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HomeminewsGene Therapy for RP Across Species

Gene Therapy for RP Across Species

Researchers from the US have found a gene therapy for retinitis pigmentosa (RP) that has now succeeded in different animal models.

The findings are significant because they show that the therapy works in different species, which is crucial to advancing it to human clinical trials, said Professor Alfred Lewin from the University of Florida (UF).

RP is the name given to a diverse group of inherited eye disorders that cause permanent changes to vision, often beginning in childhood or early adulthood.

Working in mice, the researchers used gene therapy to both knock down and replace malfunctioning genetic material that affects light-sensing photoreceptors in the retina. The therapy led to improved retinal structure and function throughout the nine month experiment, UF said in a news release.

The research was published in the journal Vision Research.1 “The objective was to confirm that this therapy, which was tested previously in other animals, works in a different species with a different mutation. We also wanted to show that the therapy could have benefit after retinal degeneration has already started,” Prof Lewin said.

In both cases, the answer was definitive, and Prof Lewin said the findings give researchers reason to believe the therapy could work long term for humans.

1. Ahmed C.M., et al., Binocular benefit following monocular subretinal AAV injection in a mouse model of autosomal dominant retinitis pigmentosa (adRP). Vision Res. 2023 Feb 9;206:108189.doi:10.1016/j.visres.2023.108189. Epub ahead of print. PMID: 36773475.