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Article

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

26 Dec 2019

Abstract excerpt

Background & Aims Genome editing technology has immense therapeutic potential and is likely to rapidly supplant contemporary gene addition approaches. Key advantages include the capacity to directly repair mutant loci with resultant recovery of physiological gene expression and maintenance of durable therapeutic effects in replicating cells. In this study, we aimed to repair a disease-causing point mutation in...

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PMID: 32039406
DOI: 10.1016/j.jhepr.2019.100065

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