Large Animal Models of Huntington's Disease: What We Have Learned and Where We Need to Go Next

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F67985904%3A_____%2F20%3A00537797" target="_blank" >RIV/67985904:_____/20:00537797 - isvavai.cz</a>

Výsledek na webu

<a href="https://content.iospress.com/articles/journal-of-huntingtons-disease/jhd200425" target="_blank" >https://content.iospress.com/articles/journal-of-huntingtons-disease/jhd200425</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.3233/JHD-200425" target="_blank" >10.3233/JHD-200425</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Large Animal Models of Huntington's Disease: What We Have Learned and Where We Need to Go Next

Popis výsledku v původním jazyce

Genetically modified rodent models of Huntington's disease (HD) have been especially valuable to our under-standing of HD pathology and the mechanisms by which the mutant HTT gene alters physiology. However, due to inherent differences in genetics, neuroanatomy, neurocircuitry and neurophysiology, animal models do not always faithfully or fully recapitulate human disease features or adequately predict a clinical response to treatment. Therefore, conducting translational studies of candidate HD therapeutics only in a single species (i.e. mouse disease models) may not be sufficient. Large animal models of HD have been shown to be valuable to the HD research community and the expectation is that the need for translational studies that span rodent and large animal models will grow. Here, we review the large animal models of HD that have been created to date, with specific commentary on differences between the models, the strengths and disadvantages of each, and how we can advance useful models to study disease pathophysiology, biomarker development and evaluation of promising therapeutics.

Název v anglickém jazyce

Large Animal Models of Huntington's Disease: What We Have Learned and Where We Need to Go Next

Popis výsledku anglicky

Genetically modified rodent models of Huntington's disease (HD) have been especially valuable to our under-standing of HD pathology and the mechanisms by which the mutant HTT gene alters physiology. However, due to inherent differences in genetics, neuroanatomy, neurocircuitry and neurophysiology, animal models do not always faithfully or fully recapitulate human disease features or adequately predict a clinical response to treatment. Therefore, conducting translational studies of candidate HD therapeutics only in a single species (i.e. mouse disease models) may not be sufficient. Large animal models of HD have been shown to be valuable to the HD research community and the expectation is that the need for translational studies that span rodent and large animal models will grow. Here, we review the large animal models of HD that have been created to date, with specific commentary on differences between the models, the strengths and disadvantages of each, and how we can advance useful models to study disease pathophysiology, biomarker development and evaluation of promising therapeutics.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

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

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2020

Kód důvěrnosti údajů

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

Název periodika

Journal of Huntington´s Disease

ISSN

1879-6397

e-ISSN

—

Svazek periodika

9

Číslo periodika v rámci svazku

3

Stát vydavatele periodika

NL - Nizozemsko

Počet stran výsledku

16

Strana od-do

201-216

Kód UT WoS článku

000578311100001

EID výsledku v databázi Scopus

2-s2.0-85092944595