Abstract
Hunting for the cure for Huntington´s disease (HD) requires extended pre-clinical studies to screen the safety, tolerability and efficacy of the potential therapeutic treatments. Therefore, the large animal models were developed for translational research to human studies. The battery of tests was designed for monitoring of phenotype similarity of the transgenic HD minipig model with HD patients. Motor and cognitive functions were monitored in the group of transgenic (tgHD) and wild-type (wt) minipigs in different ages in the 3 following years. The results showed differences between tgHD and wt animals. The sex-related differences were observed in performance of the tests. The motor and cognitive functions deteriorated with ageing. TgHD animals have started to lose their weight from the age of 4 years. The data showed that the tests are safe and perspective for longitudinal studies. Moreover, tests can discern differences between tgHD and wt animals what enables to assess safety and efficacy of potential HD treatments in pre-clinical studies prior to human clinical trials.
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