[HTML][HTML] Factors influencing therapeutic efficacy and the host immune response to helper‐dependent adenoviral gene therapy in hemophilia A mice
BD Brown, CX Shi, FEM Rawle, S Tinlin… - Journal of Thrombosis …, 2004 - Elsevier
Background: Adenoviral‐based methods of gene therapy have been ineffective at providing
sustained factor (F) VIII expression in outbred populations of large animal hemophilic
models primarily due to the immunogenicity of these vectors. Improvements have been
made in vector design leading to the development of the helper‐dependent adenoviral (HD)
system. Unfortunately, it remains unclear whether these modifications are sufficient to
circumvent the induction of inhibitor formation associated with adenoviral gene transfer …
sustained factor (F) VIII expression in outbred populations of large animal hemophilic
models primarily due to the immunogenicity of these vectors. Improvements have been
made in vector design leading to the development of the helper‐dependent adenoviral (HD)
system. Unfortunately, it remains unclear whether these modifications are sufficient to
circumvent the induction of inhibitor formation associated with adenoviral gene transfer …