CRISPR Journal最新文献_第2页

Affordable Pricing of CRISPR Treatments is a Pressing Ethical Imperative. CRISPR 治疗的平价化是迫在眉睫的伦理当务之急。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-10-01 Epub Date: 2024-10-10 DOI: 10.1089/crispr.2024.0042

Jon Rueda, Íñigo de Miguel Beriain, Lluis Montoliu

引用次数: 0

Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for The CRISPR Journal. 罗莎琳德-富兰克林学会自豪地宣布《CRISPR 期刊》2023 年度获奖者。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.55675.rfs2023

Suchita Nety

引用次数: 0

Enrichment of Allelic Editing Outcomes by Prime Editing in Induced Pluripotent Stem Cells. 通过在诱导多能干细胞中进行基质编辑，丰富等位基因编辑结果。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.0028

Ryo Niwa, Tomoko Matsumoto, Alexander Y Liu, Maki Kawato, Takayuki Kondo, Kayoko Tsukita, Peter Gee, Haruhisa Inoue, Thomas L Maurissen, Knut Woltjen

引用次数: 0

Widespread Impact of Natural Genetic Variations in CRISPR-Cas9 Outcomes. 自然基因变异对 CRISPR-Cas9 结果的广泛影响。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.0020

Victoria R Li, Tinghui Wu, Alicja Tadych, Aaron Wong, Zijun Zhang

{"title":"Widespread Impact of Natural Genetic Variations in CRISPR-Cas9 Outcomes.","authors":"Victoria R Li, Tinghui Wu, Alicja Tadych, Aaron Wong, Zijun Zhang","doi":"10.1089/crispr.2024.0020","DOIUrl":"10.1089/crispr.2024.0020","url":null,"abstract":"The clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) is a genome editing tool widely used in biological research and clinical therapeutics. Natural human genetic variations, through altering the sequence context of CRISPR-Cas9 target regions, can significantly affect its DNA repair outcomes and ultimately lead to different editing efficiencies. However, these effects have not been systematically studied, even as CRISPR-Cas9 is broadly applied to primary cells and patient samples that harbor such genetic diversity. Here, we present comprehensive investigations of natural genetic variations on CRISPR-Cas9 outcomes across the human genome. The utility of our analysis is illustrated in two case studies, on both preclinical discoveries of CD33 knockout in chimeric antigen receptor-T cell therapy and clinical applications of transthyretin (TTR) inactivation for treating TTR amyloidosis. We further expand our analysis to genome-scale, population-stratified common variants that may lead to gene editing disparity. Our analyses demonstrate pitfalls of failing to account for the widespread genetic variations in Cas9 target selection and how they can be effectively examined and avoided using our method. To facilitate broad access to our analysis, a web platform CROTONdb is developed, which provides predictions for all possible CRISPR-Cas9 target sites in the coding and noncoding regulatory regions, spanning over 5.38 million guide RNA targets and 90.82 million estimated variant effects. We anticipate CROTONdb having broad clinical utilities in gene and cellular therapies.","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":null,"pages":null},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Repurposing an Endogenous CRISPR-Cas System to Generate and Study Subtle Mutations in Bacteriophages. 重新利用内源性 CRISPR-Cas 系统，生成并研究噬菌体中的微妙突变。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-09-30 DOI: 10.1089/crispr.2024.0047

Kotaro Kamata, Nils Birkholz, Marijn Ceelen, Robert D Fagerlund, Simon A Jackson, Peter C Fineran

{"title":"Repurposing an Endogenous CRISPR-Cas System to Generate and Study Subtle Mutations in Bacteriophages.","authors":"Kotaro Kamata, Nils Birkholz, Marijn Ceelen, Robert D Fagerlund, Simon A Jackson, Peter C Fineran","doi":"10.1089/crispr.2024.0047","DOIUrl":"https://doi.org/10.1089/crispr.2024.0047","url":null,"abstract":"While bacteriophage applications benefit from effective phage engineering, selecting the desired genotype after subtle modifications remains challenging. Here, we describe a two-phase endogenous CRISPR-Cas-based phage engineering approach that enables selection of small defined edits in Pectobacterium carotovorum phage ZF40. We designed plasmids containing sequences homologous to ZF40 and a mini-CRISPR array. The plasmids allowed genome editing through homologous recombination and counter-selection against non-recombinant phage genomes using an endogenous type I-E CRISPR-Cas system. With this technique, we first deleted target genes and subsequently restored loci with modifications. This two-phase approach circumvented major challenges in subtle phage modifications, including inadequate sequence distinction for CRISPR-Cas counter-selection and the requirement of a protospacer-adjacent motif, limiting sequences that can be modified. Distinct 20-bp barcodes were incorporated through engineering as differential target sites for programmed CRISPR-Cas activity, which allowed quantification of phage variants in mixed populations. This method aids studies and applications that require mixtures of similar phages.","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":null,"pages":null},"PeriodicalIF":3.7,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Identification of a Guide RNA Targeting an Ultraconserved Element for Evaluation of Cas9 Genome Editors Across Mammalian Species. 鉴定靶向超保守元件的引导核糖核酸，以评估跨哺乳动物物种的 Cas9 基因组编辑器。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-09-23 DOI: 10.1089/crispr.2024.0053

Benjamin G Gowen, Prachi Khekare, Shannon R McCawley, Kory Melton, Craig Soares, Jean Chan, Vihasi Jani, Pierre Boivin, Ashil Bans, Weng-In Leong, Aaron J Cantor, Jack Walleshauser, Peter B Otoupal, Rina J Mepani, Adam P Silverman, Mary Haak-Frendscho, Spencer C Wei

引用次数: 0

Challenging the Boundaries Between Treatment, Prevention, and Enhancement in Human Genome Editing. 挑战人类基因组编辑中治疗、预防和增强之间的界限。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-08-01 Epub Date: 2024-07-08 DOI: 10.1089/crispr.2024.0021

Margaret Waltz, Rebecca L Walker, Michael A Flatt, Douglas MacKay, John M Conley, Eric T Juengst, R Jean Cadigan

{"title":"Challenging the Boundaries Between Treatment, Prevention, and Enhancement in Human Genome Editing.","authors":"Margaret Waltz, Rebecca L Walker, Michael A Flatt, Douglas MacKay, John M Conley, Eric T Juengst, R Jean Cadigan","doi":"10.1089/crispr.2024.0021","DOIUrl":"10.1089/crispr.2024.0021","url":null,"abstract":"Traditional distinctions between treatment and enhancement goals for human genome editing (HGE) have animated oversight considerations, yet these categories have been complicated by the addition of prevention as a possible target for HGE applications. To assess the role these three categories might play in continued HGE governance efforts, we report on interviews with genome editing scientists and governance group members. While some accepted traditional distinctions between treatment and enhancement and rejected the latter as unacceptable, others argued that the concept of enhancement is largely irrelevant or not as morally problematic as suggested. Others described how preventive goals for HGE create gray zones where prevention and enhancement may be difficult to distinguish, which may stymie uses of HGE. We conclude by discussing the governance implications of these various understandings of treatment, prevention, and enhancement as HGE research moves beyond the treatment of serious disease to embrace longer range preventive goals.","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":null,"pages":null},"PeriodicalIF":3.7,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11386990/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141560350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

AI and SynBio Meet CRISPR Heralding a New Genome Editing Era. 人工智能和 SynBio 与 CRISPR 相遇，预示着一个新的基因组编辑时代的到来。

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-08-01 DOI: 10.1089/crispr.2024.0063

Rodolphe Barrangou

引用次数: 0

Prime Editing of Vascular Endothelial Growth Factor Receptor 2 Attenuates Angiogenesis In Vitro. 血管内皮生长因子受体 2 的基因编辑抑制体外血管生成

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-08-01 Epub Date: 2024-08-07 DOI: 10.1089/crispr.2024.0019

Gaoen Ma, Hui Qi, Hongwei Deng, Lijun Dong, Qing Zhang, Junkai Ma, Yanhui Yang, Xiaohe Yan, Yajian Duan, Hetian Lei

引用次数: 0

Expanding the Genome-Editing Toolbox with Abyssicoccus albus Cas9 Using a Unique Protospacer Adjacent Motif Sequence. 利用独特的原位相邻位点序列扩展 Abyssicoccus albus Cas9 的基因组编辑工具箱

IF 3.7 4区生物学

CRISPR Journal Pub Date : 2024-08-01 Epub Date: 2024-08-07 DOI: 10.1089/crispr.2024.0013

Akiyoshi Nakamura, Hiroshi Yamamoto, Tsubasa Yano, Reika Hasegawa, Yoichi Makino, Nobutaka Mitsuda, Teruhiko Terakawa, Seiichiro Ito, Shigeo S Sugano

{"title":"Expanding the Genome-Editing Toolbox with Abyssicoccus albus Cas9 Using a Unique Protospacer Adjacent Motif Sequence.","authors":"Akiyoshi Nakamura, Hiroshi Yamamoto, Tsubasa Yano, Reika Hasegawa, Yoichi Makino, Nobutaka Mitsuda, Teruhiko Terakawa, Seiichiro Ito, Shigeo S Sugano","doi":"10.1089/crispr.2024.0013","DOIUrl":"10.1089/crispr.2024.0013","url":null,"abstract":"The genome-editing efficiency of the CRISPR-Cas9 system hinges on the recognition of the protospacer adjacent motif (PAM) sequence, which is essential for Cas9 binding to DNA. The commonly used Streptococcus pyogenes (SpyCas9) targets the 5'-NGG-3' PAM sequence, which does not cover all the potential genomic-editing sites. To expand the toolbox for genome editing, SpyCas9 has been engineered to recognize flexible PAM sequences and Cas9 orthologs have been used to recognize novel PAM sequences. In this study, Abyssicoccus albus Cas9 (AalCas9, 1059 aa), which is smaller than SpyCas9, was found to recognize a unique 5'-NNACR-3' PAM sequence. Modification of the guide RNA sequence improved the efficiency of AalCas9-mediated genome editing in both plant and human cells. Predicted structure-assisted introduction of a point mutation in the putative PAM recognition site shifted the sequence preference of AalCas9. These results provide insights into Cas9 diversity and novel tools for genome editing.","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":null,"pages":null},"PeriodicalIF":3.7,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141903602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0