Gene therapy progress and prospects: hydrodynamic gene delivery.
Over the last few years, hydrodynamic tail vein delivery has established itself as a simple, yet very effective method for gene transfer into small rodents. Hydrodynamic delivery of plasmid DNA expression vectors or small interfering RNA allows for a broad range of in vivo experiments, including the testing of regulatory elements, antibody generation, evaluation of gene therapy approaches, basic biology and disease model creation (non-heritable transgenics). The recent development of the hydrodynamic limb vein procedure provides a safe nucleic acid delivery technique with equally high efficiency in small and large research animals and, importantly, the prospects for clinical translation.
Herweijer
H
H
Mirus Bio Corporation, Madison, WI, USA.
Wolff
J A
JA
D000818:Animals; D004247:DNA / Q000008:administration & dosage*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D007275:Injections, Intravenous / Q000379:methods; D008099:Liver; D051379:Mice; D023421:Models, Animal; D018482:Muscle, Skeletal; D034741:RNA, Small Interfering / Q000008:administration & dosage; D014162:Transfection / Q000379:methods*
D034741:RNA, Small Interfering; D004247:DNA