Therapeutic gene correction for Lesch-Nyhan syndrome using CRISPR-mediated base and prime editing
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference34 articles.
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3. Diagnosis and treatment of the lesch-nyhan syndrome;Crawhall;Pediatr. Res.,1972
4. Molecular, biochemical, and genetic characterization of a female patient with Lesch-Nyhan disease;Rinat;Mol. Genet. Metabol.,2006
5. Implications of human genetic variation in CRISPR-based therapeutic genome editing;Scott;Nat. Med.,2017
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