title: Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.
abstract: Recombinant adeno-associated virus (rAAV) can be delivered to the skeletal muscle of the limb (pelvic or thoracic) by means of regional intravascular delivery. This review summarizes the evolution of this technique to deliver rAAV either via the arterial blood supply or via the peripheral venous circulation. The focus of this review is on applications in large animal models, including preclinical studies. Based on this overview of past research, we aim to inform the design of preclinical and clinical studies.
authors.affiliation: 1 Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Alisha M
authors.initials: AM
authors.lastname: Gruntman
authors.affiliation: 1 Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|2 Department of Pediatrics, University of Massachusetts Medical School , Worcester, Massachusetts.|3 Microbiology & Physiologic Systems, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Terence R
authors.initials: TR
authors.lastname: Flotte
mesh_terms: D000818:Animals; D001158:Arteries; D000229:Dependovirus / Q000145:classification / Q000235:genetics*; D057510:Endovascular Procedures; D005121:Extremities; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000235:genetics*; D006801:Humans; D023421:Models, Animal; D014161:Transduction, Genetic; D019076:Transgenes; D014680:Veins