Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods
Department of Pediatrics, Division of Pulmonary and Allergy; Gene Therapy Center
Genetics and Genomics | Therapeutics
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.
DOI of Published Version
Hum Gene Ther Clin Dev. 2015 Sep;26(3):159-64. doi: 10.1089/humc.2015.116. Epub 2015 Sep 10. Link to article on publisher's site
Human gene therapy. Clinical development
Gruntman, Alisha and Flotte, Terence R., "Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods" (2015). Pediatric Publications and Presentations. 61.