Stem Cell Therapy for Retinitis Pigmentosa: Promise, Evidence, and the Risk of Premature Clinical Claims
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Sep 22, 2026
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Keywords
Stem cell
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KARTASASMITA, Arief.
Stem Cell Therapy for Retinitis Pigmentosa: Promise, Evidence, and the Risk of Premature Clinical Claims.
International Journal of Retina, [S.l.], v. 9, n. 2, p. 1-3, sep. 2026.
ISSN 2614-8536.
Available at: <https://www.ijretina.com/index.php/ijretina/article/view/363>. Date accessed: 23 sep. 2026.
doi: https://doi.org/10.35479/ijretina.2026.vol009.iss002.363.
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Short Communication
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
References
References
1. Krungkraipetch L, Supajitgulchai D, Assawaboonyadech A, Puranawit W. Efficacy and safety of mesenchymal stem cell therapies in retinitis pigmentosa: a systematic review and meta-analysis. Int Ophthalmol. 2025;45:85. doi:10.1007/s10792-025-03478-6. Available from: https://doi.org/10.1007/s10792-025-03478-6
2. Bassuk AG, Zheng A, Li Y, Tsang SH, Mahajan VB. Precision medicine: genetic repair of retinitis pigmentosa in patient-derived stem cells. Sci Rep. 2016;6:19969. doi:10.1038/srep19969. Available from: https://doi.org/10.1038/srep19969
3. Hirami Y, Mandai M, Sugita S, Maeda A, Maeda T, Yamamoto M, et al. Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa. Cell Stem Cell. 2023;30(12):1585-1596.e6. doi:10.1016/j.stem.2023.11.004. Available from: https://doi.org/10.1016/j.stem.2023.11.004
4. U.S. National Library of Medicine. ClinicalTrials.gov. A study to investigate the safety of DSP-3077 after a unilateral eye injection in adults with retinitis pigmentosa. Identifier NCT06891885. Updated 2026 Apr 17. Accessed 2026 Aug 15. Available from: https://clinicaltrials.gov/study/NCT06891885
5. U.S. National Library of Medicine. ClinicalTrials.gov. A study to investigate the safety of OpCT-001 in adults with primary photoreceptor disease. Identifier NCT06789445. Accessed 2026 Aug 15. Available from: https://clinicaltrials.gov/study/NCT06789445
6. Pearson RA, Gonzalez-Cordero A, West EL, Ribeiro JR, Aghaizu N, Goh D, et al. Donor and host photoreceptors engage in material transfer following transplantation of post-mitotic photoreceptor precursors. Nat Commun. 2016;7:13029. doi:10.1038/ncomms13029. Available from: https://doi.org/10.1038/ncomms13029
7. Santos-Ferreira T, Llonch S, Borsch O, Postel K, Haas J, Ader M. Retinal transplantation of photoreceptors results in donor-host cytoplasmic exchange. Nat Commun. 2016;7:13028. doi:10.1038/ncomms13028. Available from: https://doi.org/10.1038/ncomms13028
8. Yang J, Kuppermann BD, Liao D, Mehta MC, Hsiang C, Menges S, et al. Retinal progenitor cells (jCell) for retinitis pigmentosa. Front Cell Neurosci. 2025;19:1646156. doi:10.3389/fncel.2025.1646156. Available from: https://doi.org/10.3389/fncel.2025.1646156
9. International Society for Stem Cell Research. New clinical data presented at ISSCR 2026 demonstrate one-year survival of stem cell-derived neural progenitor cells in patients with retinitis pigmentosa. 2026 Jul 9. Accessed 2026 Aug 15. Available from: https://www.isscr.org/isscr-news/new-clinical-data-presented-at-isscr-2026-demonstrate-one-year-survival-of-stem-cell-derived-neural-progenitor-cells-in-patients-with-retinitis-pigmentosa
10. Kuriyan AE, Albini TA, Townsend JH, Rodriguez M, Pandya HK, Leonard RE 2nd, et al. Vision loss after intravitreal injection of autologous “stem cells” for AMD. N Engl J Med. 2017;376(11):1047-1053. doi:10.1056/NEJMoa1609583. Available from: https://doi.org/10.1056/NEJMoa1609583
1. Krungkraipetch L, Supajitgulchai D, Assawaboonyadech A, Puranawit W. Efficacy and safety of mesenchymal stem cell therapies in retinitis pigmentosa: a systematic review and meta-analysis. Int Ophthalmol. 2025;45:85. doi:10.1007/s10792-025-03478-6. Available from: https://doi.org/10.1007/s10792-025-03478-6
2. Bassuk AG, Zheng A, Li Y, Tsang SH, Mahajan VB. Precision medicine: genetic repair of retinitis pigmentosa in patient-derived stem cells. Sci Rep. 2016;6:19969. doi:10.1038/srep19969. Available from: https://doi.org/10.1038/srep19969
3. Hirami Y, Mandai M, Sugita S, Maeda A, Maeda T, Yamamoto M, et al. Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa. Cell Stem Cell. 2023;30(12):1585-1596.e6. doi:10.1016/j.stem.2023.11.004. Available from: https://doi.org/10.1016/j.stem.2023.11.004
4. U.S. National Library of Medicine. ClinicalTrials.gov. A study to investigate the safety of DSP-3077 after a unilateral eye injection in adults with retinitis pigmentosa. Identifier NCT06891885. Updated 2026 Apr 17. Accessed 2026 Aug 15. Available from: https://clinicaltrials.gov/study/NCT06891885
5. U.S. National Library of Medicine. ClinicalTrials.gov. A study to investigate the safety of OpCT-001 in adults with primary photoreceptor disease. Identifier NCT06789445. Accessed 2026 Aug 15. Available from: https://clinicaltrials.gov/study/NCT06789445
6. Pearson RA, Gonzalez-Cordero A, West EL, Ribeiro JR, Aghaizu N, Goh D, et al. Donor and host photoreceptors engage in material transfer following transplantation of post-mitotic photoreceptor precursors. Nat Commun. 2016;7:13029. doi:10.1038/ncomms13029. Available from: https://doi.org/10.1038/ncomms13029
7. Santos-Ferreira T, Llonch S, Borsch O, Postel K, Haas J, Ader M. Retinal transplantation of photoreceptors results in donor-host cytoplasmic exchange. Nat Commun. 2016;7:13028. doi:10.1038/ncomms13028. Available from: https://doi.org/10.1038/ncomms13028
8. Yang J, Kuppermann BD, Liao D, Mehta MC, Hsiang C, Menges S, et al. Retinal progenitor cells (jCell) for retinitis pigmentosa. Front Cell Neurosci. 2025;19:1646156. doi:10.3389/fncel.2025.1646156. Available from: https://doi.org/10.3389/fncel.2025.1646156
9. International Society for Stem Cell Research. New clinical data presented at ISSCR 2026 demonstrate one-year survival of stem cell-derived neural progenitor cells in patients with retinitis pigmentosa. 2026 Jul 9. Accessed 2026 Aug 15. Available from: https://www.isscr.org/isscr-news/new-clinical-data-presented-at-isscr-2026-demonstrate-one-year-survival-of-stem-cell-derived-neural-progenitor-cells-in-patients-with-retinitis-pigmentosa
10. Kuriyan AE, Albini TA, Townsend JH, Rodriguez M, Pandya HK, Leonard RE 2nd, et al. Vision loss after intravitreal injection of autologous “stem cells” for AMD. N Engl J Med. 2017;376(11):1047-1053. doi:10.1056/NEJMoa1609583. Available from: https://doi.org/10.1056/NEJMoa1609583