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Article

Therapeutic Genome Editing and In Vivo Delivery.

The AAPS journal - 2 Jun 2021

Ramirez-Phillips Amanda Catalina, Liu Dexi

Abstract excerpt

Improvements in the understanding of human genetics and its roles in disease development and prevention have led to an increased interest in therapeutic genome editing via the use of engineered nucleases. Various approaches have been explored in the past focusing on the development of an effective and safe system for sequence-specific editing. Compared to earlier nucleases such as zinc finger nuclease and...

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PMID: 34080099
DOI: 10.1208/s12248-021-00613-w

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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