Gene transfer in skeletal muscle by systemic injection of DODAC lipopolyplexes.
Lipid-based vectors are a promising tool for gene therapy applications. Several studies have reported their use in vivo to transfect different organs. Few data, however, are available about lipid-mediated gene transfer in skeletal muscle. Here we report the initial results obtained after systemic administration of lipopolyplexes based on the DODAC cationic lipid in an animal model of muscle regeneration. In particular, we compared three routes of administration: intravenous (i.v.), intracardiac (IC) and intra-arterial (IA). Analysis of reporter gene expression (luciferase) showed that regenerating muscle is more efficiently transfected in all cases and that IA injection is by far the best approach.
Pampinella
F
F
Department of Biology, University of Padua, Via U. Bassi 58/B, I-35132 Padua, Italy.
Pozzobon
M
M
Zanetti
E
E
Gamba
P G
PG
McLachlan
I
I
Cantini
M
M
Vitiello
L
L
D000818:Animals; D004247:DNA / Q000494:pharmacology; D004333:Drug Administration Routes; D017930:Genes, Reporter / Q000187:drug effects / Q000502:physiology; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000138:chemical synthesis / Q000627:therapeutic use*; D008081:Liposomes / Q000138:chemical synthesis / Q000494:pharmacology; D008297:Male; D018482:Muscle, Skeletal / Q000187:drug effects* / Q000378:metabolism / Q000503:physiopathology; D009135:Muscular Diseases / Q000139:chemically induced / Q000188:drug therapy / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D010957:Plasmids / Q000494:pharmacology; D000644:Quaternary Ammonium Compounds / Q000494:pharmacology*; D051381:Rats; D017208:Rats, Wistar; D012038:Regeneration / Q000187:drug effects / Q000502:physiology; D013501:Surface-Active Agents / Q000494:pharmacology*; D016896:Treatment Outcome
D008081:Liposomes; D000644:Quaternary Ammonium Compounds; D013501:Surface-Active Agents; C015831:dimethyldioctadecylammonium; D004247:DNA