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Article

Mutation-Independent Allele-Specific Editing by CRISPR-Cas9, a Novel Approach to Treat Autosomal Dominant Disease.

Molecular therapy : the journal of the American Society of Gene Therapy - 5 Aug 2020

Christie Kathleen A, Robertson Louise J, Conway Caroline, Blighe Kevin, DeDionisio Larry A, Chao-Shern Connie, Kowalczyk Amanda M, Marshall John, Turnbull Doug, Nesbit M Andrew, Moore C B Tara

Abstract excerpt

CRISPR-Cas9 provides a tool to treat autosomal dominant disease by non-homologous end joining (NHEJ) gene disruption of the mutant allele. In order to discriminate between wild-type and mutant alleles, Streptococcus pyogenes Cas9 (SpCas9) must be able to detect a single nucleotide change. Allele-specific editing can be achieved by using either a guide-specific approach, in which the missense mutation is found...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 32416058
DOI: 10.1016/j.ymthe.2020.05.002
PMCID: PMC7403340

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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