Scientists Successfully Apply Gene Therapy against Retinitis Pigmentosa
Oxford, UK (Scicasts) – 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 addition 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.
Retinitis pigmentosa (RP) affects 1 in 4000 people, with symptoms that typically appear between age 10 and 30. Night vision and peripheral vision go first, as the photoreceptors active in low light – the ‘rods’ – start to degenerate. Eventually the condition affects the ‘cones’ – the photoreceptors responsible for central, detailed, colour vision.
The current study looked at a mouse model of retinitis pigmentosa in which the mice lack rhodopsin – the main pigment in rod photoreceptors. At age 4 weeks – after rod degeneration was underway and before cones were affected – the mice were dosed with a virus modified to produce human ciliary neurotrophic factor (CNTF) protein in the retina……
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