Gene therapy discovery gives hope for retinitis pigmentosa research

New collaborative research between scientists in the UK and the USA has found that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of retinitis pigmentosa.

The results, published in the journal Molecular Therapy, showed that the preserved cells were able to drive visually-guided behaviour, even in later stages of the condition and despite becoming less sensitive to light.

Led by Professor Robert MacLaren at the University of Oxford’s Nuffield Laboratory of Ophthalmology and funded in the UK primarily by Fight for Sight, the researchers believed the findings were significant because they opened up a new line of research to prevent nerve cell death in retinitis pigmentosa and age-related macular degeneration. They may also have a wider application to neurodegenerative disorders such as amyotrophic lateral sclerosis.