Compared to gene therapy, stem cell treatment is more widely used for various types of gene mutations causing retinitis pigmentosa (RP).

The eyes are an immune-privileged organ, meaning that stem cells introduced into the eyes are not rejected. The mechanisms of stem cell treatment include:

1. Stem cells can replace damaged cells.
2. Stem cells secrete nutritional factors.
3. Stem cells control immune responses, stimulate optic nerve regeneration, and facilitate material exchange between stem cells and recipient cells.

The summary of stem cell clinical trials for retinal diseases is as follows:

1. Retinal precursor stem cells derived from embryonic tissue are used to treat RP via subretinal injection. After a 3-year follow-up, the treatment has shown high safety, with vision improvement lasting one year for middle-stage RP patients. However, industrialization is challenging due to the difficulty in obtaining a large amount of eligible embryonic stem cells.

2. Umbilical cord mesenchymal stem cells administered via intravenous infusion have restored vision function in late-stage RP patients for one or two years, but repeated treatments may be necessary.

3. Bone marrow stem cells have been explored for diabetic retinopathy.

4. Retinal pigment epithelial cells derived from embryonic stem cells are being investigated for age-related macular degeneration.

5. Animal studies have demonstrated the feasibility of delivering stem cells intranasally to treat retinal diseases like age-related macular degeneration (AMD), retinitis pigmentosa, diabetic retinopathy, and optic nerve damage. Intranasal delivery of mesenchymal stem cells has shown promise in bypassing the blood-brain barrier, allowing them to reach the retina more effectively. This non-invasive approach is believed to promote optic cell regeneration, reduce inflammation, and support retinal function. Researchers have observed migration of stem cells from the nasal cavity to the retina, where they can integrate and potentially replace damaged cells or provide trophic support. This method holds potential for advancing treatment options for RP patients in the near future.

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