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OTC intron 4 variations mediate pathogenic splicing patterns caused by the c.386G>A mutation in humans and spfash mice, and govern susceptibility to RNA-based therapies

Crossref DOI link: https://doi.org/10.1186/S10020-021-00418-9

Published Online: 2021-12-14

Published Print: 2021-12

Update policy: https://doi.org/10.1007/SPRINGER_CROSSMARK_POLICY

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