Academic Journal
CRISPR and Beyond: Genome-Editing Strategies in Retinal Stem Cell Research.
| Τίτλος: | CRISPR and Beyond: Genome-Editing Strategies in Retinal Stem Cell Research. |
|---|---|
| Συγγραφείς: | Woronkowicz M; North Devon District Hospital, Royal Devon University Healthcare NHS Foundation Trust, Barnstaple EX31 4JB, UK.; Moorfields Eye Hospital NHS Foundation Trust, London EC1V 2PD, UK., Thomas MN; North Devon District Hospital, Royal Devon University Healthcare NHS Foundation Trust, Barnstaple EX31 4JB, UK., Saram SJ; Ministry of Health Singapore, 16 College Road College of Medicine Building, Singapore 169854, Singapore., Carr AF; Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK., Alonso-Carriazo Fernandez A; Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK., Butt Z; Institute of Ophthalmology, University College London, 11-43 Bath Street, London EC1V 9EL, UK., Skopiński P; Department of Ophthalmology, SPKSO Ophthalmic University Hospital, Medical University of Warsaw, 00-576 Warsaw, Poland.; Department of Histology and Embryology, Medical University of Warsaw, 02-004 Warsaw, Poland., Ramsden CM; Faculty of Health and Life Science, University of Exeter Medical School, Exeter EX1 2HZ, UK.; West of England Eye Unit, Royal Devon University Healthcare NHS Foundation Trust, Exeter EX2 5DW, UK. |
| Πηγή: | Cells [Cells] 2026 Mar 10; Vol. 15 (6). Date of Electronic Publication: 2026 Mar 10. |
| Τύπος έκδοσης: | Journal Article; Review |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409 (Electronic) Linking ISSN: 20734409 NLM ISO Abbreviation: Cells Subsets: MEDLINE |
| Imprint Name(s): | Original Publication: Basel, Switzerland : MDPI |
| Ιατρικοί όροι (MeSH): | Gene Editing*/methods , CRISPR-Cas Systems*/genetics , Retina*/cytology , Retina*/metabolism , Stem Cell Research*, Induced Pluripotent Stem Cells/metabolism ; Embryonic Stem Cells/metabolism ; Humans ; Animals |
| Περίληψη: | Genome editing has emerged as a transformative approach for understanding and treating retinal degenerative diseases. Combining this technology with pluripotent stem cells provides an ideal platform for modeling human development and disease, and investigating emerging therapeutic strategies ultimately aimed towards in vivo correction. This approach enables both functional studies to understand retinal degeneration and the early development of targeted therapies for inherited disease. This review offers a comprehensive overview of genome-editing techniques and the ability to create new clinically relevant models to understand human disease in retinal research, focusing on the use of the CRISPR-Cas9 system in induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs), as well as highlighting recent advancements in base and prime editing. Gene editing in various retinal diseases is discussed in context of studies focusing on disease modeling or developing therapeutic strategies. Continued refinement of these techniques will be essential for advancing translational applications in retinal disease treatment. |
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| Contributed Indexing: | Keywords: CRISPR-Cas9; ESCs; TALENs; ZFNs; base editing; iPSCs; prime editing; retina; stem cells |
| Entry Date(s): | Date Created: 20260327 Date Completed: 20260714 Latest Revision: 20260813 |
| Update Code: | 20260813 |
| PubMed Central ID: | PMC13026061 |
| DOI: | 10.3390/cells15060489 |
| PMID: | 41892280 |
| Βάση Δεδομένων: | MEDLINE |
| ISSN: | 2073-4409 |
|---|---|
| DOI: | 10.3390/cells15060489 |