Net Doctor today reports that people with an inherited sight disorder may benefit from gene therapy, US scientists have claimed.
Researchers at the University of Pennsylvania School of Medicine and the Children’s Hospital of Philadelphia believe the therapy may be particularly beneficial for young patients.
Results from a phase I trial show that the therapy – which is designed to treat a severe form of inherited retinal degeneration called Leber congenital amaurosis (LCA) – was tolerated by all patients and brought about an improvement in their vision.
Writing in the Lancet medical journal, the researchers revealed that all 12 patients ‘showed improvement in retinal function’, but that the response depended on the extent of an individual’s retinal degeneration, which is linked to age.
‘The visual recovery noted in the children confirms the hypothesis that efficacy will be improved if treatment is applied before retinal degeneration has progressed,’ they added.
Barbara McLaughlan, a spokeswoman for the charity RNIB, welcomed the progress and said that she hopes it will one day be possible to use gene therapy to treat age-related macular degeneration.