Sonodelivery Facilitates Sustained Luciferase Expression from an Episomal Vector in Skeletal Muscle

Materials

Manoel Figueiredo Neto¹  Rachel Letteri²  Delphine Chan-Seng³  Todd Emrick²  Marxa L. Figueiredo¹ 

[1] Department of Pharmacology & Toxicology, 301 University Blvd., Galveston, TX 77555, USA; E-Mail:;Department of Polymer Science and Engineering, University of Massachusetts-Amherst, Amherst, MA 01003, USA; E-Mails:;Institut Charles Sadron UPR22-CNRS, 23 Rue du Loess, 67034 Strasbourg, France; E-Mail:

关键词: sonodelivery;    episome;    polymer;    nanoplex;    microbubbles;    non-viral vector;    skeletal muscle;    sustained expression;    luciferase;   

DOI  :  10.3390/ma8074608

来源: mdpi

【 摘 要 】

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.

【 授权许可】

CC BY   
© 2015 by the authors; licensee MDPI, Basel, Switzerland.