Phenotypic Analyses of the HD Transgenic Minipig Model (A11609)

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F67985904%3A_____%2F18%3A00501519" target="_blank" >RIV/67985904:_____/18:00501519 - isvavai.cz</a>

Výsledek na webu

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DOI - Digital Object Identifier

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Jazyk výsledku

angličtina

Název v původním jazyce

Phenotypic Analyses of the HD Transgenic Minipig Model (A11609)

Popis výsledku v původním jazyce

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.

Název v anglickém jazyce

Phenotypic Analyses of the HD Transgenic Minipig Model (A11609)

Popis výsledku anglicky

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.

Druh

O - Ostatní výsledky

CEP obor

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OECD FORD obor

10603 - Genetics and heredity (medical genetics to be 3)

Projekt

<a href="/cs/project/LO1609" target="_blank" >LO1609: Modely závažných lidských onemocnění: Traumatické poškození míchy, Huntingtonova choroba, melanom a neplodnost</a><br>

Návaznosti

P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)

Rok uplatnění

2018

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů