Obiettivo
BIOIMAGE-NMD will develop and apply imaging technology to monitor response to novel therapies in neuromuscular diseases (NMD) and will use Duchenne muscular dystrophy (DMD) as an exemplar disease. DMD is well characterised genetically and clinically but to date a disease modifying treatment is not available. One of the most promising developments for future treatment of NMD is RNA modulation through antisense oligonucleotides (AON). In DMD, AON are used for exon skipping and this is a genuine example of personalised medicine, where patients are treated according to their specific gene mutation.
Quantitative Magnetic Resonance Imaging (MRI) and Magnetic Resonance Spectroscopic Imaging (MRSI) are used to assess structural and metabolic muscle pathology in NMD but their effectiveness in monitoring therapy is yet to be shown. The project will apply a simultaneous MRI/MRSI protocol in multi-centre clinical trials of AON therapy in DMD with the aim of establishing a clinical proof of principle that these imaging measures are effective biomarkers of therapeutic response. To enhance the imaging protocol, novel Diffusion Tensor MRI (DTI) methods will be developed and optimised to assess muscle microstructure and applied in these trials. The project will also develop methods for radiolabelling of AON and demonstrate the use of pre-clinical Positron Emission Tomography (PET)/MRI to assess the tissue targeting, bio-distribution and pharmacokinetics of AON in vivo.
BIOIMAGE-NMD will deliver PET/MRI and MRI/MRSI technologies for both drug development and clinical evaluation roles which will significantly contribute to bringing personalized therapeutic interventions in rare and common diseases to the market.
Campo scientifico (EuroSciVoc)
CORDIS classifica i progetti con EuroSciVoc, una tassonomia multilingue dei campi scientifici, attraverso un processo semi-automatico basato su tecniche NLP.
CORDIS classifica i progetti con EuroSciVoc, una tassonomia multilingue dei campi scientifici, attraverso un processo semi-automatico basato su tecniche NLP.
- scienze mediche e della salutemedicina di basefarmacologia e farmaciascoperta di farmaci
- scienze mediche e della salutemedicina di baseneurologiadistrofie muscolaridistrofia muscolare di duchenne
- scienze mediche e della salutescienze della salutemedicina personalizzata
- scienze mediche e della salutemedicina di basefarmacologia e farmaciafarmacocinetica
- ingegneria e tecnologiaingegneria medicadiagnostica per immaginirisonanza magnetica per immagini
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Argomento(i)
Invito a presentare proposte
FP7-HEALTH-2013-INNOVATION-1
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Meccanismo di finanziamento
CP-FP - Small or medium-scale focused research projectCoordinatore
NE1 7RU Newcastle Upon Tyne
Regno Unito