Biological Differences in rAAV Transduction of Airway Epithelia in Humans and in Old World Non-human Primates.

Non-human primates (NHPs) are considered to be among the most relevant animal models for pre-clinical testing of human therapies, on the basis of their close evolutionary relatedness to humans in terms of organ cell biology and physiology. In this study, we sought to investigate whether NHP models accurately reflect the effectiveness of recombinant adeno-associated virus (rAAV)-mediated gene delivery to the airway in humans. In order to do this, we utilized an identical model system of differentiated airway epithelia from Indian Rhesus monkeys and from humans, cultured at an air-liquid interface (ALI).

Molecular beacon genotyping for globoid cell leukodystrophy from hair roots in the twitcher mouse and rhesus macaque.

Rapid and accurate genotype determination is ideal for the maintenance of breeding colonies of laboratory animal models of genetic disease. The rhesus macaque and murine (twitcher) models of globoid cell leukodystrophy have a dinucleotide deletion or single nucleotide substitution, respectively, which abolish ceramide beta-galactosidase activity and are authentic models of Krabbe disease. We report a molecular beacon PCR assay for each species which allows unambiguous determination of the genotype in under 4h.