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22 - 12 - 2017

Cellular therapy for retinitis pigmentosa: humans next?

A cell therapy protocol for retinal diseases has been successfully tested in a rat model by French researchers. A clinical trial application will be filed.


The loss of pigment cells or photoreceptors in the retina is the source of serious visual deficiencies such as retinitis pigmentosa. A therapeutic strategy is to replace the defective cells with cells derived from embryonic cells or induced human pluripotent stem cells.


Clinical trials conducted by injecting suspensions of these cells into the eye have already shown that this strategy is tolerated by the human eye. The next step is to deposit a complete layer of these cells on the retina.


A team of researchers * has developed ways to differente of stem cells into pigment cells and deposit them on the human amniotic membrane. This tissue was surgically deposited on the retina of rats that had retinitis pigmentosa.



The researchers found that photoreceptor cell loss was halted and that the animals' visual acuity was improved, even compared to animals that had received injected suspension cells.


These results prove the value of this therapeutic approach. The research team will file a clinical trial application for approximately twelve patients, which could begin within a year. This will be the first cell therapy trial for vision disease in France.


* I-Stem (laboratory created by AFM-Telethon), University of Evry, Inserm, Institute of Vision