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Article

CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting.

Gene therapy - 1 Jan 2016

Courtney D G, Moore J E, Atkinson S D, Maurizi E, Allen E H A, Pedrioli D M L, McLean W H I, Nesbit M A, Moore C B T

Abstract excerpt

CRISPR/Cas9-based therapeutics hold the possibility for permanent treatment of genetic disease. The potency and specificity of this system has been used to target dominantly inherited conditions caused by heterozygous missense mutations through inclusion of the mutated base in the short-guide RNA (sgRNA) sequence. This research evaluates a novel approach for targeting heterozygous single-nucleotide polymorphisms...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Mice, Inbred C57BL

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Share to Mutation, Missense

Source

Open in PubMed Open full text

Identifiers

PMID: 26289666
DOI: 10.1038/gt.2015.82
PMCID: PMC4705418

Genes

KRT12
PAM

Provenance

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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