Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients.
By Gerald Schwank, Bonil Koo, Valentina Sasselli, J. Dekkers, Inha Heo, T. Demircan, Nobuo Sasaki, S. Boymans, Edwin Cuppen, C. K. van der Ent and 3 more. Cell Stem Cell, 2013
1,321 citations recorded by the source corpus.
Subfields: Molecular Biology, Pulmonary and Respiratory Medicine
View Paper: https://www.semanticscholar.org/paper/078bc29f51ed343862d13979ecd4dd724fa38c8c
How researchers see this paper
The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.
In Molecular Biology
- Feng Zhang
- Hans Clevers
- Haifeng Wang
- Marie La Russa
- Lei S. Qi
- P. Hsu
- Phillip A. Sharp
- Jennifer A. Doudna
In Pulmonary and Respiratory Medicine
Citations in the corpus
- 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”
- Delivering CRISPR: a review of the challenges and approaches (2018, background): “, 2014), correct the cystic fibrosis transmembrane conductor receptor locus in cultured intestinal stem cells of cystic fibrosis patients (Schwank et al., 2013), introduce modular ‘AND’ gate circuits based on CRISPR/Cas9 that detects bladder cancer cells, inhibits bladder cancer cell growth, induces”
- Organoids (2022, background): “Cystic fibrosis organoids have also been used to investigate gene therapy interventions and are able to rescue the effect of CFTR mutations in intestinal organoids using CRISPR–Cas9 (ref.(195)).”
- Modeling Development and Disease with Organoids. (2016, background): “The corrected allele was fully functional, as demonstrated in clonally expanded organoids (Schwank et al., 2013).”
- Identification of multipotent luminal progenitor cells in human prostate organoid cultures (2014, background): “Moreover, the organoids are easily manipulated with inhibitors, retroviruses and CRISPR/Cas9 (Schwank et al., 2013), offering a tool to study the impact of these manipulations on growth, invasive behavior, and drug sensitivity.”
- 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 .”
- 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].”
- Long-Term Culture of Genome-Stable Bipotent Stem Cells from Adult Human Liver (2015, positive): “Repair by homologous recombination using CRISPR/Cas9 technology is feasible in organoid cultures, as we recently demonstrated in colon stem cells of cystic fibrosis patients (Schwank et al., 2013).”