Genetherapy for completely colorblind children partly restores cone function uclnews
). Their cone cells do not send signals to the brain, but many remain present, so researchers have been seeking to activate the dormant cells.
The study involved four young people with achromatopsia aged 10 to 15 years old, who were taking part in two trials led by Professor James Bainbridge at UCL and Moorfields Eye Hospital, sponsored by MeiraGTx-Janssen Pharmaceuticals. Each of the four children was treated with gene therapy in one eye, enabling doctors to compare the treatment's effectiveness with the untreated eye.
The researchers say they cannot confirm whether the treatment was ineffective in the other two study participants, or if there may have been treatment effects that were not picked up by the tests they used, or if effects are delayed.
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