New therapeutic strategies for hemoglobinopathies an other hereditary anemias. From the bench to the bed side.
The aim of the course is to deepen the knowledge of how new therapeutic strategies develop from the understanding of pathophysiology and molecular basis of genetic pathologies (i.e. thalassemia syndromes, sickle cell disease and other hereditary anemias). In particular, the development of gene therapy, of new pharmacological molecules that target pathways involved in the development of the disease and the role of modifier genes as potential therapeutic targets will be addressed.
Giudizio di approvazione