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Article

Base editing: advances and therapeutic opportunities.

Nature reviews. Drug discovery - 1 Dec 2020

Porto Elizabeth M, Komor Alexis C, Slaymaker Ian M, Yeo Gene W

Abstract excerpt

Base editing - the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells - is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the...

Topics

CRISPR-Cas Systems
Disease
Gene Editing
Hearing Loss
Humans
Liver Diseases
Muscular Dystrophy, Duchenne
Neoplasms
Polymorphism, Single Nucleotide

Source

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 33077937
DOI: 10.1038/s41573-020-0084-6
PMCID: PMC7721651

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