ArticleNature communications2014
Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform.
Article in Nature communications, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 102 papers, 1 of them a synthesis that pooled it.
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Who cites it
102 citing papers in PubMed, 1 synthesis or guideline pooled it, 181 citations in OpenAlex.
- Genetic quality assurance and genetic monitoring of laboratory mice and rats: FELASA Working Group Report.Laboratory animals · 2020Guideline
- Overcoming the challenges of genome-editing essential genes.STAR protocols · 2026Review
- ARROW: Allele-Specific Recombined gRNA Design for Reduced Off-Target with Enhanced Specificity.Bioengineering (Basel, Switzerland) · 2025Article
- New hope and promise with CRISPR-Cas9 technology for the treatment of HIV.Functional & integrative genomics · 2025Review
- Development of a homeolog-specific gene editing system in an evolutionary model for the study of polyploidy in nature.Frontiers in genome editing · 2025Article
- Article
- Gene therapy for CNS disorders: modalities, delivery and translational challenges.Nature reviews. Neuroscience · 2024Review
- Rab6-Mediated Polarized Transport of Synaptic Vesicle Precursors Is Essential for the Establishment of Neuronal Polarity and Brain Formation.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2024Article
- CRISPR/Cas9-gene editing approaches in plant breeding.GM crops & food · 2023Review
- Cryopreservation of rat embryos at all developmental stages by small-volume vitrification procedure and rapid warming in cryotubes.Scientific reports · 2023Article
- Cyba and Nox2 mutant rats show different incidences of eosinophilia in the genetic background- and sex-dependent manner.Experimental animals · 2023Article
- Limitations of gene editing assessments in human preimplantation embryos.Nature communications · 2023Article
- Hunting for Genes Underlying Emotionality in the Laboratory Rat: Maps, Tools and Traps.Current neuropharmacology · 2023Review
- Article
- Brca1Cancer science · 2022Article
- Loss of the cleaved-protamine 2 domain leads to incomplete histone-to-protamine exchange and infertility in mice.PLoS genetics · 2022Article
- CRISPR/Cas therapeutic strategies for autosomal dominant disorders.The Journal of clinical investigation · 2022Review
- Homology-based repair induced by CRISPR-Cas nucleases in mammalian embryo genome editing.Protein & cell · 2022Review
- Recent Advances in the Production of Genome-Edited Rats.International journal of molecular sciences · 2022Review
- Article
42 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The bacterial CRISPR/Cas system has proven to be an efficient gene-targeting tool in various organisms. Here we employ CRISPR/Cas for accurate and efficient genome editing in rats. The synthetic chimeric guide RNAs (gRNAs) discriminate a single-nucleotide polymorphism (SNP) difference in rat embryonic fibroblasts, allowing allele-specific genome editing of the dominant phenotype in (F344 × DA)F1 hybrid embryos. Interestingly, the targeted allele, initially assessed by the allele-specific gRNA, is repaired by an interallelic gene conversion between homologous chromosomes. Using single-stranded oligodeoxynucleotides, we recover three recessive phenotypes: the albino phenotype by SNP exchange; the non-agouti phenotype by integration of a 19-bp DNA fragment; and the hooded phenotype by eliminating a 7,098-bp insertional DNA fragment, evolutionary-derived from an endogenous retrovirus. Successful in vivo application of the CRISPR/Cas system confirms its importance as a genetic engineering tool for creating animal models of human diseases and its potential use in gene therapy.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.