Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders.

Scientific reports - 23 Nov 2017

Christie Kathleen A, Courtney David G, DeDionisio Larry A, Shern Connie Chao, De Majumdar Shyamasree, Mairs Laura C, Nesbit M Andrew, Moore C B Tara

Abstract excerpt

CRISPR/Cas9 holds immense potential to treat a range of genetic disorders. Allele-specific gene disruption induced by non-homologous end-joining (NHEJ) DNA repair offers a potential treatment option for autosomal dominant disease. Here, we successfully delivered a plasmid encoding S. pyogenes Cas9 and sgRNA to the corneal epithelium by intrastromal injection and acheived long-term knockdown of a corneal...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Clustered Regularly Interspaced Short Palindromic Repeats
Share to Corneal Dystrophies, Hereditary

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to CRISPR-Cas Systems

Share to DNA End-Joining Repair

Share to Gene Editing

Share to Mice, Mutant Strains

Share to Streptococcus pyogenes

Source

Open in PubMed Open full text

Identifiers

PMID: 29170458
DOI: 10.1038/s41598-017-16279-4
PMCID: PMC5701044

Genes

PAM
TGFBI

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders.PMID 29170458Read ↗

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

Challenges of Gene Editing Therapies for Genodermatoses.Semantic proximity Read ↗

Allele-specific genome targeting in the development of precision medicine.Semantic proximity Read ↗

CRISPR/Cas therapeutic strategies for autosomal dominant disorders.Semantic proximity Read ↗

Predictable CRISPR/Cas9-Mediated COL7A1 Reframing for Dystrophic Epidermolysis Bullosa.Semantic proximity Read ↗

CRISPR Interference-Potential Application in Retinal Disease.Semantic proximity Read ↗

CRISPR Base Editing Correction of TGFBI Mutations in Autosomal Dominant Corneal Dystrophies.Semantic proximity Read ↗

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

Allele-specific CRISPR/Cas9 genome editing of the single-base P23H mutation for rhodopsin associated dominant retinitis pigmentosaSemantic proximity Read ↗

Precision genome editing in the eye.Semantic proximity Read ↗

Allele-Specific Inactivation of an Autosomal Dominant Epidermolysis Bullosa Simplex Mutation Using CRISPR-Cas9.Semantic proximity Read ↗

Efficacy and dynamics of self-targeting CRISPR/Cas constructs for gene editing in the retinaSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.