SMA Europe Call for Projects 2011

Folgende Projekte werden gefördert:

• Matthew Wood, Universität Oxford, Großbritannien
  

  “Exosome delivery of splice switching oligonucleotides for the treatment of SMA”

• Umrao Monani, Columbia Universität, USA
  

  “Investigating novel genetic determinants of the SMA phenotype”

• Tilman Achsel, Universität von Leuven, Belgien
  

  “mRNA transport in SMA pathogenesis”

• Markus Rießland, Universität zu Köln, Deutschland“
  

  Molecular and functional analysis of a new modifier of SMA”Spinal muscular atrophy (SMA) is the most common hereditary cause of early infant death. There is no cure available for this very frequent genetic disorder (heterozygousity frequency 1:35). Very few individuals – although carrying the genetic predisposition for SMA – stay asymptomatic. In this project we aim to functionally characterize a novel SMA protective modifying factor. Our research – based on common model organisms for SMA – will bring deeper insight into the pathology of SMA and will open new ways for therapies for neurodegenerative disorders.

• Yannick Tanguy, L’Institut de Myologie, Paris, Frankreich
  

  “New approaches to drug discovery in SMA: using RNA secondary structure as a new therapeutic target”

• Kathleen Mahias, Universität Rouen, Frankreich
  

  “Evaluation of the contribution of SMN1 and SMN2 sequence variants to the clinical severity of SMA” (Anschlussförderung)