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Advances in Gene Therapy for Sickle Cell Disease: From Preclinical Innovations to Clinical Implementation and Access Challenges. 镰状细胞病基因治疗的进展：从临床前创新到临床实施和获取挑战。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 Epub Date: 2025-05-12 DOI: 10.1089/crispr.2024.0101
Henna Butt, Mamatha Mandava, David Jacobsohn
{"title":"Advances in Gene Therapy for Sickle Cell Disease: From Preclinical Innovations to Clinical Implementation and Access Challenges.","authors":"Henna Butt, Mamatha Mandava, David Jacobsohn","doi":"10.1089/crispr.2024.0101","DOIUrl":"10.1089/crispr.2024.0101","url":null,"abstract":"<p><p>Sickle cell disease (SCD) is a hereditary blood disorder caused by a specific mutation in the β-globin gene, leading to the production of hemoglobin S, which deforms red blood cells, causing occlusion in small blood vessels. This results in pain, anemia, organ damage, infections, and increased stroke risk. Treatment options, including disease-modifying therapies and curative hematopoietic stem cell transplants, have limited accessibility. Recently, autologous gene therapy has emerged as a promising curative option, particularly for SCD. Gene editing techniques such as CRISPR, base editing, and prime editing offer potential to correct this mutation. In this review, we discuss recent preclinical studies and clinical trials of gene and cell therapies, focusing on the progress of FDA-approved treatments like Lyfgenia and Casgevy. We also examine the many challenges, including accessibility, safety, and long-term efficacy, which continue to shape the future of SCD gene therapy.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"174-188"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008525","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
CRISPR2025 New Zealand: Innovation and Collaboration. CRISPR2025新西兰：创新与合作。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 Epub Date: 2025-05-29 DOI: 10.1089/crispr.2025.0026
Katharina G Wandera, Jeremy Dubrulle, Russell Greene, Meric Ozturk, Gavin Knott, Dipali G Sashital, Peter C Fineran
{"title":"CRISPR2025 New Zealand: Innovation and Collaboration.","authors":"Katharina G Wandera, Jeremy Dubrulle, Russell Greene, Meric Ozturk, Gavin Knott, Dipali G Sashital, Peter C Fineran","doi":"10.1089/crispr.2025.0026","DOIUrl":"10.1089/crispr.2025.0026","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"166-173"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144163702","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
Metagenome-Derived CRISPR-Cas12a Mining and Characterization. 宏基因组衍生的CRISPR-Cas12a挖掘和表征。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 Epub Date: 2025-05-21 DOI: 10.1089/crispr.2024.0099
Kalani Gast, Sydney Baker, Adair L Borges, Stephanie Ward, Jillian F Banfield, Rodolphe Barrangou
{"title":"Metagenome-Derived CRISPR-Cas12a Mining and Characterization.","authors":"Kalani Gast, Sydney Baker, Adair L Borges, Stephanie Ward, Jillian F Banfield, Rodolphe Barrangou","doi":"10.1089/crispr.2024.0099","DOIUrl":"10.1089/crispr.2024.0099","url":null,"abstract":"<p><p>The advent of clustered regularly interspaced short palindromic repeats (CRISPR)-based technologies has revolutionized genome editing, with continued interest in expanding the CRISPR-associated proteins (Cas) toolbox with diverse, efficient, and specific effectors. CRISPR-Cas12a is a potent, programmable RNA-guided dual nickase, broadly used for genome editing. Here, we mined dairy cow microbial metagenomes for CRISPR-Cas systems, unraveling novel Cas12a enzymes. Using <i>in silico</i> pipelines, we characterized and predicted key drivers of CRISPR-Cas12a activity, encompassing guides and protospacer adjacent motifs for five systems. We next assessed their functional potential in cell-free transcription-translation assays with GFP-based fluorescence readouts. Lastly, we determined their genome editing potential <i>in vivo</i> in <i>Escherichia coli</i> by generating 1 kb knockouts. Unexpectedly, we observed natural sequence variation in the bridge-helix domain of the best-performing candidate and used mutagenesis to alter the activity of Cas12a orthologs, resulting in increased gene editing capabilities of a relatively inefficient candidate. This study illustrates the potential of underexplored metagenomic sequence diversity for the development and refinement of genome editing effectors.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"189-204"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144121294","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
Facing Systemic Uncertainty, Can the CRISPR Community Stay the Course? 面对系统的不确定性，CRISPR社区能坚持到底吗？
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 DOI: 10.1089/crispr.2025.0050
Rodolphe Barrangou
{"title":"Facing Systemic Uncertainty, Can the CRISPR Community Stay the Course?","authors":"Rodolphe Barrangou","doi":"10.1089/crispr.2025.0050","DOIUrl":"https://doi.org/10.1089/crispr.2025.0050","url":null,"abstract":"","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":"8 3","pages":"165"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144276658","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
Green Listed v2.0: A Web Application for Streamlined Design of Custom CRISPR Screens. 绿色列表v2.0：自定义CRISPR屏幕的流线型设计的Web应用程序。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 Epub Date: 2025-05-07 DOI: 10.1089/crispr.2025.0023
Esbjörn Henkel, Zhaojun Li, Daniel Uvehag, Bernhard Schmierer, Martin Henkel, Fredrik Wermeling
{"title":"Green Listed v2.0: A Web Application for Streamlined Design of Custom CRISPR Screens.","authors":"Esbjörn Henkel, Zhaojun Li, Daniel Uvehag, Bernhard Schmierer, Martin Henkel, Fredrik Wermeling","doi":"10.1089/crispr.2025.0023","DOIUrl":"10.1089/crispr.2025.0023","url":null,"abstract":"<p><p>Custom CRISPR screens are powerful tools for rapid, hypothesis-driven discovery, but their design is often complex and time-consuming. Green Listed v2.0 simplifies this process with an intuitive workflow for designing custom CRISPR spacer libraries and supports downstream analysis for all users, irrespective of their computational experience. The web application features a user-friendly graphical interface freely accessible at https://greenlisted.cmm.se. Version 2.0 includes significant upgrades to the original 2016 version that were implemented based on user feedback. This includes a new gene synonym tool, expanded library options, optimized output lists, performance improvements, and linked scripts for the rational design of custom CRISPR screen gene sets.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"216-223"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144040571","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
Optimizing Genome Editing in Mollusks (Crassostrea gigas) in Vitro Validation of sgRNA and Identifying Key Factors Influencing Efficiency. 优化软体动物（长牡蛎）基因组编辑体外验证sgRNA及确定影响效率的关键因素
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-06-01 Epub Date: 2025-02-28 DOI: 10.1089/crispr.2024.0086
Qian Li, Hong Yu, Shaojun Du, Qi Li
{"title":"Optimizing Genome Editing in Mollusks (<i>Crassostrea gigas</i>) <i>in Vitro</i> Validation of sgRNA and Identifying Key Factors Influencing Efficiency.","authors":"Qian Li, Hong Yu, Shaojun Du, Qi Li","doi":"10.1089/crispr.2024.0086","DOIUrl":"10.1089/crispr.2024.0086","url":null,"abstract":"<p><p>CRISPR-Cas9 genome editing holds tremendous potential for accelerating genetic improvements in aquaculture. The success of the CRISPR-Cas9 system relies on the specificity and efficiency of engineered single-guide RNAs (sgRNAs). In this study, we optimized an <i>in vitro</i> validation protocol for sgRNAs to streamline the gene editing process, capitalizing on the limited breeding season of the Pacific oyster (<i>Crassostrea gigas</i>). We evaluated the efficiency of 11 sgRNAs targeting four genes both <i>in vitro</i> and <i>in vivo</i> in <i>C. gigas</i>. In addition, we found that Cas9 protein differs from Cas9 mRNA in gene editing efficiency at various stages of early development. Cas9 protein proved particular efficacy in achieving early and efficient gene knockout, functioning effectively during the first cell division and facilitating biallelic gene knockouts. Statistical analysis showed that in the protein group, the biallelic editing frequency ranged from 12.5% to 57.8%, and the overall editing frequency reached as high as 75-90.6%. The mRNA group exhibited a biallelic editing frequency of 3.1-14.0% and the overall editing frequency spanning 65.6-78.1%. Contrary to expectations, low-temperature incubation (20°C) of oyster embryos prolonged the time window for the first cell division but did not improve gene editing efficiency, likely due to the high temperature sensitivity of Cas9 enzyme activity. Together, this study provides a comprehensive analysis of factors affecting the efficiency of CRISPR-Cas9 gene editing in <i>C. gigas</i>, providing a robust framework for future gene editing endeavors in mollusks and other marine invertebrates.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":"205-215"},"PeriodicalIF":3.7,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143528008","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
Biotechnologies in the World: On Global Asymmetries and the Need for Cosmopolitanism. 世界上的生物技术：关于全球不对称和世界主义的需要。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0054
Kaushik Sunder Rajan
{"title":"Biotechnologies in the World: On Global Asymmetries and the Need for Cosmopolitanism.","authors":"Kaushik Sunder Rajan","doi":"10.1089/crispr.2025.0054","DOIUrl":"https://doi.org/10.1089/crispr.2025.0054","url":null,"abstract":"<p><p>Conversations regarding genome editing are not simply about the transformative science involved. They touch upon fundamental moral questions concerning the human condition, indeed what it means to be human itself. The recent approval of a gene therapy for sickle cell disease encapsulates the relationship between scientific innovation and health care access and the relations of power and political economy that structure the world of biotech and biomedicine. Globally transformative biotechnologies must ethically situate themselves if they are not merely to reproduce longstanding historical and structural asymmetries. The time has come to embrace a cosmopolitan ethic that is attuned to the varied constitutionalisms through which debates about public good, healthy societies, and social compacts materialize around the world.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144112739","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
Science with Society at Science Gallery Bengaluru. 科学与社会在班加罗尔科学画廊。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0052
Jahnavi Phalkey
{"title":"Science with Society at Science Gallery Bengaluru.","authors":"Jahnavi Phalkey","doi":"10.1089/crispr.2025.0052","DOIUrl":"https://doi.org/10.1089/crispr.2025.0052","url":null,"abstract":"<p><p>Science Gallery Bengaluru was established to serve as a two-way bridge between the public and new and old research. This work is furthered through public engagement in the form of year-long living exhibitions, a public laboratory complex with five experimental spaces, and a mentorship initiative for young adults. There is a strong conversation about professions in science and engineering in the Indian public domain, but its cultural equivalent is less developed. In this context, ideas and topics for exploration at the Gallery are chosen not for novelty with which the public is to be familiarized, but for their already robust presence in public discourse in order to explore their complexity through research in the human, social, and natural sciences, with a view to enabling visitors to make more informed choices in everyday life.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144112751","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
Summitting CRISPR for Human Heritable Genome Editing. 人类可遗传基因组编辑CRISPR峰会。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0051
Françoise Baylis
{"title":"Summitting CRISPR for Human Heritable Genome Editing.","authors":"Françoise Baylis","doi":"10.1089/crispr.2025.0051","DOIUrl":"https://doi.org/10.1089/crispr.2025.0051","url":null,"abstract":"<p><p>The ethical issues of human heritable genome editing have been discussed at international summits held since 2015. In this Perspective, I consider how the discussions evolved over three summits held in Washington, DC (2015), Hong Kong (2018), and London (2023). The significance of safety and efficacy, meanings of a moratorium, and place of broad societal consensus are traced through publications produced surrounding these summits. Looking ahead, I highlight the difference between two fundamentally distinct ethical questions: Is human heritable genome editing ethical? Can human heritable genome editing be done ethically?</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144112753","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
Introducing Faith-Based Claims to Ethical Discourse on Scientific Matters. 将基于信仰的主张引入科学问题的伦理论述。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0045
James F Keenan
{"title":"Introducing Faith-Based Claims to Ethical Discourse on Scientific Matters.","authors":"James F Keenan","doi":"10.1089/crispr.2025.0045","DOIUrl":"https://doi.org/10.1089/crispr.2025.0045","url":null,"abstract":"<p><p>For the majority of the human community, the questions posed by heritable genome editing regarding the meanings of human life draw on various religious perspectives. Deliberations about genome editing would benefit from thinking about religious concerns in non-reductionist and non-instrumental terms. While it is often suggested that the scientific community is intolerant of religious views, it is often not the scientific community but those working in philosophy and religion who self-censor religious language. Rather than merely including people who are conversant in the language of religion or trying to derive an ethical Esperanto from generic religious beliefs, I advocate in this Perspective for the inclusion of people who voice their ethical commitments in the theological principles of their particular religious commitments.</p>","PeriodicalId":54232,"journal":{"name":"CRISPR Journal","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144112746","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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