Functional in vivo gene transfer into the myofibers of adult skeletal muscle

Massimo Donà, Marco Sandri, Katia Rossini, Isabella Dell'Aica, Marzena Podhorska-Okolow, Ugo Carraro

Research output: Contribution to journalArticlepeer-review

Abstract

The postmitotic nature and longevity of skeletal muscle fibers permit stable expression of any transfected gene. Direct in vivo injection of plasmid DNA, in both adult and regenerating muscles, is a safe, inexpensive, and easy approach. Here we present an optimized electroporation protocol based on the use of spatula electrodes to transfer cDNA in vivo into the adult myofibers of an anatomically defined muscle, which could be functionally characterized. In our hands, about 80% of adult myofibers were transfected in vivo by different plasmids for GFP fusion proteins or for β-galactosidase. The luciferase activity increased several orders of magnitude when compared to standard DNA delivery. In an anatomical defined muscle, the wide gene transfer was comparable to or better than that of retrovirus delivery, that recently has been shown to be prone to severe side-effects in human clinical studies. Furthermore, with our method the tissue damage was greatly decreased. Thus, the present work describes in vivo functional electrotransfer of genes in adult skeletal muscle fibers by a protocol that is of great potential for gene therapy, as well as for basic research.

Original languageEnglish
Pages (from-to)1132-1138
Number of pages7
JournalBiochemical and Biophysical Research Communications
Volume312
Issue number4
DOIs
Publication statusPublished - Dec 26 2003

Keywords

  • Adult skeletal muscle
  • Electroporation
  • Gene therapy

ASJC Scopus subject areas

  • Biochemistry
  • Biophysics
  • Molecular Biology

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