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

DOI 10.1016/j.stem.2013.11.002

Cited by (1,001)

Items 101–200 of 1,001

Organoid Models of Human Gastrointestinal Development and Disease
Priya H. Dedhia, Nina Bertaux‐Skeirik, Yana L. Zavros, Jason R. Spence · 2016 · Gastroenterology
Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells
Wu, Yuxuan, Zhou, Hai, Fan, Xiaoying, Zhang, Ying, Zhang, Man, Wang, Yinghua, Xie, Zhenfei, Bai, Meizhu, Yin, Qi, Liang, Dan et al. · 2015 · Cell Research
Engineering human tumour-associated chromosomal translocations with the RNA-guided CRISPR–Cas9 system
Raúl Torres-Ruiz, Marina Carrasco Martin, Angie Fernanda Rodriguez Garcia, Juan C. Cigudosa, Juan Carlos Ramı́rez, Sandra Rodríguez · 2014 · Nature Communications
What is CRISPR/Cas9?
Redman, Melody, King, Andrew, Watson, Caroline, King, David · 2016 · Archives of disease in childhood - Education & practice edition
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Xu, Li, Park, Ki Ho, Zhao, Lixia, Xu, Jing, El Refaey, Mona, Gao, Yandi, Zhu, Hua, Ma, Jianjie, Han, Renzhi · 2016 · Molecular Therapy
High-throughput and all-solution phase African Swine Fever Virus (ASFV) detection using CRISPR-Cas12a and fluorescence based point-of-care system
Qian He, Dongmei Yu, Mengdi Bao, Grant Korensky, Juhong Chen, Mingyeong Shin, Juwon Kim, Myeongkee Park, Peiwu Qin, Ke Du · 2020 · Biosensors and Bioelectronics
Transplantation of engineered organoids enables rapid generation of metastatic mouse models of colorectal cancer
Kevin P. O’Rourke, Evangelia Loizou, Geulah Livshits, Emma M. Schatoff, Timour Baslan, Eusebio Manchado, Janelle M. Simon, Paul Bernard Romesser, Benjamin I. Leach, Teng Han et al. · 2017 · Nature Biotechnology
The rapidly evolving view of lysosomal storage diseases
Parenti, Giancarlo, Medina, Diego L, Ballabio, Andrea · 2021 · EMBO Molecular Medicine
Genome editing with the CRISPR‐Cas system: an art, ethics and global regulatory perspective
Debin Zhang, Amjad Hussain, Hakim Manghwar, Kabin Xie, Shengsong Xie, Shuhong Zhao, Robert M. Larkin, Ping Qing, Shuangxia Jin, Fang Ding · 2020 · Plant Biotechnology Journal
CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation
Xin Han, Zongbin Liu, Myeong Chan Jo, Kai Zhang, Ying Li, Zihua Zeng, Nan Li, Youli Zu, Lidong Qin · 2015 · Science Advances
A CRISPR view of development
Melissa M. Harrison, Brian Jenkins, Kate M. O’Connor-Giles, Jill Wildonger · 2014 · Genes & Development
Retinal stem cell transplantation: Balancing safety and potential
Mandeep S. Singh, Susanna S. Park, Thomas Arno Albini, M. Valeria Canto-Soler, Henry J. Klassen, Robert Edward MacLaren, Masayo Takahashi, Aaron Nagiel, Steven D. Schwartz, Kapil Bharti · 2019 · Progress in Retinal and Eye Research
Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles
Ying‐Li Luo, Cong‐Fei Xu, Hongjun Li, Zhiting Cao, Jing Liu, Jilong Wang, Xiao‐Jiao Du, Xianzhu Yang, Zhen Gu, Jun Wang · 2018 · ACS Nano
CRISPR-Based Adenine Editors Correct Nonsense Mutations in a Cystic Fibrosis Organoid Biobank
Geurts, Maarten H., de Poel, Eyleen, Amatngalim, Gimano D., Oka, Rurika, Meijers, Fleur M., Kruisselbrink, Evelien, van Mourik, Peter, Berkers, Gitte, de Winter-de Groot, Karin M., Michel, Sabine et al. · 2020 · Cell Stem Cell
Pooled In Vitro and In Vivo CRISPR-Cas9 Screening Identifies Tumor Suppressors in Human Colon Organoids
Michels, Birgitta E., Mosa, Mohammed H., Streibl, Barbara I., Zhan, Tianzuo, Menche, Constantin, Abou-El-Ardat, Khalil, Darvishi, Tahmineh, Członka, Ewelina, Wagner, Sebastian, Winter, Jan et al. · 2020 · Cell Stem Cell
Principles, Applications, and Biosafety of Plant Genome Editing Using CRISPR-Cas9
Kaoutar El‐Mounadi, María Luisa Morales-Floriano, Hernán García-Ruíz · 2020 · Frontiers in Plant Science
Bioengineering Approaches for the Advanced Organoid Research
Yi, Sang Ah, Zhang, Yixiao, Rathnam, Christopher, Pongkulapa, Thanapat, Lee, Ki‐Bum · 2021 · Advanced Materials
Stem cell-derived organoids and their application for medical research and patient treatment
Sina Bartfeld, Hans C. Clevers · 2017 · Journal of Molecular Medicine
Probing the Tumor Suppressor Function of BAP1 in CRISPR-Engineered Human Liver Organoids
Artegiani, Benedetta, van Voorthuijsen, Lisa, Lindeboom, Rik G.H., Seinstra, Daniëlle, Heo, Inha, Tapia, Pablo, López-Iglesias, Carmen, Postrach, Daniel, Dayton, Talya, Oka, Rurika et al. · 2019 · Cell Stem Cell
3D cell culture models: Drug pharmacokinetics, safety assessment, and regulatory consideration
Hongbing Wang, Paul C. Brown, Edwin C.Y. Chow, Lorna C. Ewart, Stephen Ferguson, Suzanne Fitzpatrick, Benjamin Freedman, Grace Liejun Guo, William D. Hedrich, Scott A. Heyward et al. · 2021 · Clinical and Translational Science
Modeling Pancreatic Cancer with Organoids
Lindsey A. Baker, Hervé Tiriac, Hans C. Clevers, David Arthur Tuveson · 2016 · Trends in cancer
Mini-Gut Organoids: Reconstitution of the Stem Cell Niche
Shoichi Date, Toshiro Sato · 2015 · Annual Review of Cell and Developmental Biology
RNF43 germline and somatic mutation in serrated neoplasia pathway and its association with BRAF mutation
Helen H N Yan, Jeffrey C W Lai, Siu Lun Ho, Wai K. Leung, Wai Lun Law, Janet F. Y. Lee, Anthony K W Chan, Wai Yin Tsui, Annie S.Y. Chan, Bernard C. H. Lee et al. · 2016 · Gut
Organoids for Drug Discovery and Personalized Medicine
Toshio Takahashi · 2018 · The Annual Review of Pharmacology and Toxicology
CRISPR-Cas9: a new and promising player in gene therapy
Xiao-Jie, Lu, Hui-Ying, Xue, Zun-Ping, Ke, Jin-Lian, Chen, Li-Juan, Ji · 2015 · Journal of Medical Genetics
Strategies for nonviral nanoparticle‐based delivery of CRISPR/Cas9 therapeutics
Chen, Fengqian, Alphonse, Martin, Liu, Qi · 2020 · WIREs Nanomedicine and Nanobiotechnology
CRISPR-Cas9-Mediated Single-Gene and Gene Family Disruption in Trypanosoma cruzi
Duo Peng, Samarchith P. Kurup, Phil Y. Yao, Todd A. Minning, Rick L. Tarleton · 2014 · mBio
Use and application of 3D-organoid technology
Benedetta Artegiani, Hans C. Clevers · 2018 · Human Molecular Genetics
Gene editing and CRISPR in the clinic: current and future perspectives
Matthew P. Hirakawa, Raga Krishnakumar, Jerilyn A. Timlin, James P. Carney, Kimberly S. Butler · 2020 · Bioscience Reports
CRISPR-Cas Tools and Their Application in Genetic Engineering of Human Stem Cells and Organoids
Delilah F. G. Hendriks, Hans C. Clevers, Benedetta Artegiani · 2020 · Cell stem cell
Regenerative Reprogramming of the Intestinal Stem Cell State via Hippo Signaling Suppresses Metastatic Colorectal Cancer
Priscilla Cheung, Jordi Xiol, Michael T. Dill, Wei-Chien Yuan, Riccardo Panero, Jatin Roper, Fernando G. Osorio, Dejan Maglic, Qi Li, Basanta Gurung et al. · 2020 · Cell stem cell
A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9
Kelli J. Carroll, Catherine A. Makarewich, John McAnally, Douglas M. Anderson, Lorena Zentilin, Ning Liu, Mauro Giacca, Rhonda Bassel‐Duby, Eric N. Olson · 2015 · Proceedings of the National Academy of Sciences
Precision genome editing in the CRISPR era
Jayme Salsman, Graham Dellaire · 2016 · Biochemistry and Cell Biology
Human enteroids as an <i>ex-vivo</i> model of host–pathogen interactions in the gastrointestinal tract
Foulke-Abel, Jennifer, In, Julie, Kovbasnjuk, Olga, Zachos, Nicholas C, Ettayebi, Khalil, Blutt, Sarah E, Hyser, Joseph M, Zeng, Xi-Lei, Crawford, Sue E, Broughman, James R et al. · 2014 · Experimental Biology and Medicine
Disease Modeling Using 3D Organoids Derived from Human Induced Pluripotent Stem Cells
Beatrice Xuan Ho, Nicole Min Qian Pek, Boon-Seng Soh · 2018 · International Journal of Molecular Sciences
Development of CRISPR-Cas systems for genome editing and beyond
F. Zhang · 2019 · Quarterly Reviews of Biophysics
Insert, remove or replace: A highly advanced genome editing system using CRISPR/Cas9
Stanislaus Antony Ceasar, Vinothkumar Rajan, Sergey V. Prykhozhij, Jason N. Berman, Savarimuthu Ignacimuthu S.J. · 2016 · Biochimica et Biophysica Acta (BBA) - Molecular Cell Research
Organoid culture systems to study host–pathogen interactions
Devanjali Dutta, Hans C. Clevers · 2017 · Current Opinion in Immunology
CRISPR/Cas9 Immune System as a Tool for Genome Engineering
Magdalena Hryhorowicz, Daniel Lipiński, Joanna Zeyland, Ryszard Słomski · 2016 · Archivum Immunologiae et Therapiae Experimentalis
CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse
Yuting Guan, Yanlin Ma, Qi Li, Zhenliang Sun, Lie Ma, Lijuan Wu, Liren Wang, Li Zeng, Yanjiao Shao, Yuting Chen et al. · 2016 · EMBO Molecular Medicine
DNA methylation defines regional identity of human intestinal epithelial organoids and undergoes dynamic changes during development
Judith Kraiczy, Komal Nayak, Kate J. Howell, Alexander Ross, Jessica L. Forbester, Camilla Salvestrini, Roxana C. Mustata, Sally Perkins, Amanda Andersson-Rolf, Esther Leenen et al. · 2017 · Gut
In Vitro Models to Study Human Lung Development, Disease and Homeostasis.
Alyssa J. Miller, J. Spence · 2017 · Physiology
Patient-derived tumor organoids for prediction of cancer treatment response
Peter W. Nagle, John Th. M. Plukker, Christina T. Muijs, Peter van Luijk, Robert Paul Coppes · 2018 · Seminars in Cancer Biology
Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs
Cory J. Smith, Leire Abalde-Atristain, Chaoxia He, Brett R. Brodsky, Evan M. Braunstein, Pooja Chaudhari, Yoon-Young Jang, Linzhao Cheng, Zhaohui Ye · 2014 · Molecular Therapy
Mitochondrial replacement therapy in reproductive medicine
Don P. Wolf, Nargiz Mitalipov, Shoukhrat M. Mitalipov · 2014 · Trends in Molecular Medicine
Heterogeneity of hepatocellular carcinoma: from mechanisms to clinical implications
Fatema Safri, Romario Nguyen, Shadi Zerehpooshnesfchi, Jacob George, Liang Qiao · 2024 · Cancer Gene Therapy
Stem Cells Marked by the R-Spondin Receptor LGR5
Bon‐Kyoung Koo, Hans C. Clevers · 2014 · Gastroenterology
New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls
Scott Cameron Bell, K. De Boeck, Margarida Duarte Amaral · 2014 · Pharmacology & Therapeutics
Advances in CRISPR-Cas9 genome engineering: lessons learned from RNA interference
Rodolphe Barrangou, Amanda Birmingham, Stefan Wiemann, Roderick L. Beijersbergen, Veit Hornung, Anja van Brabant Smith · 2015 · Nucleic Acids Research
A Chemically Defined Hydrogel for Human Liver Organoid Culture
Shicheng Ye, Jochem W.B. Boeter, Marko Mihajlovic, Frank G. van Steenbeek, Monique E. van Wolferen, Loes A. Oosterhoff, Ary Marsee, Massimiliano Caiazzo, Luc J. W. van der Laan, Louis C. Penning et al. · 2020 · Advanced Functional Materials
Somatic cell-derived organoids as prototypes of human epithelial tissues and diseases
Masayuki Fujii, Toshiro Sato · 2020 · Nature Materials
Recurrent WNT pathway alterations are frequent in relapsed small cell lung cancer
Alex Handler Wagner, Siddhartha H. K. Devarakonda, Zachary L. Skidmore, Kilannin Krysiak, Avinash Ramu, Lee Trani, Jason Kunisaki, Ashiq Masood, Saiama Naheed Waqar, Nicholas C. Spies et al. · 2018 · Nature Communications
USP7 Is a Tumor-Specific WNT Activator for APC-Mutated Colorectal Cancer by Mediating β-Catenin Deubiquitination
Laura Novellasdemunt, Valentina Foglizzo, Laura Gómez-Cuadrado, Pedro Antas, Anna Kucharska, Vesela Encheva, Ambrosius P. Snijders, Vivian Sze Wing Li · 2017 · Cell Reports
Establishment of human fetal hepatocyte organoids and CRISPR–Cas9-based gene knockin and knockout in organoid cultures from human liver
Delilah F. G. Hendriks, Benedetta Artegiani, Huili Hu, Susana M. Chuva de Sousa Lopes, Hans C. Clevers · 2020 · Nature Protocols
CRISPR-Cas for genome editing: Classification, mechanism, designing and applications
Simran Kaur Bhatia, Pooja ., Sudesh Kumar Yadav · 2023 · International Journal of Biological Macromolecules
Lipid nanoparticles for short interfering RNA delivery
Leung, Alex K.K., Tam, Yuen Yi C., Cullis, Pieter R. · 2014 · Advances in genetics
Generation of Genomic Deletions in Mammalian Cell Lines via CRISPR/Cas9
Daniel E. Bauer, Matthew C. Canver, Stuart H. Orkin · 2014 · Journal of Visualized Experiments
A general strategy to construct small molecule biosensors in eukaryotes
Justin Feng, Benjamin W. Jester, Christine Elaine Tinberg, Daniel J. Mandell, Mauricio S. Antunes, Raj Chari, Kevin J. Morey, Xavier Rios, June I. Medford, George McDonald Church et al. · 2015 · eLife
Concise Review: Current Status of Three-Dimensional Organoids as Preclinical Models
Garima Kaushik, Moorthy Palanimuthu Ponnusamy, Surinder K. Batra · 2018 · Stem Cells
Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System
Bing Song, Yong Fan, Wenyin He, Detu Zhu, Xiaohua Niu, Ding Wang, Zhanhui Ou, Min Luo, Xiaofang Sun · 2014 · Stem Cells and Development
CRISPR/Cas 9 genome editing and its applications in organoids
Else Driehuis, Hans C. Clevers · 2017 · American Journal of Physiology-Gastrointestinal and Liver Physiology
BRCA-deficient mouse mammary tumor organoids to study cancer-drug resistance
Alexandra A. Duarte, Ewa Gogola, Norman Sachs, Marco Barazas, Stefano Annunziato, Julian R. de Ruiter, Arno Velds, Sohvi Blatter, Julia M. Houthuijzen, Marieke van de Ven et al. · 2017 · Nature Methods
International regulatory landscape and integration of corrective genome editing into in vitro fertilization
Araki, Motoko, Ishii, Tetsuya · 2014 · Reproductive Biology and Endocrinology
Organoids as Model for Infectious Diseases: Culture of Human and Murine Stomach Organoids and Microinjection of Helicobacter Pylori
Sina Bartfeld, Hans C. Clevers · 2015 · Journal of Visualized Experiments
New and emerging targeted therapies for cystic fibrosis
Bradley S. Quon, Steven Mark Rowe · 2016 · BMJ
Gut organoids: mini-tissues in culture to study intestinal physiology and disease
Mohammad Almeqdadi, Miyeko Mana, Jatin Roper, Ömer H. Yilmaz · 2019 · American Journal of Physiology-Cell Physiology
Efficient CRISPR-Cas9-Mediated Generation of Knockin Human Pluripotent Stem Cells Lacking Undesired Mutations at the Targeted Locus
Merkle, Florian T., Neuhausser, Werner M., Santos, David, Valen, Eivind, Gagnon, James A., Maas, Kristi, Sandoe, Jackson, Schier, Alexander F., Eggan, Kevin · 2015 · Cell Reports
CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles
Wei-Jing Dai, Liyao Zhu, Zhongyi Yan, Yong Xu, Qilong Wang, Xiaojie Lu · 2016 · Molecular Therapy — Nucleic Acids
Future therapies for cystic fibrosis
Lucy Allen, Lorna J Allen, S.B. Carr, Gwyneth Davies, Damian G. Downey, Marie E. Egan, Julian T. Forton, Robert D. Gray, CHARLES S. HAWORTH, Alex Robert Horsley et al. · 2023 · Nature Communications
3D Cell Cultures: Evolution of an Ancient Tool for New Applications
Andrea Cacciamali, Riccardo Villa, Silvia Dotti · 2022 · Frontiers in Physiology
Mechanisms of Pediatric Inflammatory Bowel Disease
Joanna M. Peloquin, Gautam Goel, Eduardo J. Villablanca, Ramnik J. Xavier · 2016 · Annual Review of Immunology
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
Nicole Ali McNeer, Kavitha K. Anandalingam, Rachel J. Fields, Christina R. Caputo, Sascha Kopic, Anisha Gupta, Elias Quijano, Lee A. Polikoff, Yong Kong, Raman Bahal et al. · 2015 · Nature Communications
Translational applications of adult stem cell-derived organoids
Jarno Drost, Hans C. Clevers · 2017 · Development
Modeling Human Digestive Diseases With CRISPR-Cas9–Modified Organoids
Masayuki Fujii, Hans C. Clevers, Toshiro Sato · 2018 · Gastroenterology
Organoid based personalized medicine: from bench to bedside
Yaqi Li, Peiyuan Tang, Sanjun Cai, Junjie Peng, Guoqiang Hua · 2020 · Cell Regeneration
Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids
Geurts, Maarten H, de Poel, Eyleen, Pleguezuelos-Manzano, Cayetano, Oka, Rurika, Carrillo, Léo, Andersson-Rolf, Amanda, Boretto, Matteo, Brunsveld, Jesse E, van Boxtel, Ruben, Beekman, Jeffrey M et al. · 2021 · Life Science Alliance
Current Progress in Therapeutic Gene Editing for Monogenic Diseases
Versha Prakash, Marc Moore, Rafael J. Yáñez‐Muñoz · 2016 · Molecular Therapy
DNA stretching induces Cas9 off-target activity
Matthew D. Newton, Ben Taylor, Rosalie P.C. Driessen, Leonie Roos, Nevena Cvetešić, Shenaz Allyjaun, Boris Lenhard, Maria Emanuela Cuomo, David Rueda · 2019 · Nature Structural & Molecular Biology
CRISPR-Based Diagnosis of Infectious and Noninfectious Diseases
Somayeh Jolany vangah, Camellia Katalani, Hannah A. Boone, Abbas Hajizade, Adna Sijerčić, Gholamreza Ahmadian · 2020 · Biological Procedures Online
Establishment of Human Epithelial Enteroids and Colonoids from Whole Tissue and Biopsy
Maxime M. Mahé, Nambirajan Sundaram, Carey L. Watson, Noah F. Shroyer, Michael A. Helmrath · 2015 · Journal of Visualized Experiments
Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing
Tuo Wei, Qiang Cheng, Lukas Farbiak, Daniel G. Anderson, Róbert Langer, Daniel J. Siegwart · 2020 · ACS Nano
Delivery and therapeutic applications of gene editing technologies ZFNs, TALENs, and CRISPR/Cas9
Justin S. LaFountaine, Kristin Fathe, Hugh D. C. Smyth · 2015 · International Journal of Pharmaceutics
High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia
Sriram Vaidyanathan, Ameen Abdulla Salahudeen, Zachary M. Sellers, Dawn T. Bravo, S. S. Choi, Arpit Batish, Wei Le, Ron Baik, Sean de la O, Milan P. Kaushik et al. · 2019 · Cell stem cell
Gesicle-Mediated Delivery of CRISPR/Cas9 Ribonucleoprotein Complex for Inactivating the HIV Provirus
Lee Ann Campbell, Lamarque M. Coke, Christopher T. Richie, Lowella V. Fortuno, Aaron Y. Park, Brandon K. Harvey · 2018 · Molecular Therapy
Gene therapy: progress and predictions
Mary K. Collins, Adrian James Thrasher · 2015 · Proceedings of the Royal Society B Biological Sciences
Cancer Spheroids and Organoids as Novel Tools for Research and Therapy: State of the Art and Challenges to Guide Precision Medicine
Sanae El Harane, Bochra Zidi, Nadia El Harane, Karl‐Heinz Krause, Thomas Matthes, Olivier Preynat‐Seauve · 2023 · Cells
Game changers in science and technology - now and beyond
Ulrich A. K. Betz, Loukik Arora, R.A. Assal, Hatylas F. Z de Azevedo, Jeremy Baldwin, Michael S. Becker, Stefan Bostock, Vinton W.T. Cheng, Tobias Egle, Nicola Ferrari et al. · 2023 · Technological Forecasting and Social Change
Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis
Guan, Shan, Munder, Antje, Hedtfeld, Silke, Braubach, Peter, Glage, Silke, Zhang, Longgui, Lienenklaus, Stefan, Schultze, Anja, Hasenpusch, Günther, Garrels, Wiebke et al. · 2019 · Nature Nanotechnology
Genetic editing of colonic organoids provides a molecularly distinct and orthotopic preclinical model of serrated carcinogenesis
Tamsin R.M. Lannagan, Young K Lee, Tongtong Wang, Jatin Roper, Mark L. Bettington, Lochlan Fennell, Laura Vrbanac, Lisa Jonavicius, Roshini Somashekar, Krystyna A. Gieniec et al. · 2018 · Gut
Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review)
Diana Raquel Rodríguez-Rodríguez, Ramiro Ram�rez‐Sol�s, Mario Alberto Garza‐Elizondo, Mar�a Garza‐Rodr�guez, Hugo Alberto Barrera-Saldaña · 2019 · International Journal of Molecular Medicine
Responsive Nanocarriers as an Emerging Platform for Cascaded Delivery of Nucleic Acids to Cancer
Yang Liu, Cong‐Fei Xu, Shoaib Iqbal, Xianzhu Yang, Jun Wang · 2017 · Advanced Drug Delivery Reviews
Emerging trends in organ-on-a-chip systems for drug screening
Yanping Wang, Yanfeng Gao, Yongchun Pan, Dongtao Zhou, Yuta Liu, Yi Bing Yin, Jingjing Yang, Yuzhen Wang, Yujun Song · 2023 · Acta Pharmaceutica Sinica B
CRISPR-Cas: Converting A Bacterial Defence Mechanism into A State-of-the-Art Genetic Manipulation Tool
Loureiro, Alexandre, da Silva, Gabriela Jorge · 2019 · Antibiotics
Three-dimensional models for studying development and disease: moving on from organisms to organs-on-a-chip and organoids
Emily Jackson, Hang Lu · 2016 · Integrative Biology
Convergence of microengineering and cellular self-organization towards functional tissue manufacturing
Jérémie Laurent, Guillaume Blin, François Chatelain, Valérie Vanneaux, Alexandra Fuchs, Jérôme Larghero, Manuel Théry · 2017 · Nature Biomedical Engineering
CRISPR/Cas9‐mediated genome editing: From basic research to translational medicine
Filipe V. Jacinto, Wolfgang Link, Bibiana I. Ferreira · 2020 · Journal of Cellular and Molecular Medicine
Research Progress, Challenges, and Breakthroughs of Organoids as Disease Models
Yisheng Huang, Zhijie Huang, Zhengming Tang, Yuanxin Chen, Mingshu Huang, Hongyu Liu, Weibo Huang, Qingsong Ye, Bo Jia · 2021 · Frontiers in Cell and Developmental Biology
CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting
David Courtney, Jonathan Edward Moore, Sarah D. Atkinson, Eleonora Maurizi, Edwin H. A. Allen, Deena M. Leslie Pedrioli, W.H. Irwin McLean, M. Andrew Nesbit, Tara Moore · 2015 · Gene Therapy
Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine
Iwona Maria Pranke, Anita Golec, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet‐Gaudelus · 2019 · Frontiers in Pharmacology
Protospacer adjacent motif (PAM)-distal sequences engage CRISPR Cas9 DNA target cleavage
Cencic, Regina, Miura, Hisashi, Malina, Abba, Robert, Francis, Ethier, Sylvain, Schmeing, T. Martin, Dostie, Josée, Pelletier, Jerry · 2014 · PLoS ONE