--- title: "CRISPR Gene Editing" slug: "crispr" discipline: "Biology / Genetics" description: "CRISPR-Cas9 and related gene editing technologies for precision genome modification. Covers therapeutic applications, agricultural biotech, gene drives, and ethical considerations." icon: "šŸ§¬" url: "https://science-database.com/technology/crispr" api: "https://science-database.com/api/v1/technology/crispr" llms_txt: "https://science-database.com/technology/crispr/llms.txt" articles_indexed: 15 last_updated: "2026-09-08T05:19:16.679Z" search_terms: - "CRISPR Cas9 gene editing" - "CRISPR therapeutics genome" - "CRISPR base editing prime editing" source: "science-database.com" license: "metadata CC0, abstracts belong to respective publishers" --- # CRISPR Gene Editing CRISPR-Cas9 and related gene editing technologies for precision genome modification. Covers therapeutic applications, agricultural biotech, gene drives, and ethical considerations. **Discipline:** Biology / Genetics **Indexed Papers:** 15 **Last Updated:** 2026-09-08 ## Top Publications Ranked by citation impact across Semantic Scholar, OpenAlex & arXiv. ### Genome engineering using the CRISPR-Cas9 system - **Authors:** F. Ann Ran, Patrick D. Hsu, Jason Wright, Vineeta Agarwala, David Scott, Feng Zhang - **Journal:** Nature Protocols - **Published:** 2013-10-24 - **DOI:** [10.1038/nprot.2013.143](https://doi.org/10.1038/nprot.2013.143) - **Citations:** 11,914 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nature.com/articles/nprot.2013.143.pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W1977709885/llms.txt) ### CRISPR-Cas9 Gene Editing for Sickle Cell Disease and Ī²-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 - **Published:** 2020-12-05 - **DOI:** [10.1056/nejmoa2031054](https://doi.org/10.1056/nejmoa2031054) - **Citations:** 1,936 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nejm.org/doi/pdf/10.1056/NEJMoa2031054?articleTools=true) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W3112179900/llms.txt) > Summ a r yTransfusion-dependent Ī²-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 Ī³-globin expression and fetal hemoglobin in erythroid cells.We performed electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors, with CRISPR-... ### Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers - **Authors:** Isaac B. Hilton, Anthony Dā€™Ippolito, Christopher M. Vockley, Pratiksha I. Thakore, Gregory E. Crawford, Timothy E. Reddy, Charles A. Gersbach - **Journal:** Nature Biotechnology - **Published:** 2015-04-06 - **DOI:** [10.1038/nbt.3199](https://doi.org/10.1038/nbt.3199) - **Citations:** 1,914 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.ncbi.nlm.nih.gov/pmc/articles/4430400) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2066783444/llms.txt) ### CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis - **Authors:** Julian D. Gillmore, Ed Gane, Jƶrg TƤubel, Justin Kao, Marianna Fontana, Michael L. Maitland, Jessica Seitzer, Daniel J. Oā€™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 Seppā€Lorenzino, David Lebwohl - **Journal:** New England Journal of Medicine - **Published:** 2021-06-26 - **DOI:** [10.1056/nejmoa2107454](https://doi.org/10.1056/nejmoa2107454) - **Citations:** 1,726 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nejm.org/doi/pdf/10.1056/NEJMoa2107454?articleTools=true) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W3177445623/llms.txt) > BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a singl... ### 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 - **Published:** 2015-04-16 - **DOI:** [10.1007/s13238-015-0153-5](https://doi.org/10.1007/s13238-015-0153-5) - **Citations:** 1,132 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://link.springer.com/content/pdf/10.1007/s13238-015-0153-5.pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2141825721/llms.txt) > 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. To date, a serious knowledge gap remains in our understanding of DNA repair mechanisms in human early... ### Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications - **Authors:** Chang Liu, Li Zhang, Hao Liu, Kun Cheng - **Journal:** Journal of Controlled Release - **Published:** 2017-09-11 - **DOI:** [10.1016/j.jconrel.2017.09.012](https://doi.org/10.1016/j.jconrel.2017.09.012) - **Citations:** 629 - **Source:** OpenAlex - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2755653587/llms.txt) ### Off-target effects in CRISPR/Cas9 gene editing - **Authors:** Congting Guo, Xiaoteng Ma, Fei Gao, Yuxuan Guo - **Journal:** Frontiers in Bioengineering and Biotechnology - **Published:** 2023-03-09 - **DOI:** [10.3389/fbioe.2023.1143157](https://doi.org/10.3389/fbioe.2023.1143157) - **Citations:** 596 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.frontiersin.org/articles/10.3389/fbioe.2023.1143157/pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W4323666400/llms.txt) > Gene editing stands for the methods to precisely make changes to a specific nucleic acid sequence. With the recent development of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system, gene editing has become efficient, convenient and programmable, leading to promising translational studies and clinical trials for both genetic and non-genetic diseases. A major concern ... ### Current applications and future perspective of CRISPR/Cas9 gene editing in cancer - **Authors:** Siwei Wang, Chao Gao, Yi-Min Zheng, Yi Li, Jiaā€Cheng Lu, Xiaoyong Huang, Jiabin Cai, Pengfei Zhang, Yuehong Cui, Aiā€Wu Ke - **Journal:** Molecular Cancer - **Published:** 2022-02-21 - **DOI:** [10.1186/s12943-022-01518-8](https://doi.org/10.1186/s12943-022-01518-8) - **Citations:** 466 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://molecular-cancer.biomedcentral.com/track/pdf/10.1186/s12943-022-01518-8) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W4213070361/llms.txt) > Clustered regularly interspaced short palindromic repeats (CRISPR) system provides adaptive immunity against plasmids and phages in prokaryotes. This system inspires the development of a powerful genome engineering tool, the CRISPR/CRISPR-associated nuclease 9 (CRISPR/Cas9) genome editing system. Due to its high efficiency and precision, the CRISPR/Cas9 technique has been employed to explore the f... ### A boronic acidā€“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, Ping Yuan, Yiyun Cheng - **Journal:** Science Advances - **Published:** 2019-06-01 - **DOI:** [10.1126/sciadv.aaw8922](https://doi.org/10.1126/sciadv.aaw8922) - **Citations:** 390 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://doi.org/10.1126/sciadv.aaw8922) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2951794613/llms.txt) > Cytosolic protein delivery is of central importance for the development of protein-based biotechnologies and therapeutics; however, efficient intracellular delivery of native proteins remains a challenge. Here, we reported a boronic acid-rich dendrimer with unprecedented efficiency for cytosolic delivery of native proteins. The dendrimer could bind with both negatively and positively charged prote... ### Significant enhancement of fatty acid composition in seeds of the allohexaploid, Camelina sativa , using CRISPR /Cas9 gene editing - **Authors:** Wen Zhi Jiang, Isabelle Henry, Peter G. Lynagh, Luca Comai, Edgar B. Cahoon, Donald P. Weeks - **Journal:** Plant Biotechnology Journal - **Published:** 2016-11-14 - **DOI:** [10.1111/pbi.12663](https://doi.org/10.1111/pbi.12663) - **Citations:** 360 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://onlinelibrary.wiley.com/doi/pdfdirect/10.1111/pbi.12663) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2549285722/llms.txt) > Summary The CRISPR /Cas9 nuclease system is a powerful and flexible tool for genome editing, and novel applications of this system are being developed rapidly. Here, we used CRISPR /Cas9 to target the FAD 2 gene in Arabidopsis thaliana and in the closely related emerging oil seed plant, Camelina sativa, with the goal of improving seed oil composition. We successfully obtained Camelina seeds in whi... ### Engineering the next-generation of CAR T-cells with CRISPR-Cas9 gene editing - **Authors:** Alexander J. Dimitri, Friederike Herbst, Joseph A. Fraietta - **Journal:** Molecular Cancer - **Published:** 2022-03-18 - **DOI:** [10.1186/s12943-022-01559-z](https://doi.org/10.1186/s12943-022-01559-z) - **Citations:** 347 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://molecular-cancer.biomedcentral.com/track/pdf/10.1186/s12943-022-01559-z) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W4220766750/llms.txt) > Chimeric Antigen Receptor (CAR) T-cells represent a breakthrough in personalized cancer therapy. In this strategy, synthetic receptors comprised of antigen recognition, signaling, and costimulatory domains are used to reprogram T-cells to target tumor cells for destruction. Despite the success of this approach in refractory B-cell malignancies, optimal potency of CAR T-cell therapy for many other ... ### Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing - **Authors:** Rafal Kaminski, Yilan Chen, Tracy Fischer, Ellen Tedaldi, Alessandro Napoli, Yonggang Zhang, Jonathan Karn, Wenhui Hu, Kamel Khalili - **Journal:** Scientific Reports - **Published:** 2016-03-04 - **DOI:** [10.1038/srep22555](https://doi.org/10.1038/srep22555) - **Citations:** 324 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nature.com/articles/srep22555.pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2291052616/llms.txt) > We employed an RNA-guided CRISPR/Cas9 DNA editing system to precisely remove the entire HIV-1 genome spanning between 5' and 3' LTRs of integrated HIV-1 proviral DNA copies from latently infected human CD4+ T-cells. Comprehensive assessment of whole-genome sequencing of HIV-1 eradicated cells ruled out any off-target effects by our CRISPR/Cas9 technology that might compromise the integrity of the ... ### 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 - **Published:** 2015-04-23 - **DOI:** [10.1021/ja512664v](https://doi.org/10.1021/ja512664v) - **Citations:** 276 - **Source:** OpenAlex - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2092493681/llms.txt) > 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 engineered through the site-specific installation of a caged lysine amino acid. Several potential lysine ... ### 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, Hui Xiang, Xikan Feng, Shuai Cheng Li, Jun Wang, Guojie Zhang, Marcus R. Kronforst, Wen Wang - **Journal:** Nature Communications - **Published:** 2015-09-10 - **DOI:** [10.1038/ncomms9212](https://doi.org/10.1038/ncomms9212) - **Citations:** 234 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nature.com/articles/ncomms9212.pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W2269688815/llms.txt) > 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 xuthus (contig and scaffold N50: 492 kb, 3.4 Mb) and Papilio machaon (contig and scaffold N50: 81 kb, 1... ### Spatiotemporal control of CRISPR/Cas9 gene editing - **Authors:** Chenya Zhuo, Jiabin Zhang, Jungā€Hwan Lee, Ju Jiao, Du Cheng, Li Liu, Haeā€Won Kim, Yu Tao, Mingqiang Li - **Journal:** Signal Transduction and Targeted Therapy - **Published:** 2021-06-20 - **DOI:** [10.1038/s41392-021-00645-w](https://doi.org/10.1038/s41392-021-00645-w) - **Citations:** 187 - **Source:** OpenAlex - **Access:** Open Access - **PDF:** [Download](https://www.nature.com/articles/s41392-021-00645-w.pdf) - **llms.txt:** [View](https://science-database.com/technology/crispr/paper/oa-W3177233526/llms.txt) > The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) gene editing technology, as a revolutionary breakthrough in genetic engineering, offers a promising platform to improve the treatment of various genetic and infectious diseases because of its simple design and powerful ability to edit different loci simultaneously. However, failure to conduct ... --- *Generated by [science-database.com](https://science-database.com) ā€” The Knowledge Interface* *Full data available via [JSON API](https://science-database.com/api/v1/technology/crispr)*