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

Cited by (1,001)

Items 501–600 of 1,001

  1. 3D organoids derived from the small intestine: An emerging tool for drug transport research
    Yuanjin Zhang, Shengbo Huang, Weiguo Zhong, Wenxia Chen, Bingyi Yao, Xin Wang · 2020 · Acta Pharmaceutica Sinica B
  2. Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies
    Katelin M. Allan, Nigel Farrow, Martin Donnelley, Adam Jaffé, Shafagh A. Waters · 2021 · Frontiers in Pharmacology
  3. In Silico Predictive Modeling of CRISPR/Cas9 guide efficiency
    Nicolò Fusi, Ian C. P. Smith, John G. Doench, Jennifer Listgarten · 2015 · bioRxiv (Cold Spring Harbor Laboratory)
  4. Retinal Organoids and Retinal Prostheses: An Overview
    Alessandro Bellapianta, Ana Cetkovic, Matthias Bolz, Ahmad Salti · 2022 · International Journal of Molecular Sciences
  5. CRISPR/Cas9 system: recent applications in immuno-oncology and cancer immunotherapy
    Chen Chen, Zehua Wang, Yanru Qin · 2023 · Experimental Hematology and Oncology
  6. Recent progress in organoid culture to model intestinal epithelial barrier functions
    Tetsuya Nakamura · 2018 · International Immunology
  7. Importance of Organoids for Personalized Medicine
    Lukas Perkhofer, Pierre‐Olivier Frappart, Martin Müller, Alexander Kleger · 2018 · Personalized Medicine
  8. Modeling genetic epilepsies in a dish
    Wei Niu, Jack M. Parent · 2019 · Developmental Dynamics
  9. Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies
    Claire M. Drysdale, Tina Nassehi, Jackson Gamer, Morgan Yapundich, John F. Tisdale, Naoya Uchida · 2021 · Cell stem cell
  10. CRISPR-Cas Technology as a Revolutionary Genome Editing tool: Mechanisms and Biomedical Applications
    Sahar Ebrahimi, Mohammad Ali Khosravi, Abbasali Raz, Morteza Karimipoor, Parviz Parvizi · 2023 · ̒Ulūm va funūn-i bastah/bandī
  11. Applications of CRISPR-Cas9 mediated genome engineering
    Xiao Ping Yang · 2015 · Military Medical Research
  12. 2D- and 3D-Based Intestinal Stem Cell Cultures for Personalized Medicine
    Liu, Yuan, Chen, Ye-Guang · 2018 · Cells
  13. In Vitro Validation of a CRISPR-Mediated CFTR Correction Strategy for Preclinical Translation in Pigs
    Zhichang Peter Zhou, Liang Yang, Huibi Cao, Ziyan Rachel Chen, Yiqian Zhang, Xiao‐Yan Wen, Jim Hu · 2019 · Human Gene Therapy
  14. Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator
    Olga Villamizar, Shafagh A. Waters, Tristan A. Scott, Sheena M. Saayman, Nicole Grepo, Ryan Z. Urak, Alicia M. Davis, Adam Jaffé, Kevin V. Morris · 2019 · Molecular Therapy
  15. Gene manipulation in liver ductal organoids by optimized recombinant adeno-associated virus vectors
    Jinsong Wei, Gai Ran, Xin Wang, Ning Jiang, Jianqing Liang, Xinhua Lin, Chen Ling, Bing Tian Zhao · 2019 · Journal of Biological Chemistry
  16. Hepatocellular Carcinoma
    Rodrigo Carlessi, Julia Köhn‐Gaone, John Kevin Olynyk, Janina E. E. Tirnitz‐Parker · 2019 · Codon Publications eBooks
  17. Tiny tots for a big-league in wound repair: Tools for tissue regeneration by nanotechniques of today
    R. I. Jari Litany, Prabhakaran Kala Praseetha · 2022 · Journal of Controlled Release
  18. Intestinal organoids and organoids extracellular vesicles for inflammatory bowel disease treatment
    Han Liu, Jinru Sun, Mingkai Wang, Sicheng Wang, Jiacan Su, Can Xu · 2023 · Chemical Engineering Journal
  19. Combining nanotechnology with the multifunctional roles of neutrophils against cancer and inflammatory disease
    Lu Tang, Shun He, Yue Yin, Jing Li, Qiaqia Xiao, Ruotong Wang, Lijun Gao, Wei Wang · 2022 · Nanoscale
  20. Identifying the E2F3-MEX3A-KLF4 signaling axis that sustains cancer cells in undifferentiated and proliferative state
    Xu Yang, Guilin Li, Yuhua Tian, Xin Wang, Jiuzhi Xu, Ruiqi Liu, Min Deng, Chunlei Shao, Yuwei Pan, Xi Wu et al. · 2022 · Theranostics
  21. Functional Consequences of CFTR Interactions in Cystic Fibrosis
    Yashaswini Ramananda, Anjaparavanda P. Naren, Kavisha Arora · 2024 · International Journal of Molecular Sciences
  22. Insights into next-generation immunotherapy designs and tools: molecular mechanisms and therapeutic prospects
    Hongzhuo Qin, Zhaokai Zhou, Run Shi, Yumiao Mai, Yudi Xu, Peng Fu, Guangyang Cheng, Zhenfa Zhang, Wenjie Chen, Yun Chen et al. · 2025 · Journal of Hematology & Oncology
  23. High-Throughput Silencing Using the CRISPR-Cas9 System: A Review of the Benefits and Challenges
    Mark R. Wade · 2015 · SLAS DISCOVERY
  24. CRISPR/Cas9 system as an innovative genetic engineering tool: Enhancements in sequence specificity and delivery methods
    Young-Il Jo, Bharathi Suresh, Seokjoong Kim, Suresh Ramakrishna · 2015 · Biochimica et Biophysica Acta (BBA) - Reviews on Cancer
  25. On the Road (to a Cure?) — Stem-Cell Tourism and Lessons for Gene Editing
    R. Alta Charo · 2016 · New England Journal of Medicine
  26. Concise Review: The Potential Use of Intestinal Stem Cells to Treat Patients with Intestinal Failure
    Sung Noh Hong, James Dunn, Matthias G. Stelzner, Martı́n G. Martı́n · 2016 · Stem Cells Translational Medicine
  27. Organoids Provide an Important Window on Inflammation in Cancer
    Kristi Baker · 2018 · Cancers
  28. Three-Dimensional Organoids in Cancer Research: The Search for the Holy Grail of Preclinical Cancer Modeling
    Kevin Dzobo, Arielle Rowe, Dimakatso Alice Senthebane, Mousa AlMazyadi, Victoria A. Patten, Mohamed Iqbal Parker · 2018 · OMICS A Journal of Integrative Biology
  29. 3D-printing enabled micro-assembly of a microfluidic electroporation system for 3D tissue engineering
    Qingfu Zhu, Megan Hamilton, Bryan Vasquez, Mei He · 2019 · Lab on a Chip
  30. Organoids: Past Learning and Future Directions
    Lauren O’Connell, Des C. Winter · 2019 · Stem Cells and Development
  31. CRISPR/Cas9 gene editing therapies for cystic fibrosis
    Carina Graham, Stephen L. Hart · 2021 · Expert Opinion on Biological Therapy
  32. Genetically modified cell sheets in regenerative medicine and tissue engineering
    Zhiwei Jiang, Na Li, Danji Zhu, Lingfei Ren, Qin Shao, Yu Ke, Guoli Yang · 2021 · Biomaterials
  33. Three‐dimensional pancreas organogenesis models
    Anne Grapin‐Botton · 2016 · Diabetes Obesity and Metabolism
  34. Individualized medicine using intestinal responses to CFTR potentiators and correctors
    Jeffrey Matthijn Beekman · 2016 · Pediatric Pulmonology
  35. Optimization of ionizable lipids for aerosolizable mRNA lipid nanoparticles
    Mae M. Lewis, Melissa R. Soto, Esther Y. Maier, Steven Wulfe, Sandy Bakheet, Hannah Obregon, Debadyuti Ghosh · 2023 · Bioengineering & Translational Medicine
  36. Human Inducible Pluripotent Stem Cells and Autism Spectrum Disorder: Emerging Technologies
    Michael W. Nestor, André W. Phillips, Elena Artimovich, Jonathan E. Nestor, John P. Hussman, Gene J. Blatt · 2015 · Autism Research
  37. Gene editing at CRISPR speed
    Monya Baker · 2014 · Nature Biotechnology
  38. Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes
    Ganesan Govindan, Sivaprakash Ramalingam · 2016 · Journal of Cellular Physiology
  39. Site-Directed Genome Knockout in Chicken Cell Line and Embryos Can Use CRISPR/Cas Gene Editing Technology
    Qisheng Zuo, Yinjie Wang, Shaoze Cheng, Chao Lian, Beibei Tang, Fei Wang, Zhenyu Lu, Yanqing Ji, Ruifeng Zhao, Wenhui Zhang et al. · 2016 · G3 Genes Genomes Genetics
  40. CRISPR Genome Engineering for Human Pluripotent Stem Cell Research
    Somali Chaterji, Eun Hyun Ahn, Deok‐Ho Kim · 2017 · Theranostics
  41. CRISPR-Cas9 Mediated Genome Editing in Bicyclus anynana Butterflies
    Tirtha Das Banerjee, Antónia Monteiro · 2018 · Methods and Protocols
  42. Description of CRISPR/Cas9 development and its prospect in hepatocellular carcinoma treatment
    Xiaoling Wu, Weijie Ma, Chengjie Mei, Xi Chen, Ye Yao, Yingyi Liu, Xian Qin, Yufeng Yuan · 2020 · Journal of Experimental & Clinical Cancer Research
  43. Pituitary Remodeling Throughout Life: Are Resident Stem Cells Involved?
    Emma Laporte, Annelies Vennekens, Hugo E J Vankelecom · 2021 · Frontiers in Endocrinology
  44. Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review
    Rosa María Girón Moreno, Marta María García Clemente, Layla Diab-Cáceres, Adrián Martínez-Vergara, Miguel Ángel Martínez‐García, Rosa Mar Gómez-Punter · 2021 · Antibiotics
  45. Enhancing organoid technology with carbon-based nanomaterial biosensors: Advancements, challenges, and future directions
    Zahra Rezaei, Niyou Wang, Yipei Yang, Kannan Govindaraj, Jose Joaquin Velasco, Alvaro Dario Martinez Blanco, Nam Ho Bae, HeaYeon Lee, Su Ryon Shin · 2025 · Advanced Drug Delivery Reviews
  46. Ethical issues in genetic modification and why application matters
    Megan Munsie, Christopher Gyngell · 2018 · Current Opinion in Genetics & Development
  47. Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis
    Elena K. Schneider‐Futschik · 2019 · Gene Therapy
  48. Primary Intestinal Epithelial Organoid Culture
    Tomohiro Mizutani, Hans C. Clevers · 2020 · Methods in molecular biology
  49. Inducible Pluripotent Stem Cells as a Potential Cure for Diabetes
    K. Verhoeff, Sarah Henschke, B. Marfil-Garza, Nidheesh Dadheech, A. M. Shapiro · 2021 · Cells
  50. Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor
    Simone Amistadi, Giulia Maule, Matteo Ciciani, Marjolein M. Ensinck, Liesbeth De Keersmaecker, Anabela Santo Ramalho, Daniela Guidone, Martina Buccirossi, Luis J. V. Galietta, Marianne Sylvia Carlon et al. · 2023 · Molecular Therapy
  51. Update on advances in cystic fibrosis towards a cure and implications for primary care clinicians
    Vito Terlizzi, Philip M. Farrell · 2024 · Current problems in pediatric and adolescent health care
  52. Advancements in CRISPR/Cas systems for disease treatment
    Yangsong Xu, Hao Le, Qinjie Wu, Ning Wang, Changyang Gong · 2025 · Acta Pharmaceutica Sinica B
  53. Gene delivery to the lungs: pulmonary gene therapy for cystic fibrosis
    Ilia Villate-Beitia, Jon Zárate Sesma, Gustavo Puras, José Luís Pedraz · 2017 · Drug Development and Industrial Pharmacy
  54. Development of Arrayed Colonic Organoids for Screening of Secretagogues Associated with Enterotoxins
    Dulan B. Gunasekara, Matthew DiSalvo, Yuli Wang, Daniel L. Nguyen, Mark I. Reed, Jennifer E. Speer, Christopher E. Sims, Scott T. Magness, Nancy L. Allbritton · 2017 · Analytical Chemistry
  55. Organoids for Advanced Therapeutics and Disease Models
    Suran Kim, Ann‐Na Cho, Sungjin Min, Sooyeon Kim, Seung‐Woo Cho · 2018 · Advanced Therapeutics
  56. A new era of gene editing for the treatment of human diseases
    Mandip Kc, Clifford J. Steer · 2019 · Swiss Medical Weekly
  57. Integration of organoids in peptide drug discovery: Rise of the high‐throughput screening
    Siqi Zhang, Jieting Shen, Xingkai Wang, Xiaona Sun, Yuxuan Wu, Ming‐Rong Zhang, Rui Wang, Kuan Hu · 2023 · Open Research (University of Surrey)
  58. CRISPR-Cas9: A Revolutionary Tool for Cancer Modelling
    Raúl Torres-Ruiz, Sandra Rodríguez · 2015 · International Journal of Molecular Sciences
  59. Genome Editing for Cystic Fibrosis
    Guoshun Wang · 2023 · Cells
  60. Recapitulating the adenoma–carcinoma sequence by selection of four spontaneous oncogenic mutations in mismatch-repair-deficient human colon organoids
    Tomohiro Mizutani, Matteo Boretto, Sangho Lim, Jarno Drost, Diego Montiel González, Rurika Oka, Maarten H. Geurts, Harry Begthel, Jeroen Korving, Johan H. van Es et al. · 2024 · Nature Cancer
  61. Human stem cell-based disease modeling: prospects and challenges
    Joshua Z Johnson, Dirk F. Hockemeyer · 2015 · Current Opinion in Cell Biology
  62. The third dimension: new developments in cell culture models for colorectal research
    Joana Cabrita Pereira, Nikhil T Awatade, Cláudia Almeida Loureiro, Paulo Matos, Margarida Duarte Amaral, Peter Jordan · 2016 · Cellular and Molecular Life Sciences
  63. Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene
    Jennifer A. Hollywood, Ciaran M. Lee, Martina F. Scallan, Patrick T. Harrison · 2016 · Scientific Reports
  64. Modeling Host-Virus Interactions in Viral Infectious Diseases Using Stem-Cell-Derived Systems and CRISPR/Cas9 Technology
    Jihoon Kim, Bon‐Kyoung Koo, Ki‐Jun Yoon · 2019 · Viruses
  65. Design Approaches for Generating Organ Constructs
    Yun Fei Xia, Juan Carlos Izpisúa Belmonte · 2019 · Cell stem cell
  66. Intracellular Antibody Delivery Mediated by Lipids, Polymers, and Inorganic Nanomaterials for Therapeutic Applications
    Yamin Li, Peixuan Li, Raissa Li, Qiaobing Xu · 2020 · Advanced Therapeutics
  67. Automation of Organoid Cultures: Current Protocols and Applications
    Alexandra Louey, Damián Hernández, Alice Pébay, Maciej Daniszewski · 2021 · SLAS DISCOVERY
  68. The Off-Targets of Clustered Regularly Interspaced Short Palindromic Repeats Gene Editing
    Manuel M. Vicente, Miguel Chaves‐Ferreira, João M. P. Jorge, João T. Proença, Vasco M. Barreto · 2021 · Frontiers in Cell and Developmental Biology
  69. Application of CRISPR-Cas9 based gene editing to study the pathogenesis of colon and liver cancer using organoids
    Gayatri Ramakrishna, Elizabeth Babu, Ravinder Singh, Nirupma Trehanpati · 2021 · Hepatology International
  70. CRISPR-Cas Technology: Emerging Applications in Clinical Microbiology and Infectious Diseases
    Sahar Serajian, Ehsan Ahmadpour, Sonia M. Rodrigues Oliveira, Maria de Lourdes Pereira, Siamak Heidarzadeh · 2021 · Pharmaceuticals
  71. Priming of the Cells: Hypoxic Preconditioning for Stem Cell Therapy.
    Zheng Zachory Wei, Yanbing Zhu, James Y. Zhang, Myles Randolph McCrary, Song Wang, Yongbo Zhang, Shanping Yu, Ling Wei · 2017 · PubMed Central
  72. From Definitive Endoderm to Gut—a Process of Growth and Maturation
    Jordi Guiu, Kim Bak Jensen · 2015 · Stem Cells and Development
  73. Engineered CRISPR Systems for Next Generation Gene Therapies
    Michael Pineda, Farzaneh Moghadam, Mo R. Ebrahimkhani, Samira Kiani · 2017 · ACS Synthetic Biology
  74. Gene Therapy in Rare Respiratory Diseases: What Have We Learned So Far?
    Lucía Bañuls, Daniel Pellicer, Silvia Castillo, María Mercedes Navarro‐García, María Magallón, Cruz González, Francisco Dası́ · 2020 · Journal of Clinical Medicine
  75. Drug Screening, Oral Bioavailability and Regulatory Aspects: A Need for Human Organoids
    Tamara Zietek, Wolfgang Boomgaarden, Eva Rath · 2021 · Pharmaceutics
  76. Progress in Respiratory Gene Therapy
    G. McLachlan, E. Alton, A. Boyd, Nora K. Clarke, J. Davies, D. Gill, U. Griesenbach, J. Hickmott, S. Hyde, K. Miah et al. · 2022 · Human Gene Therapy
  77. CRISPR, un système qui permet de corriger ou de modifier l’expression de gènes responsables de maladies héréditaires
    Jacques P. Tremblay · 2015 · médecine/sciences
  78. CRISPR/Cas9-Mediated Genome Editing of Mouse Small Intestinal Organoids
    Gerald Schwank, Hans C. Clevers · 2016 · Methods in molecular biology
  79. CRISPR/Cas9—Advancing Orthopoxvirus Genome Editing for Vaccine and Vector Development
    Arinze Stanley Okoli, Malachy Ifeanyi Okeke, Morten Tryland, Ugo Lionel Moens · 2018 · Viruses
  80. Recent advances in nanocomposite-based delivery systems for targeted CRISPR/Cas delivery and therapeutic genetic manipulation
    Muhammad Waseem Ghani, Ambreen Iqbal, Hammad Ghani, Sidra Bibi, Zixun Wang, Renjun Pei · 2023 · Journal of Materials Chemistry B
  81. Engineered biomaterials in stem cell-based regenerative medicine
    Fei Zhu, Guangjun Nie, Changsheng Liu · 2023 · Life Medicine
  82. Repairing organs: lessons from intestine and liver
    Helmuth Gehart, Hans C. Clevers · 2015 · Trends in Genetics
  83. The Rise of CRISPR/Cas for Genome Editing in Stem Cells
    Bing Shui, Liz Hernández-Matías, Yi Guo, Ying Peng · 2016 · Stem Cells International
  84. Organoids as preclinical models to improve intraperitoneal chemotherapy effectiveness for colorectal cancer patients with peritoneal metastases: Preclinical models to improve HIPEC
    Pétronille Roy, Charlotte Canet-Jourdan, Maxime Annereau, Olivier Zajac, Maximiliano Gelli, Sophie Broutin, Lionel Mercier, Angélo Paci, François Lemare, Michel Pierre Ducreux et al. · 2017 · International Journal of Pharmaceutics
  85. Stem cell models as an in vitro model for predictive toxicology
    S. Lynch, Chris S. Pridgeon, Carrie A. Duckworth, Parveen Sharma, B. Park, C. Goldring · 2019 · Biochemical Journal
  86. Research Progress on Nanoparticles-Based CRISPR/Cas9 System for Targeted Therapy of Tumors
    Dengyun Nie, Ting Guo, Miao Yue, Wenya Li, Xinyu Zong, Yinxing Zhu, Junxing Huang, Mei Lin · 2022 · Biomolecules
  87. Loss of SUV420H2-Dependent Chromatin Compaction Drives Right-Sided Colon Cancer Progression
    Verawan Boonsanay, Mohammed H. Mosa, Mario Looso, Dieter Weichenhan, Fatih Ceteci, Lorenz Pudelko, André Lechel, Christian Sebastian Michel, Carsten Künne, Henner F. Farin et al. · 2022 · Gastroenterology
  88. Exploring the potential of human intestinal organoids: Applications, challenges, and future directions
    Inês A. Parente, Linda Chiara, Simona Bertoni · 2024 · Life Sciences
  89. ZEB1-mediated fibroblast polarization controls inflammation and sensitivity to immunotherapy in colorectal cancer
    Constantin Menche, Harald Schuhwerk, Isabell Armstark, Pooja Gupta, Kathrin Fuchs, Ruthger van Roey, Mohammed H. Mosa, Anne Hartebrodt, Yussuf Hajjaj, Ana Clavel Ezquerra et al. · 2024 · EMBO Reports
  90. Transfecting tissue models with CRISPR/Cas9 plasmid DNA using peptide dendrimers
    Susanna Zamolo, Tamis Darbre, Jean‐Louis Reymond · 2020 · Chemical Communications
  91. Current Treatment Options for Cystic Fibrosis-Related Liver Disease
    Katharina Staufer · 2020 · International Journal of Molecular Sciences
  92. CRISPR-Cas9 for treating hereditary diseases
    Indra Mani · 2021 · Progress in molecular biology and translational science
  93. Efficient and error-free fluorescent gene tagging in human organoids without double-strand DNA cleavage
    Yannik Bollen, Joris H. Hageman, Petra van Leenen, Lucca L. M. Derks, Bas Ponsioen, Julian R. Buissant des Amorie, Ingrid Verlaan-Klink, Myrna van den Bos, Leon W.M.M. Terstappen, Ruben van Boxtel et al. · 2022 · PLoS Biology
  94. Synergy of nanocarriers with CRISPR-Cas9 in an emerging technology platform for biomedical appliances: Current insights and perspectives
    Rahul Bhattacharjee, Ankit Jana, Aditya Nandi, Adrija Sinha, Arkadyuti Bhattacharjee, Sagnik Mitra, Sulagna Kar, Abhijit Dey, Sushil Kumar Singh, Rajender S. Varma et al. · 2022 · Materials & Design
  95. Building gut from scratch — progress and update of intestinal tissue engineering
    Tullie, Lucinda, Jones, Brendan C., De Coppi, Paolo, Li, Vivian S. W. · 2022 · Nature Reviews Gastroenterology & Hepatology
  96. Nanomaterials-assisted gene editing and synthetic biology for optimizing the treatment of pulmonary diseases
    Lanjie Lei, Wenjie Pan, Xin Shou, Yunyuan Shao, Shuxuan Ye, Junfeng Zhang, Narasaiah Kolliputi, Liyun Shi · 2024 · Journal of Nanobiotechnology
  97. La révolution des CRISPR est en marche
    Hélène Gilgenkrantz · 2014 · médecine/sciences
  98. CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy
    Xiaohui Zhang, Liren Wang, Mingyao Liu, Dali Li · 2017 · Science China Life Sciences
  99. Intestinal organoids in infants and children
    Sinobol Chusilp, Bo Li, Dorothy Lee, Carol Lee, Paisarn Vejchapipat, Agostino Pierro · 2019 · Pediatric Surgery International
  100. Protease associated domain of RNF43 is not necessary for the suppression of Wnt/β-catenin signaling in human cells
    Tomasz Witold Radaszkiewicz, Vı́tězslav Bryja · 2020 · Cell Communication and Signaling