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Base Editing and Prime Editing: Potential Therapeutic Options for Rare and Common Diseases.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy - 1 Jul 2023

Testa Lauren C, Musunuru Kiran

Abstract excerpt

Collectively, genetic disorders affect approximately 350 million individuals worldwide and are a major global health burden. Despite substantial progress in identification of new disease-causing genes, variants, and molecular etiologies, nearly all rare diseases have no targeted therapeutics that can address their underlying molecular causes. Base editing (BE) and prime editing (PE), two newly described...

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PMID: 37314680
DOI: 10.1007/s40259-023-00610-9

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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