Molecular Genetics, Function & Therapy Research

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Research

 

The importance to characterise unknown gene function and develop efficient therapies for many untreated diseases imposes the need for the exploitation of novel technologies. Our mission is to investigate and develop molecular technologies in order to study gene function and design new therapeutic approaches. The focus is on the skeletal muscle and its development from single cells. More specifically our current aims are:

  1. To study the molecular pathogenesis of Myotonic Dystrophy, a common inherited neuromuscular disorder. 
  2. To investigate the pathway of commitment and differentiation of muscle cells. 
  3. To advance new gene delivery systems in mammalian cells, including muscle.

 

 

The Woodchuck post-transcriptional element enhances release of the trapped Myotonic Dystrophy transcripts from the nucleus to the cytoplasm and induces formation of multinucleated myotubes. Cells that still retain the mutant RNA foci inside the nucleus fail to differentiate, as detected by fluorescence in situ hybridisation.
Efficient transfection of differentiated muscle cells with adenoviral vectors.