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革新肺癌治疗:创新的 CRISPR-Cas9 递药策略。

Revolutionizing Lung Cancer Treatment: Innovative CRISPR-Cas9 Delivery Strategies.

机构信息

University Institute of Pharma Sciences, Chandigarh University, Gharuan, Mohali, 140413, India.

University Centre for Research and Development, Chandigarh University, Gharuan, Mohali, 140413, India.

出版信息

AAPS PharmSciTech. 2024 Jun 6;25(5):129. doi: 10.1208/s12249-024-02834-6.

DOI:10.1208/s12249-024-02834-6
PMID:38844700
Abstract

Lung carcinoma, including both non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC), remains a significant global health challenge due to its high morbidity and mortality rates. The objsective of this review is to meticulously examine the current advancements and strategies in the delivery of CRISPR-Cas9 gene-editing technology for the treatment of lung carcinoma. This technology heralds a new era in molecular biology, offering unprecedented precision in genomic modifications. However, its therapeutic potential is contingent upon the development of effective delivery mechanisms that ensure the efficient and specific transport of gene-editing tools to tumor cells. We explore a variety of delivery approaches, such as viral vectors, lipid-based nanoparticles, and physical methods, highlighting their respective advantages, limitations, and recent breakthroughs. This review also delves into the translational and clinical significance of these strategies, discussing preclinical and clinical studies that investigate the feasibility, efficacy, and safety of CRISPR-Cas9 delivery for lung carcinoma. By scrutinizing the landscape of ongoing clinical trials and offering translational perspectives, we aim to elucidate the current state and future directions of this rapidly evolving field. The review is structured to first introduce the problem and significance of lung carcinoma, followed by an overview of CRISPR-Cas9 technology, a detailed examination of delivery strategies, and an analysis of clinical applications and regulatory considerations. Our discussion concludes with future perspectives and challenges, such as optimizing delivery strategies, enhancing specificity, mitigating immunogenicity concerns, and addressing regulatory issues. This comprehensive overview seeks to provide insights into the potential of CRISPR-Cas9 as a revolutionary approach for targeted therapies and personalized medicine in lung carcinoma, emphasizing the importance of delivery strategy development in realizing the full potential of this groundbreaking technology.

摘要

肺癌,包括非小细胞肺癌(NSCLC)和小细胞肺癌(SCLC),由于其高发病率和死亡率,仍然是一个重大的全球健康挑战。本综述的目的是仔细检查 CRISPR-Cas9 基因编辑技术在治疗肺癌方面的最新进展和策略。这项技术开创了分子生物学的新时代,为基因组修饰提供了前所未有的精度。然而,其治疗潜力取决于开发有效的传递机制,以确保基因编辑工具能够高效和特异性地递送到肿瘤细胞。我们探讨了多种传递方法,如病毒载体、基于脂质的纳米颗粒和物理方法,突出了它们各自的优点、局限性和最新突破。本综述还深入探讨了这些策略的转化和临床意义,讨论了研究 CRISPR-Cas9 传递用于肺癌的可行性、疗效和安全性的临床前和临床研究。通过仔细研究正在进行的临床试验的情况并提供转化视角,我们旨在阐明这个快速发展领域的现状和未来方向。本综述的结构首先介绍了肺癌的问题和意义,然后概述了 CRISPR-Cas9 技术,详细检查了传递策略,并分析了临床应用和监管考虑因素。我们的讨论以未来的观点和挑战结束,例如优化传递策略、增强特异性、减轻免疫原性问题以及解决监管问题。本综述旨在提供有关 CRISPR-Cas9 作为肺癌靶向治疗和个性化医学的革命性方法的潜力的见解,强调在实现这项突破性技术的全部潜力方面,传递策略开发的重要性。

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Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy.生物材料介导的CRISPR/Cas9递送:基因治疗中的近期挑战与机遇
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