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GENOME EDITING

Correcting tyrosinaemia via a point mutation

In an adult mouse model of tyrosinaemia, a base editor correcting an A-to-G splice-site mutation in the Fah gene restores the translation of the functional enzyme, promoting the repopulation of the liver with the corrected cells.

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Fig. 1: Adenine base editing of the point mutation causing tyrosinaemia.

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Correspondence to Kiran Musunuru.

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Competing interests

K. M. is a co-founder and advisor of Verve Therapeutics, and an advisor of Variant Bio.

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Musunuru, K. Correcting tyrosinaemia via a point mutation. Nat Biomed Eng 4, 14–15 (2020). https://doi.org/10.1038/s41551-019-0489-x

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