ANTES
Accelerating Neuromuscular Treatment Selection
| Coordinatore | FUNDACION VASCA DE INNOVACION E INVESTIGACION SANITARIAS
Organization address
address: PLAZA ASUA 1 contact info |
| Nazionalità Coordinatore | Spain [ES] |
| Totale costo | 100˙000 € |
| EC contributo | 100˙000 € |
| 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-2013-CIG |
| Funding Scheme | MC-CIG |
| Anno di inizio | 2013 |
| Periodo (anno-mese-giorno) | 2013-09-01 - 2017-08-31 |
Partecipanti
| # | ||||
|---|---|---|---|---|
| 1 |
FUNDACION VASCA DE INNOVACION E INVESTIGACION SANITARIAS
Organization address
address: PLAZA ASUA 1 contact info |
ES (SONDIKA) | coordinator | 100˙000.00 |
Mappa
Word cloud
Esplora la "nuvola delle parole (Word Cloud) per avere un'idea di massima del progetto.
Obiettivo del progetto (Objective)
'Neuromuscular disorders (NMDs) affect an estimated 300.000 people in Europe and no therapies are available for these highly incapacitating and lethal disorders. In the last few years, this status quo has shifted dramatically with the appearance of new RNA therapies targeting several of these disorders. Several clinical trials are currently underway but future development is being slowed due to several motives, mainly related to the requirement of personalised approaches for rarer mutations and to technical difficulties to evaluate the effects of these new therapies on relevant biological outcome measures.
I have been awarded a fellowship to develop a platform to streamline drug development for NMDs by combining several resources: immortalized control and patients’ cells, specific antibodies and new imaging techniques to be able to measure immunostained cultured cells directly in plate format. Duchenne muscular dystrophy (DMD) will be the first NMD that will benefit from this approach. Several therapeutic approaches aim to restore dystrophin expression in DMD: gene therapy, stem cell therapy, nonsense mutation read-through and redirection of splicing with antisense oligonucleotides (AOs). A reliable quantitative method to measure dystrophin in cell culture is not yet available and this project will develop such system, which will not only speed up development of new AOs targeting less common mutations, but also be the stepping stone that we will use to expand the platform not only to find treatments to other NMDs, but also possibly as a diagnostic tool for some of them.'