Ultragenyx Pharmaceutical Inc.
GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA

Abstract:

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV), and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a recombinant adeno-associated virus of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical (compositions may be useful in gene therapy for the treatment of PA caused by mutations in propionyl-CoA carboxylase .alpha.-subunit (PCCA) or mutations in propionyl-CoA carboxylase .beta.-subunit (PCCB).