Myotonic dystrophy type 1 (DM1)-specific induced pluripotent stem cells (iPS): an innovative platform for myogenic and cardiomyogenic differentiation and validation of novel therapeutic approaches (FWOAL616)

Myotonic dystrophy type 1 (DM1) or Steinert's disease is a relatively common genetic disorder that results in a gradual degeneration of the function of the skeletal muscles and the heart. There is currently no cure or effective treatment available. The disease is caused by a genetic defect resulting in a toxic gene product. However, the exact mechanisms of this disease are not fully understood. The objectives of this project is to better understand how this toxic gene product interferes with normal cell function and to validate a new therapy aimed at neutralizing this toxic effect.
To achieve this we will first need to generate so-called "pluripotent" stem cells from patients suffering from DM1 and evaluate whether their ability to give rise to normal functional muscle or heart cells is affected. We will then assess whether we could correct these defects by destroying the toxic gene product.

Keywords:Fibrosis, Hepatic Stellate Cells, Histon (de)acetylation, Stellate cell activation, Liver Cell Transplantation, Liver Sinusoidal Cells, Sinusoidal Cells, Portal hypertension, cirrhosis, Cytoskeleton, Cell Biology, Fat-Storing Cells, NASH / NAFLD, Intermediate Filaments, liver stem / progenitor cells, Flow Cytometry, Metabolic Syndrome

Disciplines:Electrical and electronic engineering, Basic sciences, Biological sciences