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Gene therapy study offers hope to IRD sufferers

The visual function of mice suffering from inherited retinal disease (IRD) was restored in a US study hailed as a breakthrough.

Researchers from the University of California, Irvine (UCI), used new-generation CRISPR technology – known as base editing – to demonstrate a new therapeutic modality for a wide range of IRDs.

Co-author Krzysztof Palczewski, professor at the UCI’s Gavin Herbert Eye Institute, said: “We provide evidence of the clinical potential of base editors for the correction of mutations causing IRDs and for restoring visual function.

 

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