Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice

L Xu, M Mei, ME Haskins, TC Nichols, P O'donnell… - Thrombosis research, 2007 - Elsevier
INTRODUCTION: Gene therapy could prevent bleeding in hemophilia. However, antibodies
could inhibit coagulation, while cytotoxic T lymphocytes could destroy modified cells. The
immaturity of the newborn immune system might prevent these immune responses from
occurring after neonatal gene therapy. MATERIALS AND METHODS: Newborn dogs, cats,
or mice were injected intravenously with a retroviral vector expressing human Factor IX.
Plasma was evaluated for antigen and anti-human Factor IX antibodies. Cytotoxic T …