The astrocyte nanofilament system in Alexander disease – from molecules to function, uncovering new leads for therapy

Název projektu anglicky
The astrocyte nanofilament system in Alexander disease – from molecules to function, uncovering new leads for therapy
Anotace anglicky
Alexander disease (AxD) is a rare genetic brain disease with no cure. AxD patients show white matter degeneration and extensive loss of brain tissue. Symptoms include mental retardation, epilepsy, muscle stiffness, and AxD leads to death. About 500 AxD cases have been reported worldwide. AxD is caused by mutations in GFAP, an astrocyte intermediate filament (nanofilament) protein. The disease starts in astrocytes, cells that control many neuronal functions and homeostatic mechanisms of the brain. However, it is not known how AxD astrocytes cause the disease, and the available animal models are imperfect. ALEXANDER consortium partners have complementary expertise, and unique technologies to determine the cellular and molecular mechanisms leading to AxD and to design future treatment strategies. We will use induced pluripotent stem cells from AxD patients to derive astrocytes, neurons, and other glial cell types and to generate brain organoids. We will also generate an AxD killifish model. We will elucidate the effects of AxD astrocytes on neurons, and other aspects of AxD pathogenesis.

Kategorie VaV
ZV - Základní výzkum
OECD FORD - hlavní obor
30502 - Other medical science
OECD FORD - vedlejší obor
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OECD FORD - další vedlejší obor
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CEP - odpovídající obory <br>(dle <a href="http://www.vyzkum.cz/storage/att/E6EF7938F0E854BAE520AC119FB22E8D/Prevodnik_oboru_Frascati.pdf">převodníku</a>)
FP - Ostatní lékařské obory

Důvěrnost údajů
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Systémové označení dodávky dat
CEP24-MSM-8F-R
Datum dodání záznamu
19. 2. 2024

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