# CRISPR Gene Editing — science-database.com
> CRISPR-Cas9 and related gene editing technologies for precision genome modification. Covers therapeutic applications, agricultural biotech, gene drives, and ethical considerations.

- Discipline: Biology / Genetics
- URL: https://science-database.com/technology/crispr
- API: https://science-database.com/api/v1/technology/crispr
- Last Updated: 2026-04-11T05:58:40.505Z
- Articles Indexed: 22

## Top Publications

### CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
- Authors: Haydar Frangoul, David Altshuler, Maria Domenica Cappellini, Yi-Shan Chen, Jennifer Domm, Brenda K. Eustace, Juergen Foell, Josu de la Fuente, Stephan A. Grupp, Rupert Handgretinger, Tony W. Ho, Antonis Kattamis, Andrew Kernytsky, Julie Lekstrom-Himes, Amanda M. Li, Franco Locatelli, Markus Y. Mapara, Mariane de Montalembert, Damiano Rondelli, Akshay Sharma, Sujit Sheth, Sandeep Soni, Martin H. Steinberg, Donna A. Wall, Angela Yen, Selim Corbacioglu
- Journal: New England Journal of Medicine
- Date: 2020-12-05
- DOI: https://doi.org/10.1056/nejmoa2031054
- Citations: 1769
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W3112179900/llms.txt
- Abstract: Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in erythroid cells. We performed electroporation...

### CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
- Authors: Julian D. Gillmore, Ed Gane, Jörg Täubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica Seitzer, Daniel J. O’Connell, Kathryn Walsh, Kristy Wood, Jonathan A. Phillips, Yuanxin Xu, Adam Amaral, Adam P. Boyd, Jeffrey Cehelsky, Mark D. McKee, Andrew Schiermeier, Olivier Harari, Andrew Murphy, Christos A. Kyratsous, Brian Zambrowicz, Randy Soltys, David E. Gutstein, John P. Leonard, Laura Sepp‐Lorenzino, David Lebwohl
- Journal: New England Journal of Medicine
- Date: 2021-06-26
- DOI: https://doi.org/10.1056/nejmoa2107454
- Citations: 1572
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W3177445623/llms.txt
- Abstract: In a small group of patients with hereditary ATTR amyloidosis with polyneuropathy, administration of NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of <i>TTR</i>. (Funded by Intellia Therapeutics and Regeneron...

### CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
- Authors: Puping Liang, Yanwen Xu, Xiya Zhang, Chenhui Ding, Rui Huang, Zhen Zhang, Jie Lv, Xiaowei Xie, Yuxi Chen, Yujing Li, Ying Sun, Yaofu Bai, Zhou Songyang, Wenbin Ma, Canquan Zhou, Junjiu Huang
- Journal: Protein & Cell
- Date: 2015-04-17
- DOI: https://doi.org/10.1007/s13238-015-0153-5
- Citations: 1108
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W2141825721/llms.txt
- Abstract: Genome editing tools such as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated system (Cas) have been widely used to modify genes in model systems including animal zygotes and human cells, and hold tremendous promise for both basic research and clinical applications. T...

### CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
- Authors: H. Frangoul, D. Altshuler, M. Cappellini, Yi-Shan Chen, J. Domm, Brenda K. Eustace, J. Foell, J. de la Fuente, S. Grupp, R. Handgretinger, Tony Ho, A. Kattamis, A. Kernytsky, J. Lekstrom-Himes, Amanda M Li, F. Locatelli, M. Mapara, M. de Montalembert, D. Rondelli, Akshay Sharma, S. Sheth, S. Soni, M. Steinberg, D. Wall, Angela Yen, S. Corbacioglu
- Journal: The New England journal of medicine
- Date: 2020
- DOI: https://doi.org/10.1056/NEJMoa2031054
- Citations: 1058
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-07c69f7ddd34ca8c80618e16981e2fbdb0de6529/llms.txt
- TL;DR: Electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors was performed, with CRISPR-Cas9 targeting the BCL11A erythroid-specific enhancer, and approximately 80% of the alleles at this locus were modified, with no evidence of off-target editing.

### Delivery strategies of the CRISPR‐Cas9 gene‐editing system for therapeutic applications
- Authors: Chang Liu, Li Zhang, Hao Liu, K. Cheng
- Journal: Journal of Controlled Release
- Date: 2017
- DOI: https://doi.org/10.1016/j.jconrel.2017.09.012
- Citations: 485
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-5ee44d9fa67f9d3bd423067ca9f01ac4bb4e43c1/llms.txt
- TL;DR: The molecular mechanism and different strategies to edit genes using the CRISPR‐Cas9 system are introduced and the current systems that have been developed to deliver CRISpr‐ Cas9 in vitro and in vivo for various therapeutic purposes are highlighted.

### Off-target effects in CRISPR/Cas9 gene editing
- Authors: Congting Guo, Xiaoteng Ma, Fei Gao, Yuxuan Guo
- Journal: Frontiers in Bioengineering and Biotechnology
- Date: 2023
- DOI: https://doi.org/10.3389/fbioe.2023.1143157
- Citations: 402
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-ee3fc4548be78b7118b82051f44a3e6c4ce8ef78/llms.txt
- TL;DR: To date, many methods have been developed to nominate or detect the off-target sites of CRISPR/Cas9, which laid the basis for the successful upgrades of CRisPR/ Cas9 derivatives with enhanced precision, and about the current challenges in the management of off- target effects for future gene therapy.

### Current applications and future perspective of CRISPR/Cas9 gene editing in cancer
- Authors: Si-wei Wang, Chao Gao, Yi-Min Zheng, Li Yi, Jia-Cheng Lu, Xiao-yong Huang, Jia-Bin Cai, Peng-fei Zhang, Yuexin Cui, A. Ke
- Journal: Molecular Cancer
- Date: 2022
- DOI: https://doi.org/10.1186/s12943-022-01518-8
- Citations: 358
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-cda0679e86ce8af508a4e5b83b587d4be183cd70/llms.txt
- TL;DR: The basic principles ofCRISPR/Cas9 gene editing are explained and several new CRISPR-based gene editing modes are introduced, and the rapid progress of CRISpr screening in revealing tumorigenesis, metastasis, and drug resistance mechanisms are detailed.

### Significant enhancement of fatty acid composition in seeds of the allohexaploid, Camelina sativa, using CRISPR/Cas9 gene editing
- Authors: W. Jiang, I. Henry, Peter G. Lynagh, L. Comai, E. Cahoon, D. Weeks
- Journal: Plant Biotechnology Journal
- Date: 2017
- DOI: https://doi.org/10.1111/pbi.12663
- Citations: 302
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-6ed5821eb6473824fab4e21ecffa9e2c3dbe6246/llms.txt
- TL;DR: A CRISPR/Cas9 strategy that significantly enhanced oil composition in T3 and T4 generation Camelina seeds was associated with a combination of germ‐line mutations and somatic cell mutations in FAD2 genes in each of the three Camelina subgenomes.

### Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing
- Authors: R. Kamiński, Yilan Chen, Tracy Fischer, E. Tedaldi, A. Napoli, Yonggang Zhang, J. Karn, Wenhui Hu, K. Khalili
- Journal: Scientific Reports
- Date: 2015
- DOI: https://doi.org/10.1038/srep22555
- Citations: 295
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-8b9b236b38ce529c71a4f1aae7f3f9fa95216b1f/llms.txt
- TL;DR: Gene editing using CRISPR/Cas9 may provide a new therapeutic path for eliminating HIV-1 DNA from CD4+ T-cells and potentially serve as a novel and effective platform toward curing AIDS.

### A boronic acid–rich dendrimer with robust and unprecedented efficiency for cytosolic protein delivery and CRISPR-Cas9 gene editing
- Authors: Chongyi Liu, Tao Wan, Hui Wang, Song Zhang, Y. Ping, Yiyun Cheng
- Journal: Science Advances
- Date: 2019
- DOI: https://doi.org/10.1126/sciadv.aaw8922
- Citations: 293
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-334a60ae88d3926699991115a8ad4f632c302ed1/llms.txt
- TL;DR: The rational design of a polymer with unprecedented and robust efficiency for cytosolic delivery of native proteins and ensures efficient intracellular delivery of Cas9 protein into various cell lines and showed high efficiency in CRISPR-Cas9 genome editing.

### In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa
- Authors: Benjamin Bakondi, Wenjian Lv, Bin Lü, Melissa K. Jones, Yu-Chun Tsai, Kevin J. Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J. Breunig, Clive N. Svendsen, Shaomei Wang
- Journal: Molecular Therapy
- Date: 2015-12-15
- DOI: https://doi.org/10.1038/mt.2015.220
- Citations: 290
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W2199356015/llms.txt
- Abstract: Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of principle, we show that CRISPR/Cas9 can be used in vivo to selectively ablate the rhodopsin gene carrying the dominant S334ter...

### Optical Control of CRISPR/Cas9 Gene Editing
- Authors: James Hemphill, Erin K. Borchardt, Kalyn A. Brown, Aravind Asokan, Alexander Deiters
- Journal: Journal of the American Chemical Society
- Date: 2015-04-23
- DOI: https://doi.org/10.1021/ja512664v
- Citations: 271
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W2092493681/llms.txt
- Abstract: The CRISPR/Cas9 system has emerged as an important tool in biomedical research for a wide range of applications, with significant potential for genome engineering and gene therapy. In order to achieve conditional control of the CRISPR/Cas9 system, a genetically encoded light-activated Cas9 was engin...

### Engineering the next-generation of CAR T-cells with CRISPR-Cas9 gene editing
- Authors: Alexander J Dimitri, F. Herbst, J. Fraietta
- Journal: Molecular Cancer
- Date: 2022
- DOI: https://doi.org/10.1186/s12943-022-01559-z
- Citations: 269
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-5d923eebf9f2306e65ee75710a4468189c784dfc/llms.txt
- TL;DR: This review evaluates several of the ongoing and future directions of combining next-generation CRISPR-Cas9 gene editing with synthetic biology to optimize CAR T-cell therapy for future clinical trials toward the establishment of a new cancer treatment paradigm.

### Outbred genome sequencing and CRISPR/Cas9 gene editing in butterflies
- Authors: Xueyan Li, Dingding Fan, Wei Zhang, Guichun Liu, Lu Zhang, Li Zhao, Xiaodong Fang, Lei Chen, Yang Dong, Yuan Chen, Yun Ding, Ruoping Zhao, Mingji Feng, Yabing Zhu, Yue Feng, Xuanting Jiang, Deying Zhu, Zhonghuai Xiang, Xikan Feng, Shuai Cheng Li, Jun Wang, Guojie Zhang, Marcus R. Kronforst, Wen Wang
- Journal: Nature Communications
- Date: 2015-09-10
- DOI: https://doi.org/10.1038/ncomms9212
- Citations: 232
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W2269688815/llms.txt
- Abstract: Butterflies are exceptionally diverse but their potential as an experimental system has been limited by the difficulty of deciphering heterozygous genomes and a lack of genetic manipulation technology. Here we use a hybrid assembly approach to construct high-quality reference genomes for Papilio xut...

### Efficient oligo nucleotide mediated CRISPR-Cas9 gene editing in Aspergilli
- Authors: Christina Spuur Nødvig, Jakob Blæsbjerg Hoof, Martin E. Kogle, Zofia Dorota Jarczynska, Jan Lehmbeck, Dorte K. Klitgaard, Uffe Hasbro Mortensen
- Journal: Fungal Genetics and Biology
- Date: 2018-01-08
- DOI: https://doi.org/10.1016/j.fgb.2018.01.004
- Citations: 200
- Source: OpenAlex
- llms.txt: https://science-database.com/technology/crispr/paper/oa-W2783508188/llms.txt

### Off-target predictions in CRISPR-Cas9 gene editing using deep learning
- Authors: Jiecong Lin, Ka-chun Wong
- Journal: Bioinformatics
- Date: 2018
- DOI: https://doi.org/10.1093/bioinformatics/bty554
- Citations: 160
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-9a1edb792994e96ebdc1637dfbf365cb6c2d2ba1/llms.txt
- TL;DR: Convolutional neural network achieves the best performance on CRISPOR dataset, yielding an average classification area under the ROC curve (AUC) of 97.2% under stratified 5‐fold cross‐validation, and the deep feedforward neural network can also be competitive at the average AUC of 96.0% under the same setting.

### Spatiotemporal control of CRISPR/Cas9 gene editing
- Authors: Chenya Zhuo, Jiabin Zhang, Jung-Hwan Lee, J. Jiao, Du Cheng, Li Liu, H. Kim, Yu Tao, Mingqiang Li
- Journal: Signal Transduction and Targeted Therapy
- Date: 2021
- DOI: https://doi.org/10.1038/s41392-021-00645-w
- Citations: 151
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-111a529d67e97e8901c3175310e3ea3ffc1fefb3/llms.txt
- TL;DR: This review summarizes the latest progress of these advanced strategies involving cell-specific promoters, small-molecule activation and inhibition, bioresponsive delivery carriers, and optical/thermal/ultrasonic/magnetic activation and proposes viewpoints on directions that can be explored to further improve the spatiotemporal operability of CRISPR/Cas9.

### CRISPR/Cas9 gene editing: a new approach for overcoming drug resistance in cancer
- Authors: Mostafa Vaghari-Tabari, Parisa Hassanpour, Fatemeh Sadeghsoltani, Faezeh Malakoti, Forough Alemi, D. Qujeq, Z. Asemi, B. Yousefi
- Journal: Cellular & Molecular Biology Letters
- Date: 2022
- DOI: https://doi.org/10.1186/s11658-022-00348-2
- Citations: 103
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-edcf65933fce7aca9bcf76bb1762dcddda750704/llms.txt
- TL;DR: Studies related to the applications of this technique in overcoming drug resistance in cancer cells will be reviewed and a brief overview of the limitations of the CRISP/Cas9 gene-editing technique is given.

### Revolutionizing cancer treatment: enhancing CAR-T cell therapy with CRISPR/Cas9 gene editing technology
- Authors: Ruiyu Tao, Xiaopeng Han, Xue Bai, Jianping Yu, Youwei Ma, Weikai Chen, Dawei Zhang, Zhengkai Li
- Journal: Frontiers in Immunology
- Date: 2024
- DOI: https://doi.org/10.3389/fimmu.2024.1354825
- Citations: 66
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-2ec33a956fd6f8ee3ef3be4e3324da62b5e00375/llms.txt
- TL;DR: This review focuses on the application of CRISPR/Cas9 technology in CAR-T cell therapy, including eliminating the inhibitory effect of immune checkpoints, enhancing the ability of CAR-T cells to resist exhaustion, assisting in the construction of universal CAR-T cells, reducing the manufacturing costs of CAR-T cells, and the security problems faced.

### Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing
- Authors: Frazer H. Sinclair, Anjuman A. Begum, Charles C. Dai, I. Toth, P. Moyle
- Journal: Drug Delivery and Translational Research
- Date: 2023
- DOI: https://doi.org/10.1007/s13346-023-01320-z
- Citations: 60
- Source: Semantic Scholar
- llms.txt: https://science-database.com/technology/crispr/paper/s2-2777c265653e7b1ad7a36fd5d00c0e9590527407/llms.txt
- TL;DR: The molecular mechanisms of the CRISpr/Cas9 gene editing system are introduced, current strategies for delivering CRISPR/ Cas9-based tools, an overview of strategies for overcoming off-target genome editing, and approaches for improving genome targeting and tissue targeting are highlighted.

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