Article
Correction of murine hemophilia A by hematopoietic stem cell gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2005
Moayeri Morvarid, Hawley Teresa S, Hawley Robert G
Abstract excerpt
A serious complication of current protein replacement therapy for hemophilia A patients with coagulation factor VIII (FVIII) deficiency is the frequent development of anti-FVIII inhibitor antibodies that preclude therapeutic benefit from further treatment. Induction of tolerance by persistent high-level FVIII synthesis following transplantation with hematopoietic stem cells expressing a retrovirally delivered...
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