
Gene therapyInborn Errors of MetabolismMSUD
First in vivo success of a gene therapy for the maple syrup urine disease

Gene therapygenome editingRed blood cellsSickle cell disease
The DIM « thérapie génique » aims to federate them and accelerate the development of gene therapy to reinforce, in the light of personalized medicine, it grades among the most promising and innovative therapeutic strategies. This initiative aims to provide treatment for serious or chronic pathologies without treatment till today, or for which the medical care is still largely not satisfying.