Gene Therapy and Gene Editing: Current Trends and Future Prospects of Molecular Medicine.

IF 3.3 4区 医学 Q2 GENETICS & HEREDITY Current gene therapy Pub Date : 2025-02-18 DOI:10.2174/0115665232347734250211074241
Aniruddha Sen, Vijay Singh, Shailendra Dwivedi, Ruchika Agrawal, Akash Bansal, Shashank Shekhar, Sudhir Shyam Kushwaha
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Abstract

Gene therapy and genome editing have emerged as transformative approaches in the management of a diverse range of genetic and acquired diseases. This evaluation offers a thorough examination of the present state and prospects of these innovative technologies. Gene therapy is a prospective approach to the treatment and prevention of a variety of conditions, including complex cancers and inherited genetic disorders, which entail the introduction, removal, or modification of genetic material within a patient's cells. Genome editing, particularly through techniques such as CRISPR-Cas9, enables targeted corrections of genetic defects and opens new possibilities for personalized medicine by allowing for precise modifications at the DNA level. The review addresses the ethical implications, clinical applications, and significant advancements of these technologies. This article endeavors to underscore the substantial influence of gene therapy and genome editing on contemporary medicine by assessing the most recent research and clinical trials, thereby emphasizing their potential to revolutionize disease treatment and management.

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基因治疗和基因编辑:分子医学的当前趋势和未来展望。
基因治疗和基因组编辑已经成为管理各种遗传和获得性疾病的变革性方法。这项评估对这些创新技术的现状和前景进行了全面的审查。基因治疗是一种治疗和预防多种疾病的前瞻性方法,包括复杂的癌症和遗传性遗传病,这需要在患者细胞内引入,去除或修饰遗传物质。基因组编辑,特别是通过CRISPR-Cas9等技术,能够有针对性地纠正遗传缺陷,并通过允许在DNA水平上进行精确修改,为个性化医疗开辟了新的可能性。本文综述了这些技术的伦理意义、临床应用和重大进展。本文通过评估最新的研究和临床试验,努力强调基因治疗和基因组编辑对当代医学的重大影响,从而强调它们有可能彻底改变疾病的治疗和管理。
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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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