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Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
Open accessAbstract
Single murine and human intestinal stem cells can be expanded in culture over long time periods as genetically and phenotypically stable epithelial organoids. Increased cAMP levels induce rapid swelling of such organoids by opening the cystic fibrosis transmembrane conductor receptor (CFTR). This response is lost in organoids derived from cystic fibrosis (CF) patients. Here we use the CRISPR/Cas9 genome editing system to correct the CFTR locus by homologous recombination in cultured intestinal stem cells of CF patients. The corrected allele is expressed and fully functional as measured in clonally expanded organoids. This study provides proof of concept for gene correction by homologous recombination in primary adult stem cells derived from patients with a single-gene hereditary defect.
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In the citing paper
“The CRISPR-nuclease SpCas9 was harnessed to correct the ΔF508 mutation by homology-directed repair (HDR)22.”
From Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing (2019) · cites this paper as [22]
Experimental — accuracy not yet measuredThe source backs part of the claim, not all of it — or backs it for a different population, condition or comparison.
In the citing paper
“providing the first step to advance gene therapy in cystic fibrosis patients with CRISPR.”
From CRISPR-Cas: Converting A Bacterial Defence Mechanism into A State-of-the-Art Genetic Manipulation Tool (2019) · cites this paper as [167]
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“They demonstrated that once the mutation had been corrected, the function of the CF transmembrane conductor receptor (CFTR) was restored.4”
From What is CRISPR/Cas9? (2016) · cites this paper as [4]
The cited source says what the claim attributes to it.
In the citing paper
“Using adult intestinal stem cells obtained from two patients with CF, they successfully corrected the most common mutation causing CF in intestinal organoids.”
From What is CRISPR/Cas9? (2016) · cites this paper as [4]
The cited source says what the claim attributes to it.
In the citing paper
“Using CRISPR, researchers were able to correct mutant intestinal stem cells from cystic fibrosis patients and restore their function in vitro [167]”
From CRISPR-Cas: Converting A Bacterial Defence Mechanism into A State-of-the-Art Genetic Manipulation Tool (2019) · cites this paper as [167]
The cited source says what the claim attributes to it.
In the citing paper
“In 2013, researchers culture intestinal stem cells from two cystic fibrosis patients and corrected the mutation at the CFTR locus resulting in the expression of the correct gene and full function of the protein.”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [33]
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In the citing paper
“For instance, organoids derived from intestinal epithelial cells of pediatric IBD patients had IBD-specific DNA methylation signatures despite retaining similar morphologies195.”
From Organoids (2022) · cites this paper as [195]
Experimental — accuracy not yet measuredIn the citing paper
“Several groups have also demonstrated in vitro correction of genetic disease genes including CFTR (cystic fibrosis)9, Crygc (cataracts)10 and DMD (Duchenne muscular dystrophy)11 in organoids or mouse zygotes.”
From Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo (2016) · cites this paper as [9]
In the citing paper
“correct the cystic fibrosis transmembrane conductor receptor locus in cultured intestinal stem cells of cystic fibrosis patients (Schwank et al., 2013)”
From Delivering CRISPR: a review of the challenges and approaches (2018) · cites this paper as Schwank et al., 2013
In the citing paper
“CRISPR-Cas9 genome editing has been applied to correct disease-causing mutations in mouse zygotes and human cell lines for cataract5 and cystic fibrosis6”
From Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype (2014) · cites this paper as [6]
In the citing paper
“In human intestinal stem cells collected from patients with cystic fibrosis, the culprit gene cystic fibrosis transmembrane conductance regulator was rectified by homologous recombination during CRISPR-Cas9 genome editing while the pluripotency was retained as demonstrated by formations of organ-like expansions in cell culture.33”
From CRISPR-Cas9: a new and promising player in gene therapy (2015) · cites this paper as [33]
In the citing paper
“Since then, the potential utility of the application of CRISPR/Cas-9 for cystic fibrosis was established.33”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [33]
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Citations
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CRISPR-Induced TMPRSS2-ERG Gene Fusions in Mouse Prostate Organoids.
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InfluentialPulmonary endogenous progenitor stem cell subpopulation: Physiology, pathogenesis, and progress
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“[71] and CF patient-derived induced pluripotent cells (iPS) in”
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Duchenne muscular dystrophy: genome editing gives new hope for treatment
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Public health applications of CRISPR: How children's health can benefit
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Convergence of human pluripotent stem cell, organoid, and genome editing technologies
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CRISPR-Cas9 based genome editing for defective gene correction in humans and other mammals
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A protocol for the identification and validation of novel genetic causes of kidney disease
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“The application of CRISPR-Cas9 technology [98–100] to introduce or correct mutations in such cellular or organoid disease models [101–103] may also be required and employed.”
MethodGenome Editing Redefines Precision Medicine in the Cardiovascular Field
Dzilic, E., Lahm, H., Dreßen, M., Deutsch, M.-A., Lange, R., Wu, S. M., Krane, M., & Doppler, S. A. (2018) · Stem Cells International · doi:10.1155/2018/4136473 · Cited by 10
“showed in their study targeting the cystic fibrosis transmembrane conductor receptor by CRISPR/Cas9 in human iPSCs off-target effects of predicted locations between 0 and 4% [65].”
BackgroundEmerging pharmacotherapies in cystic fibrosis
McElvaney, O. J., Gunaratnam, C., McElvaney, O. F., Bagwe, I., Reeves, E. P., & McElvaney, N. G. (2018) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2018.1512409 · Cited by 9
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BackgroundResearch Article CRISPR/Cas Class 2 systems and their applications in biotechnological processes
Mota, D. S., Marques, J., Guimarães, J. M., & Mariúba, L. A. M. (2020) · Genetics and Molecular Research · doi:10.4238/gmr18478 · Cited by 9
Unlocking the potential of CRISPR-Cas9 for cystic fibrosis: A systematic literature review
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Harnessing induced pluripotent stem cells and organoids for disease modeling and precision medicine
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“…CFTR gene, patient-derived intestinal organoids enable quantitative functional assays of CFTR activity and allow the testing of modulator drugs such as Ivacaftor, Lumacaftor, and Trikafta, which restore chloride-channel conductance and show strong correlation with clinical response metrics [25].”
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CRISPR/Cas9 in cancer: An attempt to the present trends and future prospects
Kaligotla, V. S. A., Jasti, T., & Kandra, P. (2021) · Biotechnology and Applied Biochemistry · doi:10.1002/bab.2200 · Cited by 8
Cosenza, L. C., Zuccato, C., Zurlo, M., Gambari, R., & Finotti, A. (2022) · Genes · doi:10.3390/genes13101727 · Cited by 8
“For instance, CRISPR-Cas9 has been employed in cystic fibrosis [64,65], sickle-cell disease [66,67], Huntington’s chorea [68], Duchenne muscular dystrophy [69,70], hemophilia [71,72], and chronic granulomatous disease [73].”
BackgroundMethodThe Promises and Pitfalls of CRISPR-Mediated Base Editing in Stem Cells
Wong, P. K., Zamberi, N. N. M., Syafruddin, S. E., Cheah, F., Azmi, N., Law, J. X., & Chua, E. W. (2023) · The CRISPR Journal · doi:10.1089/crispr.2023.0013 · Cited by 8
Potential Application of Intestinal Organoids in Intestinal Diseases
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“Studies have shown that CRISPR-Cas9 system was used to modify the intestinal organoid genome to correct the cystic fibrosis transmembrane conductance regulator (CFTR) locus and repair its function [67].”
Gene editing for corneal disease management
Raikwar, S. P., Raikwar, A. S., Chaurasia, S. S., & Mohan, R. R. (2016) · World Journal of Translational Medicine · doi:10.5528/wjtm.v5.i1.1 · Cited by 8
CRISPR Editing in Biological and Biomedical Investigation
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“In 2013, one group repaired a mutation in the cystic fibrosis transmembrane conductor receptor (CFTR) locus via HDR in primary intestinal stem cells derived from cystic fibrosis patients that had a single-gene hereditary defect [Schwank et al., 2013].”
BackgroundFrontiers of CRISPR-Cas9 for Cancer Research and Therapy
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Retinal organoid differentiation methods determine organoid cellular composition
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“In a seminal study using CRISPR/Cas9 in organoids, the Clevers group demonstrated that inactivation of the APC-locus by CRISPR/Cas9-mediated introduction of frameshift mutations in both murine and human intestinal organoids causes proliferation independently from the Wnt-agonist R-Spondin1, which is essential for the…”
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Epithelial stem cell culture: modeling human disease and applications for regenerative medicine
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“Moreover, when the mutated CFTR locus was corrected using the CRISPR/Cas9 technology in intestinal organoids of CF patients, the corrected genes were shown to functionally work [48].”
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“testinal stem cells.(81) At least 1 institution has begun clinical trials integrating patient whole-exome sequencing with high-throughput tumor organoid drug screening to identify the ideal drug combinations for individual patients.”
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Recent Research Trends in Stem Cells Using CRISPR/Cas-Based Genome Editing Methods
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“Schwank et al. (113) corrected ISC organoids derived from cystic fibrosis (CF) patients induced by the F508del mutation in CFTR to normal by a CRISPR system-mediated HDR technology.”
BackgroundTranslational Utility of Organoid Models for Biomedical Research on Gastrointestinal Diseases
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“Organoid are mainly developed from malignant gastrointestinal tissues such as colorectal cancer, esophageal cancer, pancreatic cancer and rectal cancer and few other associated diseases including cystic fibrosis [143–145].”
Insights into Modeling Inflammatory Bowel Disease from Stem Cell Derived Intestinal Organoids
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AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys
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“Until now, the CRISR-Cas9 technology has been applied successfully to correct mutations in the DYSTROPHIN gene (responsible for the Duchenne muscular dystrophy) and the cystic fibrosis transmembrane conductance regulator [125,126].”
MethodEngineering the Niche for Intestinal Regeneration
Hernandez‐Gordillo, V., Koppes, A. N., Griffith, L. G., Breault, D. T., & Carrier, R. L. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-802734-9.00037-8 · Cited by 7
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“To investigate the possibility of gene correction in adult stem cells using CRISPR-Cas9, the focus was on cystic fibrosis (CF), where adult intestinal stem cells were isolated and expanded in culture, then the mutant F508del allele was corrected using the CRISPR-Cas9 and the functionality of the targeted allele was de…”
MethodPalmer, D. J., Grove, N. C., Turner, D. L., & Ng, P. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.001 · Cited by 7
“Schwank et al.(18) obtained 11 to 31 puro colonies, of which 9.”
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“CRISPR-associated protein 9 (Cas9) nuclease guided by short single guide RNAs (sgRNAs) that recognize the target DNA can generate double-strand breaks (DSBs) at specific DNA target sites.(20) Although the CRISPR/Cas9 system is effective, off-target cleavage of Cas9 has been observed.”
BackgroundFajac, I., & Sermet‐Gaudelus, I. (2021) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0000000000000827 · Cited by 7
“The first proof-of-concept study was published in 2013 and showed repair of the F508del mutation by gene editing in intestinal organoids [39].”
BackgroundRegenerative medicine for childhood gastrointestinal diseases
Jones, B. C., Shibuya, S., Durkin, N., & Coppi, P. D. (2021) · Best Practice & Research Clinical Gastroenterology · doi:10.1016/j.bpg.2021.101769 · Cited by 7
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“Proof-of-concept has already been established [9].”
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From hacking the human genome to editing organs
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“Another group used CRISPR to correct the cystic fibrosis transmembrane conductor receptor (CFTR) locus in stem cells from patients with cystic fibrosis.(29) The disease phenotype was corrected when these stem cells were expended in stable FIGURE 1.”
MethodMedical case reports in the age of genomic medicine
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Precise treatment of cystic fibrosis – current treatments and perspectives for using CRISPR
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“[8], CRISPR-Cas9 was used to correct F508”
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Katrekar, D., Hu, M. Y., & Mali, P. (2017) · Current Opinion in Biomedical Engineering · doi:10.1016/j.cobme.2017.04.001 · Cited by 7
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Scaffolds for intestinal tissue engineering
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Genome Engineering for Therapeutic Applications
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Changing the Paradigm – Treating the Basic Defect in Cystic Fibrosis
Guglani, L. (2015) · The Indian Journal of Pediatrics · doi:10.1007/s12098-015-1786-3 · Cited by 7
“[45] recently provided the proof of concept for correction of CFTR gene using the cas9/CRISPR approach in human intestinal stem cells.”
BackgroundAhsan, M. R., Alam, H. S. K., Hasan, A. R., Islam, M. Z., & Hossain, A. Z. (2017) · Bangladesh Journal of Child Health · doi:10.3329/bjch.v40i3.33060 · Cited by 7
Crispr2vec: Machine Learning Model Predicts Off-Target Cuts of CRISPR systemsPreprint
Trivedi, T. B., Boger, R. S., Kamath, G. M., Evangelopoulos, G., Cate, J. H. D., Doudna, J. A., & Hidary, J. D. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.10.28.359885 · Cited by 7
“The CRISPR-Cas system’s ability to target specific sequences in DNA and RNA in organisms has led to its widespread use, with applications of CRISPR-Cas enzymes now including diagnostics [5, 25], therapeutics [50, 43, 38], agriculture [20], cell models [14, 7], and more.”
BackgroundXu, J., Gong, W., Mo, C., Hou, X., & Ou, M. (2024) · Stem Cell Reviews and Reports · doi:10.1007/s12015-024-10799-z · Cited by 6
“…step towards the future application of stem cell gene therapy for the treat-ment of monogenic diseases. treating diseases like cystic fibrosis [ 34, 35] and hereditary leukemia [ 36–38 ], offering novel therapeutic options and hope to patients afflicted with these genetic conditions. ability…”
Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing
Carrozzo, I., Maule, G., Gentile, C., Umbach, A., Ciciani, M., Guidone, D., Santis, M. D., Petris, G., Galietta, L. J. V., Arosio, D., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.01.011 · Cited by 6
Chalkley, A. S., Lopez, M. T., Mysior, M. M., Brink, M. C., Kelly, S., & Simpson, J. C. (2025) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2025.1606573 · Cited by 6
“This technique has seen limited use thus far in 3D systems, although there are a few examples of successful applications (Haase-Kohn et al., 2022; Matano et al., 2015; Schwank et al., 2013).”
Organoid-on-a-chip (OrgOC): Advancing cystic fibrosis research
Zheng, M., Erice, E., Wang, H., Zhang, L., & Lawrie, C. H. (2025) · Materials Today Bio · doi:10.1016/j.mtbio.2025.102148 · Cited by 8
Chakraborty, C., Bhattacharya, M., Das, A., Agoramoorthy, G., & Lee, S.-S. (2026) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2026.102859 · Cited by 8
“47 This technology highlighting its application in different human diseases ranging from cancer to cardiovascular disorders (CVDs) to eye disease (Figure 2).”
Molecular mechanisms and biotechnology applications of CRISPR–Cas12a
Saha, A., Ocampo, R. F., Wright, J. T., Taylor, D. W., & Palermo, G. (2026) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-026-00969-x · Cited by 11
A high-MAPK, low-WNT cell state drives metastatic dissemination in colorectal cancer
Heinlein, M., Li, G. X., Kljavin, N. M., Moore, A. R., Biehs, B., Tao, L., Boumahdi, S., Mohammadi, F., Shi, M., Liang, Y., et al. (2026) · Nature Cancer · doi:10.1038/s43018-026-01155-w · Cited by 7
“Wild-type mouse organoids were derived from isolated crypts of the small intestine of female C57BL/6N mice and maintained as previously described 48,49 .”
Multi-faceted CRISPR/Cas technological innovation aspects in the framework of 3P medicine
Lučanský, V., Holubeková, V., Kolková, Z., Halášová, E., Samec, M., & Golubnitschaja, O. (2023) · The EPMA Journal · doi:10.1007/s13167-023-00324-6 · Cited by 6
Current progress in CRISPR-Cas systems for rare diseases
Israr, J., & Kumar, A. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2024.07.019 · Cited by 7
Harnessing CRISPR technology for next-generation microphysiological systems
Song, S., Kang, H. W., Kang, M., Chung, S. H., Choi, N., Kim, H. N., & Sung, Y. J. (2025) · TrAC Trends in Analytical Chemistry · doi:10.1016/j.trac.2025.118164 · Cited by 6
Bachtarzi, H. (2017) · Regenerative Medicine · doi:10.2217/rme-2017-0095 · Cited by 6
Personalized Medicine for the Infertile Male
Velez, D., & Hwang, K. (2020) · Urologic Clinics of North America · doi:10.1016/j.ucl.2020.07.003 · Cited by 6
Barhouse, P. S., Andrade, M. J., & Smith, Q. (2022) · Frontiers in Chemical Engineering · doi:10.3389/fceng.2022.832754 · Cited by 6
“Recent innovations have led to the formation of self-organizing embryo-like structures from pluripotent stem cells that can provide insights on embryonic development leading to organogenesis (Shahbazi et al., 2019; Simunovic et al., 2019; Hyun et al., 2020).”
BackgroundBenston, S. (2022) · Journal of Bioethical Inquiry · doi:10.1007/s11673-022-10186-8 · Cited by 6
“CRISPR and other gene-editing technologies in theory could be used to eradicate inherited monogenic diseases, such as cystic fibrosis (Schwank et al. 2013), Huntington’s Disease (Armitage 2015), and sickle cell anaemia (Elizondo et al. 2015); “improve” or edit genes underlying monogenic non-disease…”
BackgroundCocktail Formula and Application Prospects for Oral and Maxillofacial Organoids
Ou, M., Li, Q., Ling, X., Yao, J., & Mo, X. (2022) · Tissue Engineering and Regenerative Medicine · doi:10.1007/s13770-022-00455-3 · Cited by 6
Emerging medicines to improve the basic defect in cystic fibrosis
Fajac, I., & Sermet‐Gaudelus, I. (2022) · Expert Opinion on Emerging Drugs · doi:10.1080/14728214.2022.2092612 · Cited by 6
“The first proof-of-concept study was published in 2013 and showed repair of the F508del mutation by gene editing in intestinal organoids [43].”
BackgroundSuwito, B. E., Adji, A. S., Wardani, V. A. K., Widjaja, J. S., Angel, S. C. S., & Rahman, F. S. (2022) · Bali Medical Journal · doi:10.15562/bmj.v11i2.3414 · Cited by 6
Unlocking the Future: Pluripotent Stem Cell-Based Lung Repair
Goecke, T., Ius, F., Ruhparwar, A., & Martin, U. (2024) · Cells · doi:10.3390/cells13070635 · Cited by 6
“Moreover, the heterogeneity of CF mutations calls for personalized curative approaches aiming at repair or replacement of airway basal cells carrying the underlying genetic defect [28,32].”
Valetdinova, K. R., Medvedev, S. P., & Zakian, S. M. (2015) · Acta Naturae · doi:10.32607/20758251-2015-7-1-19-36 · Cited by 6
Aligning patients' needs and research priorities towards a comprehensive CF research program
Noordhoek, J. J., Gulmans, V. A. M., Heijerman, H. G. M., & van der Ent, C. K. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.03.008 · Cited by 6
Buddington, R. K., Wong, T., & Howard, S. C. (2021) · Medical Sciences · doi:10.3390/medsci9010009 · Cited by 6
“The gradual swelling of organoids composed of differentiated intestinal cells in response to CFTR activation [37] provided additional evidence for chloride-mediated fluid secretion.”
BackgroundLung development, repair and cancer: A study on the role of MMP20 gene in adenocarcinoma
Mok, P. L., Anandasayanam, A. N. K., David, H. M. O., Tong, J., Farhana, A., Khan, M. S. A., Sivaprakasam, G., Koh, A. E.-H., & Alzahrani, B. (2021) · PLoS ONE · doi:10.1371/journal.pone.0250552 · Cited by 6
“Other than that, it has also been used to make modifications in somatic cells including stem and germline cells for correcting various diseases such as pulmonary diseases [33], gastrointestinal disorders [34, 35], haematological diseases [36–38] viral diseases [39–41], vector diseases [42, 43], autoimmune and inflamma…”
BackgroundNetwork Topology of Biological Aging and Geroscience-Guided Approaches to COVID-19
LANDAY, A. L., Bartley, J. M., Banerjee, D., Hargis, G. R., Haynes, L., Keshavarzian, A., Kuo, C., Kwon, O. S., Li, S., Li, S., et al. (2021) · Frontiers in Aging · doi:10.3389/fragi.2021.695218 · Cited by 6
Organoids as a Model for Intestinal Ion Transport Physiology
de Jonge, H. R., Bijvelds, M. J. C., Strubberg, A. M., Liu, J., & Clarke, L. L. (2020) · Physiology in health and disease · doi:10.1007/978-3-030-55310-4_1 · Cited by 7
Sato, M., Saitoh, I., & Inada, E. (2016) · Stem Cell Investigation · doi:10.21037/sci.2016.11.05 · Cited by 6
“In fact, successful gene correction has been reported in iPSCs derived from patients with cystic fibrosis and β-thalassemia (6-10).”
BackgroundAdvances in applications of the CRISPR/Cas9 system for respiratory diseases
Ajaykumar, C., Rajkumar, S., Suresh, B., Birappa, G., Gowda, D. A. A., Jayachandran, A., Kim, K.-S., Hong, S.-H., & Ramakrishna, S. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2024.07.014 · Cited by 5
Kumar, A. V., Garg, V. K., & Buttar, H. S. (2025) · Elsevier eBooks · doi:10.1016/b978-0-443-22300-6.00044-9 · Cited by 10
Cho, J., Lee, M. J., Park, J. Y., Lee, J., Lee, S., Chung, C., Koo, B., & Park, Y. (2025) · Journal of Visualized Experiments · doi:10.3791/68529 · Cited by 5
Self-citationCRISPR-Cas technologies: Emerging tools from research to clinical application
Hyeon, H., Hwang, S., Luo, Y., Shin, E., Yeom, J., Kim, H., Ryu, M., & Lee, K. (2025) · The Journal of Microbiology · doi:10.71150/jm.2504012 · Cited by 7
“For gene editing, CRISPR-Cas9 systems have been successfully delivered both in vitro and in vivo using the commonly employed Lipofect-amine transfection (Schuh et al., 2018; Schwank et al., 2013; Yu et al., 2016).”
Evolution of prime editing systems: move forward to the treatment of hereditary diseases
Volodina, O. V., Fabrichnikova, A. R., Anuchina, A. A., Mishina, O. S., Lavrov, A. V., & Smirnikhina, S. A. (2025) · Current Gene Therapy · doi:10.2174/0115665232295117240405070809 · Cited by 1
Metadata
- Authors
- Schwank, G., Koo, B.-K., Sasselli, V., Dekkers, J. F., Heo, I., Demircan, T., Sasaki, N., Boymans, S., Cuppen, E., van der Ent, C. K., Nieuwenhuis, E. E. S., Beekman, J. M., & Clevers, H.
- Affiliations
- University Medical Center Utrecht (NL)
- Wilhelmina Children's Hospital (NL)
- Hubrecht Institute for Developmental Biology and Stem Cell Research (NL)
- Corresponding author
- Clevers, Hans
- ORCID
- Venue
- Cell Stem Cell
- Publisher
- Elsevier BV
- Published
- 2013-12
- Volume
- 13
- Issue
- 6
- Pages
- 653-658
- ISSN
- 1875-9777 (ISSN-L)
- 1934-5909
- OpenAlex
- W1986316308
- Cited by
- 1,424
- References
- 45
- Citations by year
- 2026: 64 · 2025: 74 · 2024: 92 · 2023: 78 · 2022: 105 · 2021: 152 · 2020: 137 · 2019: 120 · 2018: 118 · 2017: 148 · 2016: 150 · 2015: 110 · 2014: 72 · 2013: 3
- FWCI
- 50.06
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Cell stem cell (publishedVersion)
- PubMed (publishedVersion)
- KNAW research portal (Royal Academy of Art and Sciences (KNAW)) (publishedVersion)
- Data Archiving and Networked Services (DANS) (submittedVersion)
- Funders
- Wellcome Trust (079249, 097922/C/11/Z)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- cystic fibrosis, CFTR, CRISPR/Cas9 genome editing, homologous recombination, gene correction, cAMP signaling, adult stem cells
- MeSH terms
- Adult, Animals, Base Sequence, Cystic Fibrosis, Humans, Intestines, Molecular Sequence Data, Organoids, Stem Cells, Genetic Therapy, RNA Editing, Cystic Fibrosis Transmembrane Conductance Regulator, Mice, Adult Stem Cells, Clustered Regularly Interspaced Short Palindromic Repeats, CRISPR-Associated Proteins
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