脂质递送系统的工程学方面:体内基因递送、安全标准和转化策略。

IF 12.1 1区 工程技术 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Ismail Eş , Aneesh Thakur , Amin Mousavi Khaneghah , Camilla Foged , Lucimara Gaziola de la Torre
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引用次数: 0

摘要

基因组缺陷会导致遗传疾病,可以通过基因疗法进行治疗。由于基因递送过程中遇到的种种限制,基于脂质的超分子胶体材料已成为前景广阔的基因载体系统。在非功能化形式下,脂质纳米粒子通常表现出较低的转基因表达效率,导致治疗效果不理想,特别是表达转基因的细胞百分比降低。由于存在化学活性取代基,利用特定化学配体设计基因药物输送系统成为解决这些弊端和提高治疗效果的创新策略。尽管对功能化策略进行了深入研究,但此类疗法的临床效果仍有待提高。在此,我们重点介绍并全面评述了脂质递送系统功能化的工程方面及其对开发新型基因载体的疗效,为从实验室到临床的转化提供了一个完整的缩影。我们概述了递送和内化过程中的现有挑战,并叙述了核酸脂质递送系统功能化的最新进展,以提高其疗效和安全性。此外,我们还讨论了使用这些载体扩大临床应用的临床试验以及主要的安全性问题。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Engineering aspects of lipid-based delivery systems: In vivo gene delivery, safety criteria, and translation strategies

Defects in the genome cause genetic diseases and can be treated with gene therapy. Due to the limitations encountered in gene delivery, lipid-based supramolecular colloidal materials have emerged as promising gene carrier systems. In their non-functionalized form, lipid nanoparticles often demonstrate lower transgene expression efficiency, leading to suboptimal therapeutic outcomes, specifically through reduced percentages of cells expressing the transgene. Due to chemically active substituents, the engineering of delivery systems for genetic drugs with specific chemical ligands steps forward as an innovative strategy to tackle the drawbacks and enhance their therapeutic efficacy. Despite intense investigations into functionalization strategies, the clinical outcome of such therapies still needs to be improved. Here, we highlight and comprehensively review engineering aspects for functionalizing lipid-based delivery systems and their therapeutic efficacy for developing novel genetic cargoes to provide a full snapshot of the translation from the bench to the clinics. We outline existing challenges in the delivery and internalization processes and narrate recent advances in the functionalization of lipid-based delivery systems for nucleic acids to enhance their therapeutic efficacy and safety. Moreover, we address clinical trials using these vectors to expand their clinical use and principal safety concerns.

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来源期刊
Biotechnology advances
Biotechnology advances 工程技术-生物工程与应用微生物
CiteScore
25.50
自引率
2.50%
发文量
167
审稿时长
37 days
期刊介绍: Biotechnology Advances is a comprehensive review journal that covers all aspects of the multidisciplinary field of biotechnology. The journal focuses on biotechnology principles and their applications in various industries, agriculture, medicine, environmental concerns, and regulatory issues. It publishes authoritative articles that highlight current developments and future trends in the field of biotechnology. The journal invites submissions of manuscripts that are relevant and appropriate. It targets a wide audience, including scientists, engineers, students, instructors, researchers, practitioners, managers, governments, and other stakeholders in the field. Additionally, special issues are published based on selected presentations from recent relevant conferences in collaboration with the organizations hosting those conferences.
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