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Genetic diversity

Accounting for diversity in the design of CRISPR-based therapeutic genome editing

CRISPR cell and gene therapy have been designed largely with respect to a single reference human genome. A new study reveals how human genetic diversity could lead to off-target effects and presents a new tool to identify these risks.

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Fig. 1: Assaying human genetic diversity can advance the design of human therapeutics involving genome editing.


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K.S. has received funding from the US National Science Foundation, grant nos. EEC-1648035 and AWD-101645-G3/RJ375-G3, and the US National Institutes for Health, grant nos. R35GM119644, U01EY032333 and UH3NS111688.

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Correspondence to Krishanu Saha.

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K.S. receives sponsored research support from Spotlight Therapeutics and Synthego and is an advisor to Notch Therapeutics and Andson Biotech.

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Saha, K. Accounting for diversity in the design of CRISPR-based therapeutic genome editing. Nat Genet 55, 6–7 (2023).

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