Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
Schwank, Gerald, Koo, Bon-Kyoung, Sasselli, Valentina, Dekkers, Johanna F., Heo, Inha, Demircan, Turan, Sasaki, Nobuo, Boymans, Sander +5 more·2013·Cell Stem Cell
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- Unmet needs in cystic fibrosis: the next steps in improving outcomesN. West, P. Flume · 2018 · Expert Review of Respiratory Medicine
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- Disrupting the male germ line to find infertility and contraception targetsDenise R. Archambeault, Martin Matthew Matzuk · 2014 · Annales d Endocrinologie
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- Genome Engineering in Human CellsMinjung Song, Young‐Hoon Kim, Jin‐Soo Kim, Seokjoong Kim · 2014 · Methods in enzymology on CD-ROM/Methods in enzymology
- New Directions in Pulmonary Gene TherapyAmber Vu, Paul B. McCray · 2020 · Human Gene Therapy
- Does school breakfast benefit children's educational performance?Lia C. H. Fernald, Cornelius C Ani, Sally Grantham‐McGregor · 1997 · PubMed
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- In vivoandin vitrodisease modeling with CRISPR/Cas9Tomoko Kato, Shuji Takada · 2016 · Briefings in Functional Genomics
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- Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene TherapiesAmrutha Banda, Olivia Impomeni, Aparana Singh, Abdul Rasheed Baloch, Wenhui Hu, Dabbu Kumar Jaijyan · 2024 · Vaccines
- Correcting genetic defects with CRISPR–Cas9Isabel B. Lokody · 2013 · Nature Reviews Genetics
- Novel approaches: Tissue engineering and stem cells – In vitro modelling of the gutJorik M. van Rijn, Kerstin Schneeberger, Caroline L. Wiegerinck, Edward E. S. Nieuwenhuis, Sabine Middendorp · 2016 · Best Practice & Research Clinical Gastroenterology
- Stacking up CRISPR against RNA i for therapeutic gene inhibitionDirk Haussecker · 2016 · FEBS Journal
- Different in vivo and in vitro transformation of intestinal stem cells in mismatch repair deficiencyKerstin Keysselt, Tobias Kreutzmann, Karen Rother, Christiane Kerner, Knuth Krohn, Jens Przybilla, Peter Buske, Henry Loeffler‐Wirth, Markus Loeffler, Joerg Galle et al. · 2016 · Oncogene
- A stable but reversible integrated surrogate reporter for assaying CRISPR/Cas9-stimulated homology-directed repairYahong Wen, Grace Liao, Thomas Pritchard, Tingting Zhao, Jon P. Connelly, Shondra M. Pruett‐Miller, Valérie Blanc, Nicholas O. Davidson, Blair Madison · 2017 · Journal of Biological Chemistry
- Intestinal organoid as an in vitro model in studying host-microbial interactionsJun Sun · 2017 · Frontiers in Biology
- Roadmap for an early gene therapy for cystic fibrosis airway diseaseMarianne Sylvia Carlon, Dragana Vidović, Susan E. Birket · 2017 · Prenatal Diagnosis
- DMSO increases efficiency of genome editing at two non-coding lociGeorge Stratigopoulos, Maria Caterina De Rosa, Charles A. LeDuc, Rudolph L. Leibel, Claudia A. Doege · 2018 · PLoS ONE
- Distinct EpCAM-Positive Stem Cell Niches Are Engaged in Chronic and Neoplastic Liver DiseasesSamira Safarikia, Guido Carpino, Diletta Overi, Vincenzo Cardinale, Rosanna Venere, Antonio Franchitto, Paolo Onori, Domenico Alvaro, Eugenio Gaudio · 2020 · Frontiers in Medicine
- Functional Comparison between VP64-dCas9-VP64 and dCas9-VP192 CRISPR Activators in Human Embryonic Kidney CellsNasir Javaid, Thuong L. H. Pham, Sangdun Choi · 2021 · International Journal of Molecular Sciences
- Rewriting CFTR to cure cystic fibrosisGiulia Maule, Marjolein M. Ensinck, Mattijs Bulcaen, Marianne Sylvia Carlon · 2021 · Progress in molecular biology and translational science
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- Dental treatment for people with cystic fibrosisN. Harrington, Peter James Barry, Siobhan M Barry · 2016 · European Archives of Paediatric Dentistry
- Cryopreservation of OrganoidsOlena Yu. Rogulska, Jarmila Havelkova, Yuriy Petrenko · 2023 · Cryoletters
- Mitochondrial Diseases: Molecular Pathogenesis and Therapeutic AdvancesJialun Mei, Peng Ding, Chuan Gao, Jian Zhou, Z. Li, Changqing Zhang, Junjie Gao · 2025 · MedComm
- Organoids derived from digestive tract, liver, and pancreasAn Tao Xu, Jin Lu Tong, Zhi Hua Ran · 2015 · Journal of Digestive Diseases
- Genetic Engineering of Primary Mouse Intestinal Organoids Using Magnetic Nanoparticle Transduction Viral Vectors for Frozen SectioningLingling Xian, Lionel Chia, Dan Georgess, Li Luo, Shuai Shuai, Andrew J. Ewald, Linda M S Resar · 2019 · Journal of Visualized Experiments
- Stem Cell–Derived Respiratory Epithelial Cell Cultures as Human Disease ModelsJessica C. Orr, Robert E. Hynds · 2021 · American Journal of Respiratory Cell and Molecular Biology
- Advances in the Cystic Fibrosis Drug Development PipelineChristine Tanaka Esposito, Martin Kämper, Jessica Trentacoste, Susan Galvin, Halie Pfister, Janice Wang · 2023 · Life
- Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editingAmy J. Walker, Carina Graham, Miriam Greenwood, Maximillian N. J. Woodall, Ruhina Maeshima, Michelle O’Hara-Wright, David J. Sanz, Ileana Guerrini, Ahmad M. Aldossary, Christopher O’Callaghan et al. · 2023 · Molecular Therapy — Methods & Clinical Development
- A new era of targeting cystic fibrosis with non-viral delivery of genomic medicinesNamratha Turuvekere Vittala Murthy, Kseniya Yu. Vlasova, Jonas Renner, Antony Jozić, Gaurav Sahay · 2024 · Advanced Drug Delivery Reviews
- OrganoLabeler: A Quick and Accurate Annotation Tool for Organoid ImagesBurak Kahveci, Elifsu Polatlı, Yalın Baştanlar, Sinan Güven · 2024 · ACS Omega
- CRISPR-Cas9 Based Genome Engineering: Opportunities in Agri-Food-Nutrition and HealthcareSubin R. C. K. Rajendran, Yuan‐Yeu Yau, Dinesh Pandey, Anil Kumar V · 2015 · OMICS A Journal of Integrative Biology
- Developmental history and application of CRISPR in human diseasePuping Liang, Xiya Zhang, Yuxi Chen, Junjiu Huang · 2017 · The Journal of Gene Medicine
- Regulation of Genome Editing in Plant Biotechnology: European UnionBrigitte Voigt, Ansgar Münichsdorfer · 2019 · Regulation of Genome Editing in Plant Biotechnology
- Beyond Mendelian Genetics: Anticipatory Biomedical Ethics and Policy Implications for the Use of CRISPR Together with Gene Drive in HumansMichael W. Nestor, Richard L. Wilson · 2020 · Journal of Bioethical Inquiry
- CRISPR-Cas9 gene editing causes alternative splicing of the targeting mRNAQian Zhang, Yao Fu, Chitra Thakur, Zhuoyue Bi, Priya Wadgaonkar, Yiran Qiu, Liping Xu, M’Kya Rice, Wenxuan Zhang, Bandar Saeed Almutairy et al. · 2020 · Biochemical and Biophysical Research Communications
- Bioengineering methods for organoid systemsJad Saleh, Barbara Mercier, Wang Xi · 2021 · Biology of the Cell
- RETRACTED: Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal CellSadegh Shojaei Baghini, Zhanna R. Gardanova, Angelina Olegovna Zekiy, Navid Shomali, Foad Tosan, Mostafa Jarahian · 2021 · Frontiers in Bioengineering and Biotechnology · Retracted
- Trends in the global organoid technology and industry: from organogenesis in a dish to the commercialization of organoidsHanbyeol Lee, Jeong Suk Im, Da Bin Choi, Dong‐Hun Woo · 2021 · Organoid
- CFTR RNA- and DNA-based therapiesPatrick T. Harrison · 2022 · Current Opinion in Pharmacology
- A Cell-Based Optimised Approach for Rapid and Efficient Gene Editing of Human Pluripotent Stem CellsSara Cuevas Ocaña, Jin Ye Yang, Magomet Aushev, George Schlossmacher, Christine E. Bear, Nicholas R. F. Hannan, Neil D. Perkins, Janet Rossant, Amy P. Wong, Michael A. Gray · 2023 · International Journal of Molecular Sciences
- 3D intestinal organoids in metabolic research: virtual reality in a dishAnastasia Tsakmaki, Patricia Fonseca Pedro, Gavin A. Bewick · 2017 · Current Opinion in Pharmacology
- Cellular Therapy for the Treatment of Paediatric Respiratory DiseaseLaura C. Brennan, Andrew O’Sullivan, Ronan J. MacLoughlin · 2021 · International Journal of Molecular Sciences
- Organoids: A new in vitro model system for biomedical science and disease modelling and promising source for cell-based transplantationBon‐Kyoung Koo, Meritxell Huch · 2016 · Developmental Biology
- Deterministic evolution and stringent selection during pre-neoplasiaKasper Karlsson, Moritz Jakob Przybilla, Eran Kotler, Aziz Khan, Hang Xu, Kremena Karagyozova, Alexandra Sockell, Wing Hung Wong, Katherine Tin Heng Liu, Amanda T. Mah et al. · 2022 · bioRxiv (Cold Spring Harbor Laboratory)
- Human Embryos, Induced Pluripotent Stem Cells, and Organoids: Models to Assess the Effects of Environmental Plastic PollutionDragana Miloradović, D. Pavlović, Marina Gazdic, Sandra Nikolić, Miloš Papić, Nevena N. Milivojević, Miodrag Stojković, Biljana Ljujić · 2021 · Frontiers in Cell and Developmental Biology
- Overcoming the barriers of two-dimensional cell culture systems with three-dimensional cell culture systems: techniques, drug discovery, and biomedical applicationsAnkita Borah, D. Sakthi Kumar · 2022 · Elsevier eBooks
- The Effect of Complex Alleles of the CFTR Gene on the Clinical Manifestations of Cystic Fibrosis and the Effectiveness of Targeted TherapyMaria Krasnova, Anna Sergeevna Efremova, Artem Bukhonin, Elena Kestutisovna Zhekaite, Tatiana Borisovna Bukharova, Yuliya L. Melyanovskaya, Dmitry Vadimovich Goldshtein, E. Kondratyeva · 2023 · International Journal of Molecular Sciences
- Live Organoid Cyclic ImagingDavid E. Reynolds, Yusha Y. Sun, Xin Wang, Phoebe Vallapureddy, Jianhua Lim, Menghan Pan, Andres Fernandez del Castillo, Jonathan Carlson, Mark A. Sellmyer, MacLean P. Nasrallah et al. · 2024 · Advanced Science
- Bioengineering toolkits for potentiating organoid therapeuticsSewon Park, Seung‐Woo Cho · 2024 · Advanced Drug Delivery Reviews
- Engineering the Future of Regenerative Medicines in Gut Health with Stem Cell-Derived Intestinal OrganoidsDinesh Kumar, Sonia Gupta, Vrinda Gupta, Rajni Tanwar, Anchal Chandel · 2025 · Stem Cell Reviews and Reports