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"Muu (välisriiklik avalik sektor)" projekt VMVBS19195
VMVBS19195 "Lifelong treatment with GLP 1 receptor agonist in rat model of Wolfram syndrome (1.03.2019−28.02.2022)", Mario Plaas, Tartu Ülikool, Meditsiiniteaduste valdkond, bio- ja siirdemeditsiini instituut.
VMVBS19195
Lifelong treatment with GLP 1 receptor agonist in rat model of Wolfram syndrome
Lifelong treatment with GLP 1 receptor agonist in rat model of Wolfram syndrome
1.03.2019
28.02.2022
Teadus- ja arendusprojekt
Muu (välisriiklik avalik sektor)
ETIS klassifikaatorAlamvaldkondCERCS klassifikaatorFrascati Manual’i klassifikaatorProtsent
3. Terviseuuringud3.1. BiomeditsiinB470 Füsioloogia 3.1 Biomeditsiin100,0
AsutusRiikTüüp
Eye Hope Foundation |VZWBelgia Kuningriikfond
PerioodSumma
01.03.2019−28.02.2022194 396,25 EUR
194 396,25 EUR

Wolfram syndrome (WS) is a rare autosomal recessive disease that is caused by mutations in WFS1 gene. The syndrome first manifests as diabetes mellitus, followed by optic nerve atrophy, deafness and neurodegeneration. Underlying mechanism is believed to be a dysregulation of endoplasmic reticulum (ER) stress response, which ultimately leads to cellular death. Treatment with GLP1 receptor agonists has been shown to normalize ER stress response in several in vitro and in vivo models. Recent research has shown beneficial effect of GLP1 receptor agonist (RA) treatment in rat and mouse models of WS . Early treatment with Liraglutide was effective to prevent the development of diabetic phenotype in a rat model of WS ( https://www.nature.com/articles/s41598-018-28314-z ). Our preliminary results indicate that liraglutide has also neuroprotective effect in a rat model of WS. WS is a lifelong condition and therefore, any pharmacological treatment of WS patients will also be life-long. GLP1 receptor agonist treatment is currently the only known treatment strategy that was shown to be effective in several animal models of WS and in a human patient. However, the long-lasting effect of such treatment has never been evaluated. The aim of this project is to evaluate the safety and efficacy of life-long GLP1 RA treatment on progression of WS in a rat model.
KirjeldusProtsent
Rakendusuuring100,0