基于 CRISPR 的疗法:革新药物开发和精准医疗。

IF 3.8 4区 医学 Q2 GENETICS & HEREDITY Current gene therapy Pub Date : 2024-01-01 DOI:10.2174/0115665232275754231204072320
Dilip Kumar Chanchal, Jitendra Singh Chaudhary, Pushpendra Kumar, Neha Agnihotri, Prateek Porwal
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引用次数: 0

摘要

随着 CRISPR-Cas9 的发现,药物开发和精准医疗发生了重大变化。这篇综述文章探讨了基于CRISPR技术的疗法的新应用方式,以及它们如何改变了医疗方式。CRISPR 技术能够精确、灵活地编辑基因,为寻找、验证和开发药物靶点开辟了新途径。此外,它还为个性化基因疗法、精确基因编辑和先进筛选技术开辟了道路,所有这些都为治疗各种疾病带来了巨大希望。在本文中,我们将介绍最新的研究和临床试验,这些研究和临床试验表明,CRISPR 可用于治疗遗传病、癌症、传染病和其他难以治疗的疾病。然而,本文也讨论了与基于CRISPR的疗法相关的伦理问题和法规问题,这表明安全、负责任地使用这些疗法是多么重要。随着CRISPR不断改变药物的制造和使用方式,这篇综述揭示了这一瞬息万变的领域已经取得的惊人成就以及未来可能发生的变化。
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CRISPR-Based Therapies: Revolutionizing Drug Development and Precision Medicine.

With the discovery of CRISPR-Cas9, drug development and precision medicine have undergone a major change. This review article looks at the new ways that CRISPR-based therapies are being used and how they are changing the way medicine is done. CRISPR technology's ability to precisely and flexibly edit genes has opened up new ways to find, validate, and develop drug targets. Also, it has made way for personalized gene therapies, precise gene editing, and advanced screening techniques, all of which hold great promise for treating a wide range of diseases. In this article, we look at the latest research and clinical trials that show how CRISPR could be used to treat genetic diseases, cancer, infectious diseases, and other hard-to-treat conditions. However, ethical issues and problems with regulations are also discussed in relation to CRISPR-based therapies, which shows how important it is to use them safely and responsibly. As CRISPR continues to change how drugs are made and used, this review shines a light on the amazing things that have been done and what the future might hold in this rapidly changing field.

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来源期刊
Current gene therapy
Current gene therapy 医学-遗传学
CiteScore
6.70
自引率
2.80%
发文量
46
期刊介绍: Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.
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