Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups
Department of Pediatrics; Horae Gene Therapy Center
Genetics and Genomics | Nervous System Diseases | Pediatrics | Therapeutics
In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported.
DOI of Published Version
Hum Gene Ther. 2017 Mar;28(3):228-230. doi: 10.1089/hum.2017.037. Link to article on publisher's site
Human gene therapy
Gruntman, Alisha; Su, Lin; and Flotte, Terence R., "Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups" (2017). Pediatric Publications and Presentations. 138.