NOVEL ADENO-ASSOCIATED VIRAL (AAV) VECTORS TO TREAT HEREDITARY METHYLMALONIC ACIDEMIA (MMA) CAUSED BY METHYLMALONYL-COA MUTASE (MMUT) DEFICIENCY

摘要

The present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV) and methods of using such vectors and viruses in gene therapy for treating methylmalonic acidemia in patients with methylmalonyl-coA mutase (MMUT) deficiency. Also provided are pharmaceutical compositions comprising recombinant adeno-associated virus (rAAV) and a pharmaceutically acceptable carrier or excipient.