Sonodelivery facilitates sustained luciferase expression from an episomal vector in skeletal muscle

Manoel Figueiredo Neto, Rachel Letteri, Delphine Chan-Seng, Todd Emrick, Marxa L. Figueiredo

Research output: Contribution to journalArticlepeer-review

5 Scopus citations

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 expression in vivo with the promise of a minimal inflammatory gene expression profile.

Original languageEnglish (US)
Pages (from-to)4608-4617
Number of pages10
JournalMaterials
Volume8
Issue number7
DOIs
StatePublished - 2015

Keywords

  • Episome
  • Luciferase
  • Microbubbles
  • Nanoplex
  • Non-viral vector
  • Polymer
  • Skeletal muscle
  • Sonodelivery
  • Sustained expression

ASJC Scopus subject areas

  • Condensed Matter Physics
  • General Materials Science

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