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The realization of CRISPR gene therapy

The inaugural CRISPR-based drug Casgevy has been approved by several medical agencies, with other CRISPR-based therapies currently in clinical trials. Although there are technological hurdles to overcome, chemical biology has a vital role in developing recent breakthroughs in base editing, prime editing and epigenetic editing into future treatments.

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Fig. 1: A non-exhaustive summary of ongoing clinical trials or preclinical therapies and their targeted organs, genes and systems.


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Y.M. is supported by a Cancer Research Institute Dr. Keith Landesman Memorial Fellowship (CRI5247). L.S.Q. acknowledges support by National Science Foundation, National Institutes of Health and the Chau Hoi Shuen Foundation Limited. This work is supported by National Human Genome Research Institute (R21HG013133) and NIH Director’s Pioneer Award (DP1NS137219). L.S.Q. is a Chan Zuckerberg Biohub – San Francisco Investigator.

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Correspondence to Lei S. Qi.

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L.S.Q. is a founder of Epic Bio and scientific advisor of Laboratory of Genomic Research and Kytopen Corp. Y.M. declares no competing interests.

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Ma, Y., Qi, L.S. The realization of CRISPR gene therapy. Nat Chem Biol 20, 791–795 (2024).

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