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Therapeutic gene correction for Lesch-Nyhan syndrome using CRISPR-mediated base and prime editing

14 Feb 2023

Abstract excerpt

). Patients with LNS show various clinical phenotypes, including hyperuricemia, gout, devastating behavioral abnormality, intellectual disability, and self-harm. Although uric acid overproduction can be modulated with the xanthine oxidase inhibitor allopurinol, there exists no treatment for behavioral and neurological manifestations of LNS. In the current study, CRISPR-mediated base editors (BEs) and prime...

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PMID: 36910714
DOI: 10.1016/j.omtn.2023.02.009

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