Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups.
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.
1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|2 Department of Pediatrics, University of Massachusetts Medical School , Worcester, Massachusetts.|3 Department of Clinical Sciences, Tufts University Cummings School of Veterinary Medicine , North Grafton, Massachusetts.
Alisha M
AM
Gruntman
4 Center of Geriatrics and Gerontology, West China Hospital, Sichuan University , Chengdu, P.R. China .
Lin
L
Su
1 Horae Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|2 Department of Pediatrics, University of Massachusetts Medical School , Worcester, Massachusetts.
Terence R
TR
Flotte
D000818:Animals; D000831:Animals, Newborn; D001921:Brain / Q000166:cytology / Q000378:metabolism*; D002478:Cells, Cultured; D000229:Dependovirus / Q000235:genetics*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000235:genetics*; D049452:Green Fluorescent Proteins / Q000378:metabolism*; D007275:Injections, Intravenous; D051379:Mice; D008810:Mice, Inbred C57BL; D009915:Orbit / Q000098:blood supply; D012160:Retina / Q000166:cytology / Q000378:metabolism*; D014161:Transduction, Genetic / Q000379:methods*; D014680:Veins
D049452:Green Fluorescent Proteins
CNS; eye; intravenous; single-stranded