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Article

Peptide Nucleic Acid-Mediated Regulation of CRISPR-Cas9 Specificity.

Nucleic acid therapeutics - 1 Oct 2024

Carufe Kelly E W, Economos Nicholas G, Glazer Peter M

Abstract excerpt

Although CRISPR-Cas9 gene therapies have proven to be a powerful tool across many applications, improvements are necessary to increase the specificity of this technology. Cas9 cutting in off-target sites remains an issue that limits CRISPR's application in human-based therapies. Treatment of autosomal dominant diseases also remains a challenge when mutant alleles differ from the wild-type sequence by only one...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39037032
DOI: 10.1089/nat.2024.0007
PMCID: PMC11564683

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