Journal article
Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature
P Gregorevic, BR Schultz, JM Allen, JB Halldorson, MJ Blankinship, NA Meznarich, CS Kuhr, C Doremus, E Finn, D Liggitt, JS Chamberlain
Molecular Therapy | CELL PRESS | Published : 2009
DOI: 10.1038/mt.2009.116
Abstract
A growing body of research supports the development of recombinant adeno-associated viral (rAAV) vectors for delivery of gene expression cassettes to striated musculature as a method of treating severe neuromuscular conditions. However, it is unclear whether delivery protocols that achieve extensive gene transfer in mice can be adapted to produce similarly extensive gene transfer in larger mammals and ultimately patients. Consequently, we sought to investigate methodological modifications that would facilitate rAAV-mediated gene transfer to the striated musculature of canines. A simple procedure incorporating acute (i) occlusion of limb blood flow, (ii) exsanguination via compression bandage..
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Awarded by National Institutes of Health
Funding Acknowledgements
We acknowledge the excellent assistance provided by the veterinarians and the veterinary technicians in the UW Department of Comparative Medicine. We thank Miki Haraguchi for performing cryosectioning and histochemistry. This work was supported by grants ( to J. S. C.) from the National Institutes of Health ( NIH R37 AR040864) and the Muscular Dystrophy Association ( USA). P. G. was supported in part by a development grant from the MDA, and B. R. S. was supported in part by the University of Washington Medical Scientist Training Program and by an ARCS Foundation Fellowship. This work was performed in Seattle, Washington, USA.