Phenotypic Analyses of the HD Transgenic Minipig Model (A11609)

0501519 - ÚŽFG 2019 RIV CZ eng V - Výzkumná zpráva
Ellederová, Zdeňka
Phenotypic Analyses of the HD Transgenic Minipig Model (A11609).
CHDI Foundation, 2018. 38 s.
Grant CEP: GA MŠMT(CZ) LO1609
Institucionální podpora: RVO:67985904
Klíčová slova: minipig
Obor OECD: Genetics and heredity (medical genetics to be 3)

The transgenic Huntington's disease minipigs (TgHD) express N‐terminal part of human mutated huntingtin (124Q) under the control of human huntingtin promoter. The founder animal, born in 2009, gave birth to four subsequent generations with an equal contribution of wild‐type (WT) and transgenic (TgHD) piglets in all litters. The model is being used for preclinical huntingtin lowering studies. Here we take different non-invasive and invasive approaches, some of which are unique for large animal models, to study the phenotype development comparing WT and TgHD siblings. We show gradual progression of the disease in these TgHD animals. Moreover, some biomarkers were identified. These markers could serve for monitoring of organism response to HD treatment to assess efficacy and safety in preclinical studies prior to human clinical trials.
Trvalý link: http://hdl.handle.net/11104/0293534