Work

1086. T Cell Responses to Canine Factor IX and AAV Capsid Antigens in Hemophilia B Dogs after Intravascular Gene Delivery to Skeletal Muscle

Year: 2006

Type: article

Abstract: Previous studies have shown successful correction of hemophilia B in dogs by muscle-directed gene delivery of canine F.IX (cF.IX) using an adeno-associated virus (AAV) serotype 2. Intravascular delivery of the cF.IX gene to skeletal muscle allowed long-term expression of cF.IX of 4-20%, thereby correcting the disease phenotype. However, concerns over the risk of immune responses against the transgene and viral vector antigens have focused recent work on further characterizing the immunogenicity of both AAV capsid and F.IX in a gene therapy setting.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2006.08.1188