Mid-Term Report Summary - MITCARE (Mitochondrial Medicine: developing treatments of OXPHOS-defects in recombinant mammalian models.)
MitCare is a multidisciplinary project aimed at developing rational treatments for mitochondrial disorders, a group of as yet incurable conditions in humans characterised by failure of mitochondria to produce enough energy to sustain the efficient activity of critical organs such as brain, heart, or skeletal muscle. In recombinant in vivo models carrying specific defects of mitochondrial bioenergetics machinery, we have shown that remarkable amelioration of the clinical and biochemical phenotypes can be achieved by using drugs that increase mitochondrial biogenesis, or by correcting the disarray of the mitochondrial network and the deconstruction of the energy producing biochemical machinery, or by replacing the missing gene by using ad hoc viral vector, called aden-associated viruses, that are per se virtually harmless in mammalian organisms. These encouraging results constitute the basis to further develop the MitCare project in the next years. The ultimate scope of this research is to offer proof of principles evidence which will help transfer the most promising results and methods to the treatment of patients affected by mitochondrial disease.
James, Irina (Unit Manager)
Record Number: 183343 / Last updated on: 2016-05-27
Information source: SESAM