Gene Efficacy Assessment in Batten Mice
CLN2 disease, a form of Batten disease, is a neurodegenerative lysosomal storage disorder caused by mutations in the gene encoding the soluble enzyme tripeptidyl- peptidase-1 (TPP1). This model recapitulates most features of the human disease such as shortened lifespan, seizures, or abnormal gait. Monitoring of the neurobehavioral function in this model is challenging, however, as they are prone to noise- or stress-induced fatal seizures when handled. Real time non-invasive continuous monitoring using smart cages (Vium, Inc.) allows sensitive and non-biased recording of disease phenotype and treatment-related rescue in a seizure-prone mouse model, while limiting handling-related deaths.
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Niemann- Pick Type C
Read the full article at Perlara (see link below)
"A lot of exciting things are happening but we still have to characterize our lead compound PERL101 to determine possible mechanism of action and see if it will correct the disease in Niemann Pick Type C mouse models. As mentioned in a previous post, we run our mouse studies at Vium. And we were lucky to get some excellent disease characterization data using their sensored cages, such as what happens to motion, breathing rate, weight loss and more. They also conducted some behavioral studies on the animals to capture neurodegeneration. This included rotarod, gait in an open field, and walking on a beam. With rotarod we looked at how long animals were able to stay on the rotating rod, in the gait assay we looked at the change in animal movement or if the mouse was shaking, stumbling or hunched and with the beam test we looked to see how far the mice could walk on a straight beam. These tests were extremely valuable, and based on the results, we felt that the neurodegenerative effect in the disease state was best captured by the rotarod assay. So going forward, we will most likely add that to future studies."
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