AAVrh10-PCCA

AAVrh10-PCCA is an adeno-associated virus (AAV) gene therapy developed by the Mayo Clinic for the treatment of propionic acidemia (PA). It utilizes a replication-deficient AAV serotype rh.10 vector to deliver a functional copy of the human PCCA cDNA, which encodes the alpha subunit of the mitochondrial enzyme propionyl-CoA carboxylase (PCC). PA is a rare metabolic disorder caused by mutations in either the PCCA or PCCB genes, leading to a deficiency in PCC activity and the toxic accumulation of propionyl-CoA and its metabolites. By intravenously administering the vector, the therapy aims to restore PCC enzyme activity in the liver and other tissues, thereby reducing metabolic crises and improving clinical outcomes in affected individuals.