This antioxidant therapy reduces reactive oxygen levels in cells to treat propionic and methylmalonic acidemia. Propionic and methylmalonic acidemia prevent the metabolic processing of certain proteins and can cause life threatening conditions including neonatal intermittent acidosis, hyperammonemia, and metabolic stroke. Long-term health effects of these diseases include chronic malnutrition, cardiomyopathy and neurological disorders. Propionic and methylmalonic acidemia are both uncommon in the general population but occur at relatively high rates in Amish and Mennonite communities where they present serious health risks for newborns and adults. Current treatments for propionic and methylmalonic acidemias include liver transplantation and extremely limited dietary protein intake, both of which include serious health risks and can cause long-term, severe side effects.
Description
Researchers at the University of Pittsburgh have developed a new, non-invasive treatment for propionic and methylmalonic acidemia. Propionic acidemia is caused by a lack of the enzyme, propionyl-CoA carboxylase, which is key in metabolizing certain components of proteins and fatty-acid chains. Without this enzyme, reactive oxygen levels increase in cell mitochondria. This therapy is composed of mitochondria-targeted antioxidants that reduce levels of reactive oxygen in cells, thereby limiting the oxidation damage resulting from propionic and methylmalonic acidemia. By reducing reactive oxygen levels in cells, this therapy can mitigate physiological damage caused by these diseases.
Applications
· Mitochondria-targeted antioxidants that reduce levels of reactive oxygen in cells to treat propionic and methylmalonic acidemia
Advantages
· New, non-invasive and simple treatment option for life-threatening propionic and methylmalonic acidemia
· Mitigates long-term health consequences of propionic and methylmalonic acidemia by reducing reactive oxygen levels in the mitochondria
Invention Readiness
In vivo data
IP Status
https://patents.google.com/patent/US20220202800A1
