ADENO-ASSOCIATED VIRUS FACTOR VIII VECTORS, ASSOCIATED VIRAL PARTICLES AND THERAPEUTIC FORMULATIONS COMPRISING THE SAME

摘要

The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)- encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.