Article
Precision genome editing in the eye.
Proceedings of the National Academy of Sciences of the United States of America - 27 Sept 2022
Suh Susie, Choi Elliot H, Raguram Aditya, Liu David R, Palczewski Krzysztof
Abstract excerpt
CRISPR-Cas-based genome editing technologies could, in principle, be used to treat a wide variety of inherited diseases, including genetic disorders of vision. Programmable CRISPR-Cas nucleases are effective tools for gene disruption, but they are poorly suited for precisely correcting pathogenic mutations in most therapeutic settings. Recently developed precision genome editing agents, including base editors and...
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