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Hemophilia A

Nonviral Delivery Of Factor VIII Is Safe And Well-Tolerated

Published in Blood Weekly, July 26th, 2001

by Deborah W. Heinrich, PhD, staff medical writer - Phase I trials began for nonviral gene delivery of factor VIII in the treatment of severe hemophilia A.

Though viral vectors are efficient modes of gene transfer, there are certain drawbacks to their use in a clinical setting. Some virus vectors induce strong host immunity to viral gene products resulting in loss of transgene expression. Others that integrate into the host genome may disrupt critical cell growth regulators. Still others may recombine with naturally occurring viruses and regain recombination and reproductive capacities. This may result in systemic infection and viral pathogenesis.

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