title: Sonodelivery Facilitates Sustained Luciferase Expression from an Episomal Vector in Skeletal Muscle.
abstract: 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 expressionin vivowith the promise of a minimal inflammatory gene expression profile.
authors.affiliation: Department of Pharmacology & Toxicology, 301 University Blvd., Galveston, TX 77555, USA.
authors.forename: Manoel Figueiredo
authors.initials: MF
authors.lastname: Neto
authors.affiliation: Department of Polymer Science and Engineering, University of Massachusetts-Amherst, Amherst, MA 01003, USA.
authors.forename: Rachel
authors.initials: R
authors.lastname: Letteri
authors.affiliation: Institut Charles Sadron UPR22-CNRS, 23 Rue du Loess, 67034 Strasbourg, France.
authors.forename: Delphine
authors.initials: D
authors.lastname: Chan-Seng
authors.affiliation: Department of Polymer Science and Engineering, University of Massachusetts-Amherst, Amherst, MA 01003, USA.
authors.forename: Todd
authors.initials: T
authors.lastname: Emrick
authors.affiliation: Department of Pharmacology & Toxicology, 301 University Blvd., Galveston, TX 77555, USA.
authors.forename: Marxa L
authors.initials: ML
authors.lastname: Figueiredo
keywords: episome; luciferase; microbubbles; nanoplex; non-viral vector; polymer; skeletal muscle; sonodelivery; sustained expression
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