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Peer-reviewedCC BY 4.0Cells

CRISPR-Based Engineering of Mesenchymal Stem Cells: Functional Enhancement, Translational Challenges, and Clinical Perspectives

  • Geofanny Facicilia1,2
  • Cynthia Retna Sartika2
  • Yuni Elsa Hadisaputri1
  • Tina Rostinawati1
  1. 1Department of Pharmaceutical Biology, Faculty of Pharmacy, Universitas Padjadjaran, Jatinangor 45363, Indonesia
  2. 2Prodia StemCell Indonesia, DKI Jakarta, Jakarta 13410, Indonesia
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Abstract

Mesenchymal stem cells (MSCs) are promising platforms for regenerative and immunomodulatory therapies, but their clinical performance is limited by donor heterogeneity, variable potency, and limited persistence. CRISPR-based engineering provides programmable approaches to modify MSC function through genome editing and transcriptional regulation. This review examines CRISPR/Cas9-mediated knockout and knock-in, dCas9-based transcriptional regulation, and emerging base-, prime-, and epigenome-editing technologies for MSC engineering. These approaches can enhance survival and stress resistance, homing and tissue retention, paracrine and extracellular vesicle activity, immunomodulation, immune compatibility, and lineage-associated functions. Key translational challenges include efficient delivery, engineered-population heterogeneity, genomic safety, immunogenicity, phenotypic stability, scalable manufacturing, and mechanism-linked potency assessment. The appropriate engineering strategy should therefore be selected according to the intended therapeutic mechanism and product development requirements. Clinical translation will ultimately depend on demonstrating reproducible functional enhancement, manufacturing consistency, genomic and phenotypic stability, and validated therapeutic potency.

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Cite

BibTeXRIS
BibTeX
@article{facicilia2026crisprbased,
  title = {{CRISPR-Based Engineering of Mesenchymal Stem Cells: Functional Enhancement, Translational Challenges, and Clinical Perspectives}},
  author = {Geofanny Facicilia and Cynthia Retna Sartika and Yuni Elsa Hadisaputri and Tina Rostinawati},
  journal = {Cells},
  year = {2026},
  month = nov,
  doi = {10.3390/cells15212029},
  url = {https://www.mdpi.com/2073-4409/15/21/2029},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - CRISPR-Based Engineering of Mesenchymal Stem Cells: Functional Enhancement, Translational Challenges, and Clinical Perspectives
AU  - Geofanny Facicilia
AU  - Cynthia Retna Sartika
AU  - Yuni Elsa Hadisaputri
AU  - Tina Rostinawati
PY  - 2026
DA  - 2026/11/06
JO  - Cells
DO  - 10.3390/cells15212029
UR  - https://www.mdpi.com/2073-4409/15/21/2029
AB  - Mesenchymal stem cells (MSCs) are promising platforms for regenerative and immunomodulatory therapies, but their clinical performance is limited by donor heterogeneity, variable potency, and limited persistence. CRISPR-based engineering provides programmable approaches to modify MSC function through genome editing and transcriptional regulation. This review examines CRISPR/Cas9-mediated knockout and knock-in, dCas9-based transcriptional regulation, and emerging base-, prime-, and epigenome-editing technologies for MSC engineering. These approaches can enhance survival and stress resistance, homing and tissue retention, paracrine and extracellular vesicle activity, immunomodulation, immune compatibility, and lineage-associated functions. Key translational challenges include efficient delivery, engineered-population heterogeneity, genomic safety, immunogenicity, phenotypic stability, scalable manufacturing, and mechanism-linked potency assessment. The appropriate engineering strategy should therefore be selected according to the intended therapeutic mechanism and product development requirements. Clinical translation will ultimately depend on demonstrating reproducible functional enhancement, manufacturing consistency, genomic and phenotypic stability, and validated therapeutic potency.
SN  - 2073-4409
ER  -