Gene editing possible for inherited retinal diseases

By The Science Advisory Board staff writers

September 26, 2022 -- Precision genome editing agents can enable gene correction and disease rescue in inherited retinal diseases (IRDs), according to University of California, Irvine researchers.

These disorders affect approximately 1 in 3,000 individuals worldwide and reduce quality of life. IRDs are caused by gene mutations that are critical for the development and/or function of the retina or RPE, and more than 270 causative genes have been identified.

The researchers argue in their article that precision genome editing agents, including base editors and prime editors, have enabled precise gene correction and disease rescue in multiple preclinical models of genetic disorders (Proceedings of the National Academy of Sciences, September 19, 2022). Why not use them for IRDs?

The paper stresses there is hope that in vivo gene editing will be the future treatment paradigm for IRDs. The authors said there will be an increasing number of clinical trials for targeting IRDs and any mutation that causes them will be amenable to treatment with precision gene editing.

"Precision medicine for IRDs has a promising outlook, as basic science has consistently led to the development of therapeutic tools to target patient-specific genetic mutations," the authors wrote. "The results of initial clinical trials, that use in vivo gene editing to treat IRDs, will be essential for informing the design and translation of future precision genome editing therapies."

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