Sonodelivery Facilitates Sustained Luciferase Expression from an Episomal Vector in Skeletal Muscle.
Sonodelivery Facilitates Sustained Luciferase Expression from an Episomal Vector in Skeletal Muscle.
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DOI：
10.3390/ma8074608
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发表时间：
2015-07-01
期刊：
影响因子：
--
通讯作者：
Figueiredo ML
中科院分区：
文献类型：
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作者：
Neto MF;Letteri R;Chan-Seng D;Emrick T;Figueiredo ML
成功地将基因递送至骨骼肌是期望的目标，不仅用于治疗肌肉疾病，而且用于免疫、治疗代谢紊乱和/或递送可治疗全身性病症（例如骨转移癌）的基因表达。虽然裸DNA摄取到骨骼肌中是可能的，但在没有额外的化学或物理递送方法的情况下，它在很大程度上是低效的。我们描述了一种系统，用于非病毒或质粒DNA的骨骼肌使用超声波辅助声致孔的纳米复合物结合质粒DNA和基于聚（环辛烯-接枝-寡肽）的支链聚合物。本文所述的材料和方法有望推进用于治疗应用的超声递送和基因递送到肌肉的领域，因为提出了一种简单的系统，其能够在体内长期基因表达，并具有最小炎症基因表达谱的前景。
Successful gene delivery to skeletal muscle is a desirable goal, not only for treating muscle diseases, but also for immunization, treatment of metabolic disorders, and/or delivering gene expression that can treat systemic conditions, such as bone metastatic cancer, for example. Although naked DNA uptake into skeletal muscle is possible, it is largely inefficient in the absence of additional chemical or physical delivery methods. We describe a system for delivery of non-viral or plasmid DNA to skeletal muscle using ultrasound-assisted sonoporation of a nanoplex combining plasmid DNA and a branched polymer based on poly(cyclooctene-graft-oligopeptide). The materials and methods described herein promise to advance the field of sonodelivery and of gene delivery to muscle for therapeutic applications since a simple system is presented that enables long-term gene expression in vivo with the promise of a minimal inflammatory gene expression profile.
影响因子：
4.8
作者：
Zolochevska, Olga;Figueiredo, Marxa L.
通讯作者：
Figueiredo, Marxa L.