Title |
Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR–Cas platform
|
---|---|
Published in |
Nature Communications, June 2014
|
DOI | 10.1038/ncomms5240 |
Pubmed ID | |
Authors |
K. Yoshimi, T. Kaneko, B. Voigt, T. Mashimo |
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. |
X Demographics
As of 1 July 2024, you may notice a temporary increase in the numbers of X profiles with Unknown location. Click here to learn more.
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 13% |
Australia | 1 | 7% |
Germany | 1 | 7% |
Japan | 1 | 7% |
Unknown | 10 | 67% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 11 | 73% |
Scientists | 2 | 13% |
Science communicators (journalists, bloggers, editors) | 2 | 13% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 6 | 2% |
Japan | 3 | <1% |
Spain | 2 | <1% |
United Kingdom | 2 | <1% |
Netherlands | 1 | <1% |
Australia | 1 | <1% |
India | 1 | <1% |
Germany | 1 | <1% |
Unknown | 346 | 95% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 83 | 23% |
Researcher | 74 | 20% |
Student > Master | 38 | 10% |
Student > Bachelor | 37 | 10% |
Other | 17 | 5% |
Other | 61 | 17% |
Unknown | 53 | 15% |
Readers by discipline | Count | As % |
---|---|---|
Agricultural and Biological Sciences | 168 | 46% |
Biochemistry, Genetics and Molecular Biology | 81 | 22% |
Medicine and Dentistry | 25 | 7% |
Engineering | 8 | 2% |
Neuroscience | 7 | 2% |
Other | 24 | 7% |
Unknown | 50 | 14% |