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Updated: Apr 18, 2026
Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model
Published on: August 5, 2021
Personalized therapeutic strategies for patients with retinitis pigmentosa
Andrew Zheng1, Yao Li, Stephen H Tsang
1Columbia University, College of Physicians and Surgeons , 50 Haven Ave, Box #123, Bard Hall, New York, NY 10032 , USA.
Introduction:
Retinitis pigmentosa (RP) encompasses many different hereditary retinal degenerations that are caused by a vast array of different gene mutations and have highly variable disease presentations and severities. This heterogeneity poses a significant therapeutic challenge, although an answer may eventually be found through two recent innovations: induced pluripotent stem cells (iPSCs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas genome editing.
Areas Covered:
This review discusses the wide-ranging applications of iPSCs and CRISPR-including disease modelling, diagnostics and therapeutics - with an ultimate view towards understanding how these two technologies can come together to address disease heterogeneity and orphan genes in a novel personalized medicine platform. An extensive literature search was conducted in PubMed and Google Scholar, with a particular focus on high-impact research published within the last 1 - 2 years and centered broadly on the subjects of retinal gene therapy, iPSC-derived outer retina cells, stem cell transplantation and CRISPR/Cas gene editing.
Expert Opinion:
For the retinal pigment epithelium, autologous transplantation of gene-corrected grafts derived from iPSCs may well be technically feasible in the near future. Photoreceptor transplantation faces more significant unresolved technical challenges but remains an achievable, if more distant, goal given the rapid pace of advancements in the field.
Insights
Induced pluripotent stem cells (iPSCs) and CRISPR/Cas gene editing offer new hope for treating retinitis pigmentosa (RP). These technologies may enable personalized medicine platforms to address the genetic diversity of RP.
Area of Science:
- Ophthalmology
- Genetics
- Regenerative Medicine
Background:
- Retinitis pigmentosa (RP) is a group of hereditary retinal diseases with diverse genetic causes and clinical presentations.
- This genetic heterogeneity presents a significant challenge for developing effective treatments.
Purpose of the Study:
- To review the applications of induced pluripotent stem cells (iPSCs) and CRISPR/Cas genome editing in addressing RP.
- To explore how these technologies can form the basis of a personalized medicine platform for retinal diseases.
Main Methods:
- Comprehensive literature search of PubMed and Google Scholar focusing on recent high-impact research.
- Review centered on retinal gene therapy, iPSC-derived retinal cells, stem cell transplantation, and CRISPR/Cas gene editing.
Main Results:
- Induced pluripotent stem cells (iPSCs) and CRISPR/Cas gene editing show promise for disease modeling, diagnostics, and therapeutics in RP.
- The combination of these technologies could lead to a personalized medicine approach for rare genetic eye diseases.
Conclusions:
- Autologous transplantation of iPSC-derived retinal pigment epithelium grafts is a near-future possibility.
- Photoreceptor transplantation remains a more distant but achievable goal, driven by rapid advancements in the field.
