Gene therapy for RP trialled

A new treatment approach has preserved the vision of dogs with a similar condition to retinitis pigmentosa


Scientists have halted vision loss in dogs using gene therapy.

The study, which was published in Proceedings of the National Academy of Sciences, could lead to the development of a new treatment approach for patients with autosomal dominant retinitis pigmentosa.

Researchers from the University of Pennsylvania School of Veterinary Medicine tested the gene therapy approach in dogs with a rhodopsin mutation – a condition that is similar to retinitis pigmentosa.

Following treatment, the dogs maintained healthy, functional photoreceptors.

The research team hope to start pre-clinical safety trials over the next two years, before moving on to human trials.

Rather than regenerating photoreceptors, the new approach prevents photoreceptor loss.

For this reason, the treatment could be helpful in slowly progressing forms of autosomal dominant retinitis pigmentosa where the patient still has areas of the retina with living photoreceptor rod cells.