Researchers Set Sights on Retinitis Pigmentosa with New Gene Therapy

According to a report by Newswise, a new form of gene therapy has been developed which may slow or prevent retinitis pigmentosa. Researchers funded by the National Eye Institute (NEI) developed the new approach. So far it has been successful treating the disease in canine models. The developers hope it will soon be ready to do the same for people living with the degenerative eye disorder. Keep reading to learn more, or follow the original story here for more information.

What is Retinitis Pigmentosa?

Retinitis pigmentosa describes a rare group of genetic eye disorders. In cases of retinitis pigmentosa, the cells in the retina break down over time. Because the retina is light sensitive and partially responsible for sight, a decrease in vision is common as the condition worsens.

Initially loss of vision may be restricted to low-light or peripheral vision. Over time, retinitis pigmentosa may result in a form of tunnel vision. Click here for more information on retinitis pigmentosa.

A New Method

Researchers associated with the NEI project say they have shown proof-of-concept for a treatment. This treatment focuses on one of the most common form of retinitis pigmentosa. Using gene therapy techniques, the team hopes they will be able to slow or treat the symptoms of autosomal dominant retinitis pigmentosa.

Gene therapy allows researchers to insert material into specific cells. In the case of the retinitis pigmentosa therapy researchers use what’s known as shRNA. Special viruses, known as vectors, are engineered to deliver the material without causing disease.

One Paw in Front of the Other

Progress on the new therapy has been a challenge. Researchers describe the necessity of finding a perfect balance in how the treatment is delivered to patients.

In order to test their new treatment concept, researchers have been using a model disease found in dogs. In a similar manner to retinitis pigmentosa, dogs with this eye condition lose important photoreceptors in their eyes.

When treated with the proper balance of vectors, dogs maintained healthy vision. What’s most promising about this is that the vectors were already designed to deliver the human version of the same treatment. Therefore, the research being currently conducted should prove applicable in the next stage of testing.

The research team says it hopes to begin clinical trials within the next two years. Because the treatment focuses on preventing photoreceptor loss (as opposed to rebuilding damaged photoreceptors) the treatment may be especially useful in cases where retinitis pigmentosa develops slowly.

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