Creating Disease Models

ReGenX AAV Vector Technology can be used to create animal models of disease in which to test your small molecule or biological therapeutics. The model can be created by overexpressing a gene or genes that lead to disease, or by knocking down gene expression in the appropriate tissue using shRNA. With the long-term, stable transgene expression from ReGenX AAV Vector Technology, therapeutics can be evaluated for effects on disease state in vivo. It is also possible to express a human protein in an animal, and then test your therapeutic for its effects on the human protein rather than on its animal orthologue.

Some examples of using AAV to create animal models are shown below. For assistance in choosing your optimal AAV, and in designing or refining your animal model, please contact us.

Tissue	Disease	Gene delivered
Brain–hypothalamus	Hyperphagia/obesity	shRNA to melanocortin-4 receptor (MC4R)1
Brain	Neuronal degeneration (alpha-synucleinopathy)	mutant alpha-synuclein2
Brain–hippocampus	Alzheimer’s disease	amyloid-beta peptide3
Brain–substantia nigra	Parkinson’s disease	alpha-synuclein4
Brain–facial nuclei	ALS-like motor neuron defect	Cre recombinase (CNTFRLoxP mouse)5
Eye	Diabetic ocular neovascularization	VEGF6
Lung	Emphysema	Cre recombinase (VEGFLoxP mouse)7,8
Liver	Hypertension	C-Reactive Protein9
Liver	Heart Failure (transition from hypertrophy)	Decoy VEGF receptor10