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CRISPR 与基因编辑:药物研发的变革性技术。

CRISPR and Gene Editing: A Game-changer in Drug Development.

机构信息

Department of Pharmaceutics, ISF College of Pharmacy, Moga, Punjab 142001, India.

出版信息

Curr Pharm Des. 2024;30(15):1133-1135. doi: 10.2174/0113816128298080240328053845.

DOI:10.2174/0113816128298080240328053845
PMID:38584552
Abstract

CRISPR and gene editing technologies have emerged as transformative tools in medicine, offering unprecedented precision in targeting genetic disorders and revolutionizing drug development. This review explores the multifaceted impact of CRISPR across various medical domains, from hereditary diseases to infectious diseases and cancer. The potential of CRISPR in personalized medicine, therapeutic innovation, and pandemic prevention is highlighted, along with its role in reshaping traditional drug development processes. However, alongside its promise, ethical considerations loom large, particularly regarding germline editing and equitable access to treatments. The commercialization of CRISPR poses further challenges, raising questions about affordability and healthcare equity. Collaboration among scientists, policymakers, and the public is emphasized to navigate the ethical and societal implications of CRISPR responsibly. As the field advances, it is essential to ensure that the benefits of CRISPR are realized while addressing potential risks and maintaining a commitment to the well-being of future generations.

摘要

CRISPR 和基因编辑技术已经成为医学领域的变革性工具,在靶向遗传疾病方面提供了前所未有的精准度,并彻底改变了药物研发。本文探讨了 CRISPR 在多个医学领域的多方面影响,从遗传性疾病到传染病和癌症。突出了 CRISPR 在个性化医疗、治疗创新和大流行病预防方面的潜力,以及它在重塑传统药物开发过程中的作用。然而,除了其承诺之外,还存在着巨大的伦理考虑因素,特别是涉及生殖系编辑和公平获得治疗的问题。CRISPR 的商业化带来了进一步的挑战,引发了关于可负担性和医疗保健公平性的问题。强调了科学家、政策制定者和公众之间的合作,以负责任地应对 CRISPR 的伦理和社会影响。随着该领域的发展,必须确保在解决潜在风险的同时实现 CRISPR 的益处,并承诺关注子孙后代的福祉。

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本文引用的文献

1
Targeting miRNA by CRISPR/Cas in cancer: advantages and challenges.通过 CRISPR/Cas 靶向 miRNA 治疗癌症:优势与挑战。
Mil Med Res. 2023 Jul 17;10(1):32. doi: 10.1186/s40779-023-00468-6.
2
Insights into the Mechanism of CRISPR/Cas9-Based Genome Editing from Molecular Dynamics Simulations.基于分子动力学模拟对CRISPR/Cas9基因组编辑机制的深入洞察
ACS Omega. 2022 Dec 30;8(2):1817-1837. doi: 10.1021/acsomega.2c05583. eCollection 2023 Jan 17.
3
The myotonic dystrophy type 1 drug development pipeline: 2022 edition.2022年版1型强直性肌营养不良症药物研发进展
Drug Discov Today. 2023 Mar;28(3):103489. doi: 10.1016/j.drudis.2023.103489. Epub 2023 Jan 9.
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CRISPR-Cas9: A History of Its Discovery and Ethical Considerations of Its Use in Genome Editing.CRISPR-Cas9:从发现到应用于基因组编辑的伦理考量简史。
Biochemistry (Mosc). 2022 Aug;87(8):777-788. doi: 10.1134/S0006297922080090.
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CRISPR-Cas9 library screening approach for anti-cancer drug discovery: overview and perspectives.CRISPR-Cas9 文库筛选方法在抗癌药物发现中的应用:概述与展望。
Theranostics. 2022 Apr 11;12(7):3329-3344. doi: 10.7150/thno.71144. eCollection 2022.
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Engineering the next-generation of CAR T-cells with CRISPR-Cas9 gene editing.利用CRISPR-Cas9基因编辑技术构建下一代嵌合抗原受体T细胞(CAR T细胞)
Mol Cancer. 2022 Mar 18;21(1):78. doi: 10.1186/s12943-022-01559-z.
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Current applications and future perspective of CRISPR/Cas9 gene editing in cancer.CRISPR/Cas9 基因编辑在癌症中的当前应用和未来展望。
Mol Cancer. 2022 Feb 21;21(1):57. doi: 10.1186/s12943-022-01518-8.
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The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases.CRISPR/Cas9基因编辑作为遗传性疾病治疗策略的潜力
Front Cell Dev Biol. 2021 Dec 15;9:699597. doi: 10.3389/fcell.2021.699597. eCollection 2021.
9
CRISPR-enhanced human adipocyte browning as cell therapy for metabolic disease.CRISPR 增强的人类脂肪细胞棕色化作为代谢疾病的细胞疗法。
Nat Commun. 2021 Nov 26;12(1):6931. doi: 10.1038/s41467-021-27190-y.
10
Patents, ethics, biosafety and regulation using CRISPR technology.利用 CRISPR 技术的专利、伦理、生物安全和监管
Prog Mol Biol Transl Sci. 2021;181:345-365. doi: 10.1016/bs.pmbts.2021.01.023. Epub 2021 Feb 24.