Gene therapy gives long-term protection to photoreceptor cells in a mouse model of retinitis pigmentosa
Fight for Sight results published in Molecular Therapy show that the cells could drive visually-guided behaviour, even in late stage disease.
A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder.
Results published in the journal Molecular Therapy demonstrate 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.
These findings are significant because they open 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 (ALS).
The research was 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, with additional support from the Wellcome Trust, the Health Foundation, the Medical Research Council, the Royal College of Surgeons of Edinburgh, the Oxford Stem Cell Institute and the NIHR Ophthalmology (Moorfields) and Oxford Biomedical Research Centres.