Retinitis Pigmentosa & Inherited Retinal Diseases and Regenerative Medicine Research

Last medically reviewed: October 7, 2026

⚠ Important — educational information only, not medical advice

EyeCyte provides educational and scientific information and does not provide medical advice, diagnosis, or treatment. Emerging regenerative and biologic technologies discussed on this website may be experimental, investigational, unapproved, or supported only by preclinical evidence. Patients should discuss medical decisions with appropriately licensed healthcare professionals.
Not an FDA-approved treatment unless explicitly stated. Except where a page names an FDA-approved product, the stem-cell, MSC, exosome, extracellular-vesicle, Wharton’s jelly and other biologic approaches described here are not FDA-approved for any eye condition. Research findings — especially laboratory and animal studies — are not proof that anything works or is safe in people.
Research-Use-Only (RUO): Research-Use-Only (RUO) materials referenced on EyeCyte are for laboratory research by qualified professionals only. They are not drugs, biologics or devices approved for human use, are not intended for administration to people, and are not intended to diagnose, treat, cure, mitigate or prevent any disease.
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What is retinitis pigmentosa & inherited retinal diseases?

Retinitis pigmentosa (RP) is a group of inherited retinal diseases in which photoreceptors gradually degenerate, typically starting with night vision and peripheral vision.

What causes retinitis pigmentosa & inherited retinal diseases?

Variants in many different genes can cause RP; the gene involved affects the pattern of inheritance and progression.

What structures or cells are affected?

Rod and cone photoreceptors and, secondarily, the retinal pigment epithelium.

How is retinitis pigmentosa & inherited retinal diseases currently treated or managed?

● Established / Approved

Diagnosis, genetic testing and counseling with an inherited-retinal-disease specialist.
Low-vision rehabilitation and orientation and mobility services.

Are these approaches FDA approved for retinitis pigmentosa & inherited retinal diseases?

● Established / Approved A gene therapy (voretigene neparvovec) is FDA-approved for a specific genetic form of inherited retinal disease caused by biallelic RPE65 variants. It is a gene therapy, not a stem cell therapy, and applies only to that genetic cause.^[1]

No stem cell, MSC, exosome or Wharton’s jelly product is FDA-approved for retinitis pigmentosa, to our knowledge as of our last review.^[4]

Why are researchers studying regenerative approaches?

Because photoreceptors are lost, researchers are investigating whether progenitor cells could support remaining photoreceptors or replace lost ones, and how to protect remaining cells.

Research is not treatment. The studies summarized below are investigational. They are presented for education only, do not show that any approach is safe or effective in patients, and are not a recommendation. Unless stated as FDA-approved, none of these approaches is approved for this condition. RUO materials are not for human use.

Stem Cell Research and Retinitis Pigmentosa & Inherited Retinal Diseases

● Laboratory / In Vitro EvidenceInduced Pluripotent Stem Cells (iPSCs)
iPSCs are used in the laboratory to model inherited retinal disease and to derive photoreceptor precursor cells for research.^[3]
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: iPSC photoreceptor retinitis pigmentosa

MSC Research and Retinitis Pigmentosa & Inherited Retinal Diseases

● Insufficient EvidenceMesenchymal Stromal/Stem Cells (MSCs)
Evidence for MSC procedures in RP is limited and inconsistent; MSC injections offered outside regulated trials carry risk.^[2]
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: mesenchymal stem cells retinitis pigmentosa

Extracellular Vesicle / Exosome Research and Retinitis Pigmentosa & Inherited Retinal Diseases

We have not identified research in this category for retinitis pigmentosa & inherited retinal diseases that meets our standards for inclusion.

Other Cell-Based Research

● Early Human ResearchRetinal Progenitor Cells
Clinical trials have investigated intravitreal retinal progenitor cells in retinitis pigmentosa. Published and registered results should be reviewed directly; outcomes have not established a treatment.
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: retinal progenitor cells retinitis pigmentosa clinical trial

What Has Been Demonstrated in Laboratory Research?

● Laboratory / In Vitro EvidenceInduced Pluripotent Stem Cells (iPSCs)
iPSCs are used in the laboratory to model inherited retinal disease and to derive photoreceptor precursor cells for research.^[3]
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: iPSC photoreceptor retinitis pigmentosa

What Has Been Demonstrated in Animal Research?

We have not identified research in this category for retinitis pigmentosa & inherited retinal diseases that meets our standards for inclusion.

What Human Research Exists?

● Early Human ResearchRetinal Progenitor Cells
Clinical trials have investigated intravitreal retinal progenitor cells in retinitis pigmentosa. Published and registered results should be reviewed directly; outcomes have not established a treatment.
EVIDENCE VERIFICATION REQUIRED. Specific studies have not yet been verified by our reviewers. Search the literature: PubMed search: retinal progenitor cells retinitis pigmentosa clinical trial

Are Clinical Trials Underway?

Trial status changes often. Search the official U.S. registry directly: Retinitis Pigmentosa + retinal progenitor, Retinitis Pigmentosa + stem cell. A registered trial is not an endorsement, and registration does not mean a treatment works.

What Has NOT Yet Been Proven

● Insufficient Evidence

That any cell therapy restores vision in retinitis pigmentosa.
Which genetic forms, if any, might benefit from cell-based approaches.

Potential Biological Mechanisms Researchers Are Investigating

● Mechanistic / Theoretical

Neuroprotection Cell Replacement

Risks and Scientific Unknowns

Unapproved cell and biologic procedures offered outside clinical trials have been associated with serious harm, including permanent vision loss.
Products sold as "stem cells" or "exosomes" vary widely in what they actually contain; composition, purity and potency are often not independently verified.
Results in cells and animals frequently do not carry over to people; many approaches that look promising in the laboratory never reach approval.

Questions to Ask a Qualified Eye Doctor

What type and stage of this condition do I have, and how is it likely to progress?
Which treatments are established for my situation, and what are their benefits and risks?
Are there clinical trials I might qualify for, and how would I verify a trial is legitimate?
If I have seen a clinic offering stem cell or exosome procedures for my eyes, what questions should I ask?
Which gene is involved in my condition, and does that affect which trials or therapies apply?

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Explore Retinitis Pigmentosa & Inherited Retinal Diseases

Research topics

Retinal Progenitor Cells Mesenchymal Stromal/Stem Cells (MSCs)Induced Pluripotent Stem Cells (iPSCs)

Mechanisms