite

BibTeXRIS
BibTeX
@article{hao2026therapeutic,
  title = {{Therapeutic applications of stem cells in human diseases.}},
  author = {Cheng Hao and Mansoor Bolideei and Jamal Alshorman and Kingsley Miyanda Tembo and Mahdi Morshedi Yekta and Mohammad Javad Mehran and Zhe Zhang},
  journal = {Stem cell research \& therapy},
  year = {2026},
  month = jul,
  doi = {10.1186/s13287-026-05203-1},
  url = {https://europepmc.org/article/MED/42972583},
  note = {CC BY-NC-ND},
}
RIS
TY  - JOUR
TI  - Therapeutic applications of stem cells in human diseases.
AU  - Cheng Hao
AU  - Mansoor Bolideei
AU  - Jamal Alshorman
AU  - Kingsley Miyanda Tembo
AU  - Mahdi Morshedi Yekta
AU  - Mohammad Javad Mehran
AU  - Zhe Zhang
PY  - 2026
DA  - 2026/07/31
JO  - Stem cell research & therapy
DO  - 10.1186/s13287-026-05203-1
UR  - https://europepmc.org/article/MED/42972583
AB  - Stem-cell-based interventions encompass biologically distinct therapeutic strategies with markedly different levels of clinical maturity. Hematopoietic stem cell transplantation is an established treatment for selected hematologic disorders, whereas many other approaches remain investigational and require more rigorous evidence of durable efficacy and long-term safety. This review critically examines the major cellular platforms used in regenerative medicine, including embryonic stem cells (ESCs), tissue-specific adult stem cells, hematopoietic stem cells (HSCs), mesenchymal stromal cells (MSCs), and induced pluripotent stem cells (iPSCs). We compare their biological properties, therapeutic rationale, translational suitability, and principal limitations across cardiovascular, ocular, hepatic, oncologic, neurodegenerative, gastrointestinal, and autoimmune diseases. Particular attention is given to the distinction between direct cell replacement and indirect reparative effects mediated by immunomodulation, trophic signaling, angiogenesis, extracellular vesicles, and remodeling of the tissue microenvironment. We also discuss enabling technologies, including genome editing, organoid systems, and stem-cell-derived extracellular vesicles, as well as the major barriers to clinical translation. These include donor- and source-dependent heterogeneity, limited engraftment and persistence, incomplete differentiation or maturation, tumorigenic and immunologic risks, manufacturing reproducibility, quality-control requirements, and regulatory complexity. Rather than functioning as interchangeable therapeutic products, different stem-cell platforms are likely to have disease-specific roles that depend on their mechanism of action and the biological requirements of the target tissue. Greater mechanistic precision, standardized product characterization, and carefully designed clinical studies will be essential for broader and more reliable implementation.
SN  - 1757-6512
ER  -