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Gene Therapy for Fatty Acid Oxidation Deficiencies, VLCAD

Dr. Mai Elmallah from the Elmallah Lab (Horae Gene Therapy Center) is conducting research and developing therapeutic strategies for rare inherited diseases such as the Fatty Acid Oxidation Deficiencies, VLCAD


What is Fatty Acid Oxidation Deficiencies, VLCAD?

Fatty acid oxidation (FAO) deficiencies are a class of metabolic disorders, where the body is unable to oxidize breakdown fatty acids to make energy because an enzyme is either missing or not working correctly. The main source of energy for the body is a sugar called glucose. Normally when the glucose runs out, fat is broken down into energy. However, that energy is not available to children and adults with these disorders. If untreated, these disorders can lead to serious complications affecting the liver, heart, eyes and general muscle development.