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Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine. 将治疗性基因组编辑推向全球临床试验和医学。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0049
Kiran Musunuru, Fyodor Urnov
{"title":"Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine.","authors":"Kiran Musunuru, Fyodor Urnov","doi":"10.1089/crispr.2025.0049","DOIUrl":"https://doi.org/10.1089/crispr.2025.0049","url":null,"abstract":"<p><p>Moving CRISPR-based therapies from discovery to dosing patients in clinical trials and ultimately to approval involves navigating a challenging terrain of highs and lows. In this interview, physician-scientist Kiran Musunuru and genome editor Fyodor Urnov reflect on the past 20 years of their nonclinical and clinical programs in the field, the current landscape of innovation, and what they see on the horizon.</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":"144112748","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
Will Advances in Genetic Medicine Lessen the Need for Germline Modifications? 遗传医学的进步会减少对生殖系修饰的需求吗？
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0039
Timothy D Hunt
{"title":"Will Advances in Genetic Medicine Lessen the Need for Germline Modifications?","authors":"Timothy D Hunt","doi":"10.1089/crispr.2025.0039","DOIUrl":"https://doi.org/10.1089/crispr.2025.0039","url":null,"abstract":"<p><p>The cell and gene therapy/gene editing industry sits at the intersection of scientific research, technology development, regulation, ethics, and society. Hence, the biotechnology industry has a role in ensuring the inclusion of ethical considerations, diverse stakeholders, and assessments of medical progress, necessity, and realistic timelines for technological feasibility. The Alliance for Regenerative Medicine has sought to facilitate this role, fostering continued discussions of the scientific, ethical, and governance difficulties surrounding heritable human genome editing (HHGE), particularly in light of enormous advances in somatic gene therapies. The need for HHGE to address rare diseases is significantly diminished by the development of alternative approaches. Thoughtful deliberation about HHGE that takes into account the expertise and experience of the biotechnology industry is essential-as are guardrails to prevent unwarranted and irresponsible approaches to HHGE.</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":"144112757","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
Intent to Cure: The Need for a Rare Disease Platform in India and Across the Global South. 治愈的意图：印度和全球南方需要一个罕见疾病平台。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0046
Vijay Chandru, Vaijayanti Gupta, Vandana Hegde, Arvind Venkatesan, Reety Arora
{"title":"Intent to Cure: The Need for a Rare Disease Platform in India and Across the Global South.","authors":"Vijay Chandru, Vaijayanti Gupta, Vandana Hegde, Arvind Venkatesan, Reety Arora","doi":"10.1089/crispr.2025.0046","DOIUrl":"https://doi.org/10.1089/crispr.2025.0046","url":null,"abstract":"<p><p>The democratization of genomic technologies presents substantial opportunities for addressing rare genetic diseases, particularly in collaborations between the Global South and North. In this Perspective, we describe the current progress in gene therapy, including CRISPR, in India and see an upward trajectory of innovation. We propose the establishment of a rare disease platform in India and across the Global South designed to bridge scientific, clinical, and economic gaps, transforming untapped genetic diversity into shared opportunities for therapeutic innovation and health care equity. This platform would encompass a comprehensive data infrastructure capturing clinical, genomic, and biosample data, complemented by an artificial intelligence-powered analytics layer to enhance patient engagement and clinical trial matching, ultimately enabling cost-effective research and development (R&D) of novel therapies.</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":"144112745","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
Embryonic Stem Cell Research Oversight Committees: A Model for Oversight of Innovative Research? 胚胎干细胞研究监督委员会：创新研究的监督模式？
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0056
Robert D Truog
{"title":"Embryonic Stem Cell Research Oversight Committees: A Model for Oversight of Innovative Research?","authors":"Robert D Truog","doi":"10.1089/crispr.2025.0056","DOIUrl":"https://doi.org/10.1089/crispr.2025.0056","url":null,"abstract":"<p><p>Ethically challenging areas of research raise the question of how science can proceed in an ethically reflective and careful way and what role ethical deliberative bodies at the local level play in this. Important lessons can be drawn from the experience of Embryonic Stem Cell Research Oversight Committees (ESCROs). ESCROs have evolved as reflective spaces in response to changes in the science, including research on \"humanization\" of experimental rodents, embryogenesis, and developing human germ cells. Institutional efforts can contribute to broader goals of open and democratic deliberation.</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":"144112742","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
Editing the Future: A Call for Justice, Humility, and Communion. 编辑未来：呼吁正义、谦卑和共融。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0041
Sharon F Terry
{"title":"Editing the Future: A Call for Justice, Humility, and Communion.","authors":"Sharon F Terry","doi":"10.1089/crispr.2025.0041","DOIUrl":"https://doi.org/10.1089/crispr.2025.0041","url":null,"abstract":"<p><p>Technology must exist in the service of people, particularly those suffering from illness and disease. Drawing on decades of experience as the cofounder of a rare genetic disease research organization and my participation in the liberation theology movement of the Roman Catholic Church, in this Perspective, I discuss the high stakes of human heritable genome editing. Values forged in civil rights and social justice movements can inform how genome editing projects consider the vulnerable, the poor, and the voiceless. Those empowered in academia, biotech, philanthropy, and policy should understand that questions about genome editing entail responsibility for shaping the very concept of shared humanity.</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":"144112741","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
Starlink Medicines in Jurassic Healthcare Systems-Are We Ready? 侏罗纪医疗系统中的星链药物——我们准备好了吗？
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0042
Tay Salimullah
{"title":"Starlink Medicines in Jurassic Healthcare Systems-Are We Ready?","authors":"Tay Salimullah","doi":"10.1089/crispr.2025.0042","DOIUrl":"https://doi.org/10.1089/crispr.2025.0042","url":null,"abstract":"<p><p>Genomic medicines are transforming the landscape of human health-but their promise risks being confined to the few. While science has entered the 21<sup>st</sup> century, most health systems remain unequipped to deliver one-time curative therapies. This essay calls for a global reckoning: what do we truly value in innovation, and how can we align systems, incentives, and ethics to democratize access? Drawing on real-world experience across four continents, it argues for a new social compact-embedding equity, rethinking value, and modernizing delivery-to ensure that the miracles of modern medicine become global, sustainable, and just.</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":"144112752","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
The New Old Colonialism. 新老殖民主义。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0059
Patricia J Williams
{"title":"The New Old Colonialism.","authors":"Patricia J Williams","doi":"10.1089/crispr.2025.0059","DOIUrl":"https://doi.org/10.1089/crispr.2025.0059","url":null,"abstract":"<p><p>The question of how law should regulate the manipulation of the human genome or germline is inflected by the interconnected, intersectional parrying among different systems of moral value. Contract law and constitutional law reflect two poles of interest: the transactional aspects of market valuation and the relational aspects of the web of life that acknowledge \"pricelessness.\" In the decades from the initial decoding of the human genome in 2000 to the emergence of CRISPR technologies, powerful companies and powerful individuals now all but own the fate of our species and the health of our planet. The destructive effects of the realignments we are undergoing are still largely invisible (if not for long) and largely unresponsive to conventional checks.</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":"144112754","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
Can Genome Editing Therapies Offer Hope Rather Than Frustration? A Perspective on Ethics in India. 基因组编辑疗法能带来希望而不是沮丧吗？印度伦理观。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0040
Roli Mathur
{"title":"Can Genome Editing Therapies Offer Hope Rather Than Frustration? A Perspective on Ethics in India.","authors":"Roli Mathur","doi":"10.1089/crispr.2025.0040","DOIUrl":"https://doi.org/10.1089/crispr.2025.0040","url":null,"abstract":"<p><p>India's vast population contains a full spectrum of diseases and their variations. The potential of gene editing can be unlocked, but only if the technology is used for the betterment of many, not exclusively the elite. The moral and ethical aspects of this research must be considered in governance frameworks and ethical review to promote access and public engagement. A dialogue between technologists, policymakers, and communities can help align gene editing initiatives with human values. Can these promising technologies offer India's diverse population hope rather than frustration?</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":"144112740","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
The Roles and Responsibilities of Scientific Journals in Research Governance: Editorial Policymaking at Nature and Springer Nature (1995-2023). 科学期刊在科研治理中的角色与责任：Nature和施普林格Nature的编辑决策（1995-2023）。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0047
Philip Campbell
{"title":"The Roles and Responsibilities of Scientific Journals in Research Governance: Editorial Policymaking at <i>Nature</i> and Springer Nature (1995-2023).","authors":"Philip Campbell","doi":"10.1089/crispr.2025.0047","DOIUrl":"https://doi.org/10.1089/crispr.2025.0047","url":null,"abstract":"<p><p>Scientific journals develop and enforce editorial guidelines that are a component of governing science. In this essay, the former editor-in-chief of <i>Nature</i> reflects on several prominent examples of how scientific journals have been involved in setting and judging ethical norms in scientific research. Editors, in consultation with external experts, can balance transparency and public trust, stakeholder engagement, inclusive consultation, and access to research to address concerns surrounding dual-use research, societal harms, and research integrity.</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":"144112755","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
Toward a Global Deliberative System for Genome Editing. 迈向基因组编辑的全球审议系统。
IF 3.7 4区 生物学
CRISPR Journal Pub Date : 2025-05-21 DOI: 10.1089/crispr.2025.0044
John S Dryzek
{"title":"Toward a Global Deliberative System for Genome Editing.","authors":"John S Dryzek","doi":"10.1089/crispr.2025.0044","DOIUrl":"https://doi.org/10.1089/crispr.2025.0044","url":null,"abstract":"<p><p>Despite widespread agreement on the need for public participation in genome editing governance, questions remain about how best to structure citizen deliberation such that it can better inform policymaking. Representative and transformative conversations are not necessarily enabled by random sampling. Reflecting on lessons learned from recent citizens' juries and assemblies, in this essay I identify group-building, skilling up, recruiting invested participants, and over-sampling of marginal discourses as strategies to advance the contribution of citizen deliberation to a global deliberative system for human heritable genome editing governance.</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":"144112756","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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