AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta).

ABSTRACT: Many genetic metabolic diseases manifest in infancy, therefore, it is important to develop effective treatments that could be implemented at this time. Adeno-associated virus serotype 8 (AAV8) gene transfer has been studied in neonatal mouse, cat, and dog models and shown some efficacy with a single hepatic injection at birth. AAV8-mediated liver gene transfer has also generated sustained therapeutic effects in feline and canine models of lysosomal storage disorders. In these models, delaying the age of vector treatment increased gene transfer stability. The growth rate of infant nonhuman primates is more similar to the growth trajectory of humans, thus infant monkeys provide an excellent model to study AAV gene transfer efficiency, stability, and safety. In this study, we report for the f

SUBMITTER: Wang L 

PROVIDER: S-EPMC3222523 | biostudies-literature | 2011 Nov

REPOSITORIES: biostudies-literature