POLYQ MUTANT AR/SBMA

SBMA as a model of polyglutamine diseases: generation of a suitable cell system to study the post-transcriptional modifications of mutant androgen receptor and to discover potential therapeutic drugs

 Coordinatore FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA 

 Organization address address: VIA MOREGO 30
city: GENOVA
postcode: 16163

contact info
Titolo: Ms.
Nome: Claudia
Cognome: Schiaffino
Email: send email
Telefono: 3971781757

 Nazionalità Coordinatore Italy [IT]
 Totale costo 178˙760 €
 EC contributo 178˙760 €
 Programma FP7-PEOPLE
Specific programme "People" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013)
 Code Call FP7-PEOPLE-2011-IOF
 Funding Scheme MC-IOF
 Anno di inizio 2013
 Periodo (anno-mese-giorno) 2013-04-01   -   2015-03-31

 Partecipanti

# participant  country  role  EC contrib. [€] 
1    FONDAZIONE ISTITUTO ITALIANO DI TECNOLOGIA

 Organization address address: VIA MOREGO 30
city: GENOVA
postcode: 16163

contact info
Titolo: Ms.
Nome: Claudia
Cognome: Schiaffino
Email: send email
Telefono: 3971781757

IT (GENOVA) coordinator 178˙760.70

Mappa


 Word cloud

Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.

dependent    model    molecular    aggregates    sbma    ar    ligand    diseases    pka    therapy    phosphorylation    protein    disease    toxicity    polyglutamine    hallmark    mutant    differentiated    camp    disorders    neurodegenerative    accumulation    modifications   

 Obiettivo del progetto (Objective)

'Accumulation of toxic aggregation-prone protein into inclusions and aggregates is the hallmark of most neurodegenerative diseases, a broad class of disorders including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis and polyglutamine diseases. In this proposal, using SBMA as a model of neurodegenerative polyglutamine-related disorder, we intend to move a step forward expanding knowledge of the neurodegeneration. In particular, we will provide a suitable model system such as differentiated iPSCs from SBMA patients to study molecular mechanism and to find out potential therapeutic drugs for neurodegenerative diseases. To define the disease-phenotype of differentiated iPS cells we will take advantage of specific features of SBMA disorders: the ligand-dependent toxicity of expanded AR. The ligand binding induces many modifications of AR protein such as phosphorylation. Since phosphorylation of mutant AR is an important determinant of SBMA pathogenesis, we will also characterize the impact of cyclic adenosine monophosphatate (cAMP)-dependent protein kinase A (PKA) signaling on the mutant AR toxicity. Then, we will use this information to identify agents that promote such modifications to find out potential therapy. Therefore, in order to develop treatment for SBMA, we will test the effect of neuropeptides that stimulates the generation of cAMP and activation of PKA, to target SBMA spinal cord.'

Introduzione (Teaser)

The hallmark of most neurodegenerative disorders is the aberrant accumulation of protein aggregates. Identifying the molecular aetiology of these disorders is the only way to advance effective therapy.

Altri progetti dello stesso programma (FP7-PEOPLE)

TALENTS (2010)

Talents for an International House

Read More  

RIANS (2010)

Rydberg Interactions at Nanostructured Surfaces

Read More  

ODYSNIGHT (2008)

Olsztyn Science Days Researchers Night 2008

Read More