British scientists are hoping to make gene therapy work for patients with a rare eye disease – but say only some will benefit.
A new project sets out to identify which patients will benefit from the therapy, developed for Leber’s congenital amaurosis.
The therapy, aimed at the gene AIPL1, has been shown to work in mice.
But doctors say it is not guaranteed to work in all human patients.
This is because the gene has a many natural variations in the human population and most of these do not cause disease, they say.
The therapy aims to “rescue” working photoreceptors in the affected patients. Doctors say this may be possible for some patients.
The project at the University College London Institute of Ophthalmology and Moorfields Eye Hospital, London, aims to test whether genetic changes found in these patients are responsible for disease.
Dr Dolores Conroy, director of research at Fight for Sight, said: “Knowing which Leber’s congenital amaurosis patients can benefit from targeted AIPL1 gene-therapy is a vital step before clinical trials can take place.
“The results will also lead to a much better understanding of which clinical signs and symptoms are associated with different AIPL1 variants, giving us a much fuller picture of Leber’s congenital amaurosis.”
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