The AMDA is excited to announce that the 2012 AMDA Research Grant Recipient is Dr. Nuno Raimundo of the University of Goettingen in Germany for his Project: “Mitochondria and Autophagy as Therapeutic Targets for Acid Maltase Deficiency.”
Lysosomes are responsible for recycling damaged cellular material. Lysosomal storage diseases like Pompe Disease arise from biochemical deficiencies in the recycling machinery of the lysosomes. It is becoming increasingly clear that deficits in lysosomal performance result in impairment of other cellular organelles, particularly mitochondria, which further contributes to the pathology of lysosomal storage disorders.
The long-term goal of Dr. Raimundo’s project is to improve the skeletal muscle phenotypes of Pompe’s disease by enhancing both the recycling capacity of lysosomes, and the generation of new mitochondria, using both biological manipulations and pharmacologic approaches with FDA-approved drugs. This strategy will increase the capacity of lysosomes to clear up damaged cellular components, and increase the amount of “healthy” mitochondria.