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Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for The CRISPR Journal. 罗莎琳德·富兰克林协会自豪地宣布了2023年CRISPR杂志的获奖者。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.55675.rfs2023
Suchita Nety
{"title":"Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for <i>The CRISPR Journal</i>.","authors":"Suchita Nety","doi":"10.1089/crispr.2024.55675.rfs2023","DOIUrl":"https://doi.org/10.1089/crispr.2024.55675.rfs2023","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"211"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142808608","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
Five Years of Progress in CRISPR Clinical Trials (2019-2024). CRISPR 临床试验的五年进展（2019-2024 年）。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.0081
Kevin Davies, Alex Philippidis, Rodolphe Barrangou
{"title":"Five Years of Progress in CRISPR Clinical Trials (2019-2024).","authors":"Kevin Davies, Alex Philippidis, Rodolphe Barrangou","doi":"10.1089/crispr.2024.0081","DOIUrl":"10.1089/crispr.2024.0081","url":null,"abstract":"<p><p>In July 2019, Victoria Gray became the first patient with sickle cell disease to receive a CRISPR-based cell therapy as a volunteer in the exa-cel clinical trial, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics. Barely four years later, the ensuing therapy, branded as Casgevy, received approval from regulatory agencies in Europe, the United States, and the Middle East, ushering in a new era of CRISPR-based medicines. During this period, scores of other clinical trials have been launched, including many actively recruiting patients across phase 1, phase 2, and phase 3 clinical trials around the world. In this brief Perspective, we collate the latest information on therapeutic clinical trials featuring CRISPR, base and prime editing, across a range of both <i>in vivo</i> and <i>ex vivo</i> gene and cell therapies.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"227-230"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480628","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
Characterization of Research Support of Genome Editing Technologies and Transition to Clinical Trials. 基因组编辑技术研究支持的特点以及向临床试验的过渡。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 Epub Date: 2024-09-26 DOI: 10.1089/crispr.2024.0011
Riya Mohan, Susanne B Haga
{"title":"Characterization of Research Support of Genome Editing Technologies and Transition to Clinical Trials.","authors":"Riya Mohan, Susanne B Haga","doi":"10.1089/crispr.2024.0011","DOIUrl":"10.1089/crispr.2024.0011","url":null,"abstract":"<p><p>Genome editing technologies have become widely used research tools. To assess the rate of growth with respect to federal funding of gene editing projects, we analyzed publicly available data retrieved from the NIH RePORTER and Clinicaltrials.gov databases. We identified 6,111 awards between 1977 and 2023, the majority being extramural, investigator-driven R (noneducational) awards (66.7%). There was an average growth rate of 40% between 2008 and 2022, and the biggest increase in awards was observed between 2017 and 2018 (doubling from 140 to 280). Five administering institutes/centers accounted for more than 60% of awards with the highest number of awards from the National Cancer Institute (20.0%). The majority of clinical trials involving some type of genome editing (75%) started in or after 2020. This analysis illuminates the rapid and widespread growth of gene editing research across disciplines and the eventual launch of clinical trials using gene editing tools.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"249-257"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332257","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
Genome Editing Therapy for the Blood: Ex Vivo Success and In Vivo Prospects. 血液基因组编辑疗法：体内成功与体内前景。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 Epub Date: 2024-09-26 DOI: 10.1089/crispr.2024.0036
Christy A George, Srishti U Sahu, Lorena de Oñate, Bruno Solano de Freitas Souza, Ross C Wilson
{"title":"Genome Editing Therapy for the Blood: <i>Ex Vivo</i> Success and <i>In Vivo</i> Prospects.","authors":"Christy A George, Srishti U Sahu, Lorena de Oñate, Bruno Solano de Freitas Souza, Ross C Wilson","doi":"10.1089/crispr.2024.0036","DOIUrl":"10.1089/crispr.2024.0036","url":null,"abstract":"<p><p>Hematopoietic stem cells (HSCs) provide the body with a continuous supply of healthy, functional blood cells. In patients with hematopoietic malignancies, immunodeficiencies, lysosomal storage disorders, and hemoglobinopathies, therapeutic genome editing offers hope for corrective intervention, with even modest editing efficiencies likely to provide clinical benefit. Engineered white blood cells, such as T cells, can be applied therapeutically to address monogenic disorders of the immune system, HIV infection, or cancer. The versatility of CRISPR-based tools allows countless new medical interventions for diseases of the blood, and rapid <i>ex vivo</i> success has been demonstrated in hemoglobinopathies via transplantation of the patient's HSCs following genome editing in a laboratory setting. Here we review recent advances in therapeutic genome editing of HSCs and T cells, focusing on the progress in <i>ex vivo</i> contexts, the promise of improved access via <i>in vivo</i> delivery, as well as the ongoing preclinical efforts that may enable the transition from <i>ex vivo</i> to <i>in vivo</i> administration. We discuss the challenges, limitations, and future prospects of this rapidly developing field, which may one day establish CRISPR as the standard of care for some diseases affecting the blood.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"231-248"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142332258","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
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
{"title":"Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for <i>The CRISPR Journal</i>.","authors":"Suchita Nety","doi":"10.1089/crispr.2024.55675.rfs2023","DOIUrl":"10.1089/crispr.2024.55675.rfs2023","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"211"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142669659","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
Give Cas a Chance: An Actionable Path to a Platform for CRISPR Cures. 给 Cas 一个机会：通往 CRISPR 治疗平台的可行之路。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.0082
Fyodor D Urnov
{"title":"Give Cas a Chance: An Actionable Path to a Platform for CRISPR Cures.","authors":"Fyodor D Urnov","doi":"10.1089/crispr.2024.0082","DOIUrl":"10.1089/crispr.2024.0082","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"212-219"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480629","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
Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the ELANE Gene. 比较针对 ELANE 基因中导致严重先天性中性粒细胞减少症突变的基因编辑方法
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2024-10-01 DOI: 10.1089/crispr.2024.0006
Malte Ulrich Ritter, Masoud Nasri, Benjamin Dannenmann, Perihan Mir, Benjamin Secker, Diana Amend, Maksim Klimiankou, Karl Welte, Julia Skokowa
{"title":"Comparison of Gene-Editing Approaches for Severe Congenital Neutropenia-Causing Mutations in the <i>ELANE</i> Gene.","authors":"Malte Ulrich Ritter, Masoud Nasri, Benjamin Dannenmann, Perihan Mir, Benjamin Secker, Diana Amend, Maksim Klimiankou, Karl Welte, Julia Skokowa","doi":"10.1089/crispr.2024.0006","DOIUrl":"10.1089/crispr.2024.0006","url":null,"abstract":"<p><p>Safety considerations for gene therapies of inherited preleukemia syndromes, including severe congenital neutropenia (CN), are paramount. We compared several strategies for CRISPR/Cas9 gene editing of autosomal-dominant <i>ELANE</i> mutations in CD34<sup>+</sup> cells from two CN patients head-to-head. We tested universal and allele-specific <i>ELANE</i> knockout, <i>ELANE</i> mutation correction by homology-directed repair (HDR) with AAV6, and allele-specific HDR with ssODN. All strategies were not toxic, had at least 30% editing, and rescued granulopoiesis <i>in vitro</i>. In contrast to published data, allele-specific indels in the last exon of <i>ELANE</i> also restored granulopoiesis. Moreover, by implementing patient-derived induced pluripotent stem cells for GUIDE-Seq off-target analysis, we established a clinically relevant \"personalized\" assessment of off-target activity of gene editing on the background of the patient's genome. We found that allele-specific approaches had the most favorable off-target profiles. Taken together, a well-defined head-to-head comparison pipeline for selecting the appropriate gene therapy is essential for diseases, with several gene editing strategies available.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"258-271"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480625","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
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
{"title":"Affordable Pricing of CRISPR Treatments is a Pressing Ethical Imperative.","authors":"Jon Rueda, Íñigo de Miguel Beriain, Lluis Montoliu","doi":"10.1089/crispr.2024.0042","DOIUrl":"10.1089/crispr.2024.0042","url":null,"abstract":"<p><p>Casgevy, the world's first approved CRISPR-based cell therapy, has been priced at $2.2 million per patient. Although this hefty price tag was widely anticipated, the extremely high cost of this and other cell and gene therapies poses a major ethical issue in terms of equitable access and global health. In this Perspective, we argue that lowering the prices of future CRISPR therapies is an urgent ethical imperative. Although we focus on Casgevy as a case study, much of our analysis can be extrapolated to the controversies over affordable access to other gene and cell therapies. First, we explain why this first-of-its-kind CRISPR therapy might be so expensive. We then analyze the ethical issues of equity and global health of early CRISPR treatments. Next, we discuss potential solutions to lower the prices of CRISPR gene therapies. We conclude that the approval of CRISPR transforms our obligations of justice and compels us to bring future gene therapies to the maximum possible number of patients with serious genetic diseases at affordable prices.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"220-226"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401956","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
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
{"title":"Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for <i>The CRISPR Journal</i>.","authors":"Suchita Nety","doi":"10.1089/crispr.2024.55675.rfs2023","DOIUrl":"https://doi.org/10.1089/crispr.2024.55675.rfs2023","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"211"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480630","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
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
{"title":"Enrichment of Allelic Editing Outcomes by Prime Editing in Induced Pluripotent Stem Cells.","authors":"Ryo Niwa, Tomoko Matsumoto, Alexander Y Liu, Maki Kawato, Takayuki Kondo, Kayoko Tsukita, Peter Gee, Haruhisa Inoue, Thomas L Maurissen, Knut Woltjen","doi":"10.1089/crispr.2024.0028","DOIUrl":"10.1089/crispr.2024.0028","url":null,"abstract":"<p><p>Gene editing in human induced pluripotent stem (iPS) cells with programmable nucleases facilitates reliable disease models, but methods using double-strand break repair often produce random on-target by-products. Prime editing (PE) combines Cas9 nickase with reverse transcriptase and PE guide RNA (pegRNA) encoding a repair template to reduce by-products. We implemented a GMP-compatible protocol for transfecting Cas9- or PE-2A-mCherry plasmids to track and fractionate human iPS cells based on PE expression level. We compared the editing outcomes of Cas9- and PE-based methods in a GFP-to-BFP conversion assay at the <i>HEK3</i> benchmark locus and at the <i>APOE</i> Alzheimer's risk locus, revealing superior precision of PE at high expression levels. Moreover, sorting cells for PE expression level influenced allelic editing outcomes at the target loci. We expect that our findings will aid in the creation of gene-edited human iPS cells with intentional heterozygous and homozygous genotypes.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"7 5","pages":"293-304"},"PeriodicalIF":3.7,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480627","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
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