Correction of a genetic disease in mouse via use of CRISPR-Cas9.

By Yuxuan Wu, Dan Liang, Yinghua Wang, M. Bai, Wei-Yao Tang, Shi-min Bao, Zhiqiang Yan, Dangsheng Li, Jinsong Li. Cell Stem Cell, 2013

616 citations recorded by the source corpus.

Subfields: Aging, Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/062438dfceaa150fd8fae3df4f7854bd9c5f75eb

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Aging

1. Eric S. Lander
2. Phillip A. Sharp

In Genetics

1. Jennifer A. Doudna
2. Lei S. Qi
3. Haifeng Wang
4. Marie La Russa
5. P. Hsu
6. Feng Zhang
7. Eric S. Lander
8. E. Charpentier

In Molecular Biology

1. Jennifer A. Doudna
2. Feng Zhang
3. Lei S. Qi
4. Haifeng Wang
5. Marie La Russa
6. P. Hsu
7. Eric S. Lander
8. E. Charpentier

Citations in the corpus

• The new frontier of genome engineering with CRISPR-Cas9 (2014, positive): “A dominant mutation in the Crygc gene responsible for cataracts was successfully corrected in mice (103).”
• sgRNAcas9: A Software Package for Designing CRISPR sgRNA and Evaluating Potential Off-Target Cleavage Sites (2014, background): “An interesting report shows that the CRISPRCas9 system can be used successfully to correct a genetic disease in mice with cataracts [23].”
• Delivering CRISPR: a review of the challenges and approaches (2018, background): “, 2014), correction of a cataract-causing mutation in mice (Wu et al., 2013), and correction of a Duchenne muscular dystrophy (DMD)-causing mutation in mice (Long et al.”
• A Robust CRISPR/Cas9 System for Convenient, High-Efficiency Multiplex Genome Editing in Monocot and Dicot Plants. (2015, background): “A previous report showed that CRISPR/Cas9-induced DSB in a mutated gene in mice could be corrected to wild-type through the HR mechanism, also termed homology-directed repair (HDR), with the endogenous wild-type allele as the template (Wu et al., 2013).”
• Target specificity of the CRISPR-Cas9 system (2014, positive): “With exogenous template oligos, specific sequences such as HA-tag or GFP could be inserted into genes to label proteins [19,20], or to correct mutations in disease genes in human and mouse [21–23].”
• CRISPR/Cas9 in Genome Editing and Beyond. (2016, background): “A partial list includes the Fah gene in hereditary tyrosinemia (125), Dystrophin in Duchenne’s muscular dystrophy (126, 133), Crygc in cataracts (127, 128), CTFR in cystic fibrosis (129), HBB in β-thalassemia”
• Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells (2014, positive): “The simplicity of targeting any locus with a single protein and a programmable sgRNA has quickly led to widespread use of Cas9 11,12 in applications such as genome editing 7,8,13–16 , disease gene repair 17,18 and knock-in of specific tags 8,19 .”
• Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery. (2019, neutral): “147 – 149 These approaches reduce the risk of off-target genetic perturbations, as only the pre-selected and correctly edited cells can be implanted into patients.”