The US Food and Drug Administration have just approved a genetically engineered swine research model for the rare disease ataxia telangiectasia for commercial use. The model, officially named the ExeGen ATM MiniSwine model, was produced by Exemplar Genetics to provide a research model that is more similar in its biology to humans than those currently used. Click here to read the full report at PR Newswire.
Ataxia telangiectasia (AT), the disease modelled by the swine, is a rare, inherited form of ataxia. AT affects approximately one in every 100,000 children. The symptoms of the disease tend to begin appearing early in childhood, and typically include difficulty moving, speaking, swallowing, and a weakened immune system amongst others. The disease is fast acting, and affected children often need a wheelchair by age 10. It is also life shortening, with most patients only living into their twenties, although some may survive significantly longer. AT is caused by a genetic mutation that results in the degradation of the cerebellum (an area in the brain) and/or spinal cord. The current lack of a cure for AT makes this disease a priority for research, and the swine models produced by ExeGen will support the work of researchers by more closely mimicking human symptoms and responses. Read more AT articles here, or learn more about it here.
Most research currently uses rodents, and in particular mice, to model diseases. However, this is often problematic because these animals are in many ways dissimilar to humans, and so their responses can be misleading about the processes that would be seen in humans. This can significantly hinder research efforts into understanding rare diseases and producing treatments. Since the failure rate for treatment development programs exceeds 80%, and the average cost of creating a successful treatment is more than $1 billion, Exemplar argues that improved animal models are needed to make headway in research programs. The similarities between their swine models and humans in terms of genetics, anatomy, and physiology mean that they are expected to provide a better reflection of human biological processes.