Acyl-CoA dehydrogenase substrate promiscuity limits the potential for development of substrate reduction therapy in disorders of valine and isoleucine metabolism
S. M. Houten, T. Dodatko, W. Dwyer, H. Chen, B. Stauffer, R. J. DeVita, F. M. Vaz, C. Yu, J. Leandro
This study investigates whether inhibiting ACAD8 and SBCAD can reduce toxic metabolite accumulation in valine and isoleucine metabolism disorders, finding that MCPA inhibits multiple ACADs and partially rescues lipoylation defects in ECHS1-deficient cells.
bioRxiv · Nov 22, 2022Biochemistry