Kevin Leandro , David Rufino-Ramos , Koen Breyne , Emilio Di Ianni , Sara M. Lopes , Rui Jorge Nobre , Benjamin P. Kleinstiver , Pedro R.L. Perdigão , Xandra O. Breakefield , Luís Pereira de Almeida
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引用次数: 0
Abstract
Gene editing technologies have the potential to correct genetic disorders by modifying, inserting, or deleting specific DNA sequences or genes, paving the way for a new class of genetic therapies. While gene editing tools continue to be improved to increase their precision and efficiency, the limited efficacy of in vivo delivery remains a major hurdle for clinical use. An ideal delivery vehicle should be able to target a sufficient number of diseased cells in a transient time window to maximize on-target editing and mitigate off-target events and immunogenicity.
Here, we review major advances in novel delivery platforms based on cell-derived vesicles − extracellular vesicles and virus-like particles − for transient delivery of gene editing payloads. We discuss major findings regarding packaging, in vivo biodistribution, therapeutic efficacy, and safety concerns of cell-derived vesicles delivery of gene editing cargos and their potential for clinical translation.
基因编辑技术有可能通过修改、插入或删除特定的 DNA 序列或基因来纠正遗传疾病，从而为新型基因疗法铺平道路。虽然基因编辑工具在不断改进，以提高其精确度和效率，但体内给药的有限功效仍是临床应用的一大障碍。理想的递送载体应能在瞬时时间窗内靶向足够数量的病变细胞，从而最大限度地进行靶上编辑，并减少脱靶事件和免疫原性。在此，我们回顾了基于细胞源囊泡--细胞外囊泡和类病毒颗粒--的新型递送平台在基因编辑有效载荷瞬时递送方面的主要进展。我们讨论了细胞源性囊泡递送基因编辑载荷的包装、体内生物分布、疗效和安全性方面的主要发现及其临床转化的潜力。
期刊介绍：
The aim of the Journal is to provide a forum for the critical analysis of advanced drug and gene delivery systems and their applications in human and veterinary medicine. The Journal has a broad scope, covering the key issues for effective drug and gene delivery, from administration to site-specific delivery.
In general, the Journal publishes review articles in a Theme Issue format. Each Theme Issue provides a comprehensive and critical examination of current and emerging research on the design and development of advanced drug and gene delivery systems and their application to experimental and clinical therapeutics. The goal is to illustrate the pivotal role of a multidisciplinary approach to modern drug delivery, encompassing the application of sound biological and physicochemical principles to the engineering of drug delivery systems to meet the therapeutic need at hand. Importantly the Editorial Team of ADDR asks that the authors effectively window the extensive volume of literature, pick the important contributions and explain their importance, produce a forward looking identification of the challenges facing the field and produce a Conclusions section with expert recommendations to address the issues.
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