Experimental gene therapy shows promise for RP

A new gene therapy could preserve vision in people with retinitis pigmentosa and might effectively treat other degenerative nerve disorders, according to researchers from the United States, the United Kingdom, and Australia.

Reporting in the American Society of Gene & Cell Therapy’s Molecular Therapy, the researchers said they had shown for the first time that mice responded to visual stimulation after treatment with genes to produce ciliary neurotrophic factor (CNTF).

The therapy appeared to give life-long protection of photoreceptor cells from a genetic disorder in mice that closely resembles retinitis pigmentosa in humans.

“These findings provide insights into potential novel therapeutic avenues for diseases such as retinitis pigmentosa and amyotrophic lateral sclerosis [ALS], for which CNTF has been evaluated unsuccessfully in clinical trials,” the researchers wrote.

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The researchers were based at the University of Oxford; the University of Florida in Gainesville, Florida, USA; the University of Sheffield, Moorfields Eye Hospital & UCL NIHR Biomedical Research Centre for Ophthalmology, and the University of Western Australia in Perth, Australia.

Previous research showed that CNTF can protect against ganglion cell death in models of oxidative stress and experimental glaucoma as well as retinal diseases.

But these earlier studies noted suppression of physiological function in these photoreceptors as measured by electroretinography.

Additionally, CNTF proved ineffective and toxic when given in large systemic doses to people with ALS.

The researchers in the current study wanted to find other measures of vision loss, measure how long the protection might last, find ways of controlling the CNTF dosing and test the therapy in a model where degeneration had already begun.

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