Correction of a genetic disease in mouse via use of CRISPR-Cas9

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Items 101–200 of 620

1. Genome Editing of eIF4E1 in Tomato Confers Resistance to Pepper Mottle Virus
   Yoo-Joung Yoon, Jelli Venkatesh, Joung‐Ho Lee, Jinhee Kim, Hye‐Eun Lee, Do-Sun Kim, Byoung‐Cheorl Kang · 2020 · Frontiers in Plant Science
2. Precision cancer mouse models through genome editing with CRISPR-Cas9
   Haiwei Mou, Zachary Kennedy, Daniel G. Anderson, Hao Yin, Wen Xue · 2015 · Genome Medicine
3. Simple generation of albino C57BL/6J mice with G291T mutation in the tyrosinase gene by the CRISPR/Cas9 system
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4. Genome engineering through CRISPR/Cas9 technology in the human germline and pluripotent stem cells
   Rita Vassena, Björn Heindryckx, Rubén Peco, Guido Pennings, Ángel Raya, Karen Sermon, Anna Veiga · 2016 · Human Reproduction Update
5. Hox11 expressing regional skeletal stem cells are progenitors for osteoblasts, chondrocytes and adipocytes throughout life
   Kyriel M. Pineault, Jane Y. Song, Kenneth M. Kozloff, Daniel Lucas, Deneen M. Wellik · 2019 · Nature Communications
6. Inter-homologue repair in fertilized human eggs?
   Egli, Dieter, Zuccaro, Michael V., Kosicki, Michael, Church, George M., Bradley, Allan, Jasin, Maria · 2018 · Nature
7. Systemic delivery of CRISPR/Cas9 with PEG-PLGA nanoparticles for chronic myeloid leukemia targeted therapy
   Yang Liu, Gui Zhao, Cong‐Fei Xu, Ying‐Li Luo, Zi-Dong Lu, Jun Wang · 2018 · Biomaterials Science
8. A glance at genome editing with CRISPR–Cas9 technology
   Antara Barman, Bornali Deb, Supriyo Chakraborty · 2019 · Current Genetics
9. CRISPR-based technologies: prokaryotic defense weapons repurposed
   Rebecca M. Terns, Michael P. Terns · 2014 · Trends in Genetics
10. Gene Therapy in Retinal Dystrophies
    Ziccardi, Lucia, Cordeddu, Viviana, Gaddini, Lucia, Matteucci, Andrea, Parravano, Mariacristina, Malchiodi-Albedi, Fiorella, Varano, Monica · 2019 · International Journal of Molecular Sciences
11. Patient-specific induced pluripotent stem cells (iPSCs) for the study and treatment of retinal degenerative diseases
    Luke A. Wiley, Erin R. Burnight, Allison E. Songstad, Arlene V. Drack, Robert F. Mullins, Edwin M. Stone, Budd A. Tucker · 2014 · Progress in Retinal and Eye Research
12. CRISPR/Cas9 for Human Genome Engineering and Disease Research
    Xin Xiong, Meng Chen, Wendell A. Lim, Dehua Zhao, Lei S. Qi · 2016 · Annual Review of Genomics and Human Genetics
13. Mouse Genome Informatics (MGI) Resource: Genetic, Genomic, and Biological Knowledgebase for the Laboratory Mouse
    Janan T. Eppig · 2017 · ILAR Journal
14. Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression
    Pan Gao, Qing Lyu, Amr R. Ghanam, Cícera Regina Lazzarotto, Gregory A. Newby, Wei Zhang, Mi‐Hyun Choi, Orazio J. Slivano, Kevin Holden, John A. Walker et al. · 2021 · Genome biology
15. CRISPR/Cas System and Factors Affecting Its Precision and Efficiency
    Nasir Javaid, Sangdun Choi · 2021 · Frontiers in Cell and Developmental Biology
16. Guide RNA modification as a way to improve CRISPR/Cas9-based genome-editing systems
    Julia A. Filippova, Anastasiya M. Matveeva, Evgenii S. Zhuravlev, Grigory A. Stepanov · 2019 · Biochimie
17. Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
    Conway, Anthony, Mendel, Matthew, Kim, Kenneth, McGovern, Kyle, Boyko, Alisa, Zhang, Lei, Miller, Jeffrey C., DeKelver, Russell C., Paschon, David E., Mui, Barbara L. et al. · 2019 · Molecular Therapy
18. Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy
    Nari Ryu, Min-A Kim, Dongsik Park, Byeonghyeon Lee, Ye‐Ri Kim, Kyung‐Hee Kim, Jeong‐In Baek, Won Jong Kim, Kyu-Yup Lee, Un‐Kyung Kim · 2018 · Nanomedicine Nanotechnology Biology and Medicine
19. Modification of Lipid-Based Nanoparticles: An Efficient Delivery System for Nucleic Acid-Based Immunotherapy
    Chi Zhang, Yifan Ma, Jingjing Zhang, Jimmy Chun‐Tien Kuo, Zhongkun Zhang, Haotian Xie, Jing Zhu, Tongzheng Liu · 2022 · Molecules
20. CRISPR-DT: designing gRNAs for the CRISPR-Cpf1 system with improved target efficiency and specificity
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21. Effective gene editing by high-fidelity base editor 2 in mouse zygotes
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22. Experiments that led to the first gene-edited babies: the ethical failings and the urgent need for better governance
    Jing-ru Li, Simon Thomas Walker, Jing-Bao Nie, Xin-qing Zhang · 2019 · Journal of Zhejiang University SCIENCE B
23. In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway
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24. Genetically modified pigs are protected from classical swine fever virus
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25. Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin
    Honghai Tang, Hui Wang, Shengyi Wang, Shao Wei Hu, Jun Lv, Mengzhao Xun, Kaiyu Gao, Fang Wang, Yuxin Chen, Daqi Wang et al. · 2022 · Human Genetics
26. Advances in CRISPR/Cas9
    Youmin Zhu · 2022 · BioMed Research International
27. Ligase IV inhibitor SCR7 enhances gene editing directed by CRISPR–Cas9 and ssODN in human cancer cells
    Zheng Mao Hu, Zhaoying Shi, Xiaogang Guo, Baishan Jiang, Guo Wang, Dixian Luo, Yonglong Chen, Yuan‐Shan Zhu · 2018 · Cell & Bioscience
28. Protein-based lateral flow assays for COVID-19 detection
    Farbod Mahmoudinobar, Dustin Britton, Jin Kim Montclare · 2021 · Protein Engineering Design and Selection
29. Efficient embryonic homozygous gene conversion via RAD51-enhanced interhomolog repair
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30. CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes
    Hua Zhong, Yiyun Chen, Yumei Li, Rui Chen, Graeme Mardon · 2015 · Scientific Reports
31. Germ line genome editing in clinics: the approaches, objectives and global society
    Tetsuya Ishii · 2015 · Briefings in Functional Genomics
32. Optimization of the production of knock-in alleles by CRISPR/Cas9 microinjection into the mouse zygote
    Aurélien Raveux, Sandrine Vandormael‐Pournin, Michel Cohen‐Tannoudji · 2017 · Scientific Reports
33. Germline genome-editing research and its socioethical implications
    Tetsuya Ishii · 2015 · Trends in Molecular Medicine
34. Comparative analysis of single-stranded DNA donors to generate conditional null mouse alleles
    Denise Grant Lanza, Angelina Gaspero, Isabel Lorenzo, Lan Liao, Ping Zheng, Ying Wang, Yu Deng, Chonghui Cheng, Chuansheng Zhang, John R. Seavitt et al. · 2018 · BMC Biology
35. Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications
    Gargi Bhattacharjee, Nisarg Gohil, Khushal Khambhati, Indra Mani, Rupesh Maurya, Janardhan Keshav Karapurkar, Jigresh Gohil, Dinh‐Toi Chu, Hue Vu‐Thi, Khalid Jamaan Alzahrani et al. · 2022 · Journal of Controlled Release
36. Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine
    Sarkar Sardar Azeez, Rahin Shareef Hamad, BahraK Hamad, Mudhir Sabir Shekha, Peter Bergsten · 2024 · Frontiers in Genome Editing
37. The new CRISPR–Cas system: RNA-guided genome engineering to efficiently produce any desired genetic alteration in animals
    Davide Seruggia, Lluı́s Montoliu · 2014 · Transgenic Research
38. ASXL1 mutation correction by CRISPR/Cas9 restores gene function in leukemia cells and increases survival in mouse xenografts
    Simona Valletta, Hamid Dolatshad, Matthias Bartenstein, Bon Ham Yip, Erica Bello, Shanisha A. K. Gordon, Yiting Yu, Jacqueline M. Shaw, Swagata Roy, Laura Scifo et al. · 2015 · Oncotarget
39. Genome Editing and Its Applications in Model Organisms
    Dongyuan Ma, Feng Liu · 2015 · Genomics Proteomics & Bioinformatics
40. Genome editing and assisted reproduction: curing embryos, society or prospective parents?
    Giulia Cavaliere · 2017 · Medicine Health Care and Philosophy
41. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency
    Chun‐Qing Song, Dan Wang, Tingting Jiang, Kevin W. O’Connor, Qiushi Tang, Lingling Cai, Xiangrui Li, Zhiping Weng, Hao Yin, Guangping Gao et al. · 2018 · Human Gene Therapy
42. CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice
    Cong Huai, Chenqiang Jia, Ruilin Sun, Peipei Xu, Taishan Min, Qihan Wang, Chengde Zheng, Hongyan Chen, Daru Lu · 2017 · Human Genetics
43. Spermatogonial stem cell autotransplantation and germline genomic editing: a future cure for spermatogenic failure and prevention of transmission of genomic diseases
    Callista L. Mulder, Yi Zheng, Sabrina Z. Jan, Robin B. Struijk, Sjoerd Repping, Geert Hamer, Ans M. M. van Pelt · 2016 · Human Reproduction Update
44. Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina
    Hickey, D G, Edwards, T L, Barnard, A R, Singh, M S, de Silva, S R, McClements, M E, Flannery, J G, Hankins, M W, MacLaren, R E · 2017 · Gene Therapy
45. Imbalance of Excitatory/Inhibitory Neuron Differentiation in Neurodevelopmental Disorders with an NR2F1 Point Mutation
    Ke Zhang, Yu Fang, Jian Kang Zhu, Sue Han, Jiehui Chen, Xuanyuan Wu, Yingying Chen, Tingyu Shen, Jiaoyang Liao, Wenke Guo et al. · 2020 · Cell Reports
46. Strategies for Efficient Genome Editing Using CRISPR-Cas9
    Behnom Farboud, Aaron F. Severson, Barbara J Meyer · 2018 · Genetics
47. Nanotechnology based CRISPR/Cas9 system delivery for genome editing: Progress and prospect
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48. CRISPR–Cas system: a powerful tool for genome engineering
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49. Nanoparticles-mediated CRISPR/Cas9 delivery: Recent advances in cancer treatment
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50. Responsible innovation in human germline gene editing: Background document to the recommendations of ESHG and ESHRE
    Guido M W R de Wert, Björn Heindryckx, Guido Pennings, Angus John Clarke, Ursula Eichenlaub-Ritter, Carla Geertruida van El, Francesca Forzano, Mariëtte Goddijn, Heidi Carmen Howard, Dragica P. Radojkovic et al. · 2018 · European Journal of Human Genetics
51. Selective targeting of the oncogenic KRAS G12S mutant allele by CRISPR/Cas9 induces efficient tumor regression
    Qianqian Gao, Wenjie Ouyang, Bin Kang, Xu Han, Ying Xiong, Renpeng Ding, Yijian Li, Fei Wang, Lei Huang, Lei Chen et al. · 2020 · Theranostics
52. LCOR mediates interferon-independent tumor immunogenicity and responsiveness to immune-checkpoint blockade in triple-negative breast cancer
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53. Gene therapy strategies in the treatment of hypertrophic cardiomyopathy
    Maksymilian Prondzynski, Giulia Mearini, Lucie Carrier · 2018 · Pflügers Archiv - European Journal of Physiology
54. Genome Editing in Mice Using CRISPR/Cas9 Technology
    Bradford Hall, Andrew Cho, Advait Limaye, Kyoungin Cho, Jaspal Singh Khillan, Ashok B. Kulkarni · 2018 · Current Protocols in Cell Biology
55. THUMPD3–TRMT112 is a m2G methyltransferase working on a broad range of tRNA substrates
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56. Optimization of lipid-assisted nanoparticle for disturbing neutrophils-related inflammation
    Yang Liu, Zhiting Cao, Cong‐Fei Xu, Zi-Dong Lu, Ying‐Li Luo, Jun Wang · 2018 · Biomaterials
57. Cell Line Techniques and Gene Editing Tools for Antibody Production: A Review
    Arun Kumar Dangi, Rajeshwari Sinha, Shailja Dwivedi, Sanjeev Kumar Gupta, Pratyoosh Shukla · 2018 · Frontiers in Pharmacology
58. Bacterial CRISPR/Cas DNA endonucleases: A revolutionary technology that could dramatically impact viral research and treatment
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59. CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
    Mujeeb Ur Rahman, Muhammad Bilal, Junaid Ali Shah, Ajeet Kumar Kaushik, Pierre‐Louis Teissèdre, Małgorzata Kujawska · 2022 · Pharmaceutics
60. Application of CRISPR/Cas9 genome editing to the study and treatment of disease
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61. Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases
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62. A practical guide to induced pluripotent stem cell research using patient samples
    Katherine E. Santostefano, Takashi Hamazaki, Nikolett M. Biel, Shouguang Jin, Akihiro Umezawa, Naohiro Terada · 2014 · Laboratory Investigation
63. Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
    Han‐Xiang Deng, Hong Zhai, Yong Shi, Guoxiang Liu, Jessica Lowry, Bin Liu, Éanna B. Ryan, Jianhua Yan, Yi Yang, Nigel Zhang et al. · 2021 · Communications Biology
64. Application of CRISPR/Cas9 for biomedical discoveries
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65. Customizing the genome as therapy for the β-hemoglobinopathies.
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66. CRISPR/Cas9 genome editing – new and old ethical issues arising from a revolutionary technology
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67. Intellectual disability‐associated gene ftsj1 is responsible for 2′‐O‐methylation of specific tRNAs
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68. Nanocarrier‐Based Systems for Targeted Delivery: Current Challenges and Future Directions
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69. Dual Supramolecular Nanoparticle Vectors Enable CRISPR/Cas9‐Mediated Knockin of Retinoschisin 1 Gene—A Potential Nonviral Therapeutic Solution for X‐Linked Juvenile Retinoschisis
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70. Endonucleases: new tools to edit the mouse genome
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71. Molecular mechanisms of psychiatric diseases
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72. A CRISPR Path to Engineering New Genetic Mouse Models for Cardiovascular Research
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73. The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
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74. Highly efficient base editing in human tripronuclear zygotes
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75. CRISPR/Cas9: Implications for Modeling and Therapy of Neurodegenerative Diseases
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76. Application of the gene editing tool, CRISPR-Cas9, for treating neurodegenerative diseases
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77. The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy
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78. CRISPR/Cas9: molecular tool for gene therapy to target genome and epigenome in the treatment of lung cancer
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79. Single-Base Resolution: Increasing the Specificity of the CRISPR-Cas System in Gene Editing
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80. Detailed Phenotypic and Molecular Analyses of Genetically Modified Mice Generated by CRISPR-Cas9-Mediated Editing
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81. Antiestrogen Resistant Cell Lines Expressing Estrogen Receptor α Mutations Upregulate the Unfolded Protein Response and are Killed by BHPI
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82. AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease
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83. Progress on the role of DNA methylation in aging and longevity
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84. Survival and Evolution of CRISPR–Cas System in Prokaryotes and Its Applications
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85. CRISPR/Cas9-Based Genome Editing in Plants
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86. CRISPR technology: The engine that drives cancer therapy
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87. Optogenetic Medicine: Synthetic Therapeutic Solutions Precision-Guided by Light
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88. Hox genes maintain critical roles in the adult skeleton
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89. CRISPR-Cas9: A fascinating journey from bacterial immune system to human gene editing
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90. A class of circadian long non-coding RNAs mark enhancers modulating long-range circadian gene regulation
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91. APOBEC3 induces mutations during repair of CRISPR–Cas9-generated DNA breaks
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92. Genes and genetics in eye diseases: a genomic medicine approach for investigating hereditary and inflammatory ocular disorders
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93. Gene Therapy for Cardiovascular Disease: Basic Research and Clinical Prospects
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94. Cas9 Ribonucleoprotein Delivery via Microfluidic Cell‐Deformation Chip for Human T‐Cell Genome Editing and Immunotherapy
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95. CRISPR therapeutic tools for complex genetic disorders and cancer (Review)
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96. Pluripotent stem cells and livestock genetic engineering
    Delia Alba Soto, Pablo J. Ross · 2016 · Transgenic Research
97. Repair of the TGFBI gene in human corneal keratocytes derived from a granular corneal dystrophy patient via CRISPR/Cas9-induced homology-directed repair
    Yukako Taketani, Kohdai Kitamoto, Toshihiro Sakisaka, Mikiko Kimakura, Tetsuya Toyono, Satoru Yamagami, Shiro Amano, Masahiko Kuroda, Tara Moore, Tomohiko Usui et al. · 2017 · Scientific Reports
98. Use of CRISPR/Cas9 gene-editing tools for developing models in drug discovery
    Gulzar Ahmad, Mansoor M. Amiji · 2018 · Drug Discovery Today
99. CRISPR/Cas9 System: A Bacterial Tailor for Genomic Engineering
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100. Current trends in gene recovery mediated by the CRISPR-Cas system
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