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Title: Angiogenesis enhances factor IX delivery and persistence from retrievable human bioengineered muscle implants
Authors: Thorrez, Lieven
Vandenburgh, Herman
Callewaert, Nico
Mertens, Nico
Shansky, Janet
Wang, Lin
Arnout, Jozef
Collen, Desire
Chuah, Marinee
Vandendriessche, Thierry # ×
Issue Date: Sep-2006
Publisher: Academic Press
Series Title: Molecular Therapy vol:14 issue:3 pages:442-451
Abstract: Human muscle progenitor cells transduced with lentiviral vectors secreted high levels of blood clotting factor IX (FIX). When bioengineered into postmitotic myofibers as human bioartificial muscles (HBAMs) and subcutaneously implanted into immunodeficient mice, they secreted FIX into the circulation for >3 months. The HBAM-derived FIX was biologically active, consistent with the cells' ability to conduct the necessary posttranslational modifications. These bioengineered muscle implants are retrievable, an inherent safety feature that distinguishes this "reversible" gene therapy approach from most other gene therapy strategies. When myofibers were bioengineered from human myoblasts expressing FIX and vascular endothelial growth factor, circulating FIX levels were increased and maintained long term within the therapeutic range, consistent with the generation of a vascular network around the HBAM. The present study implicates an important role for angiogenesis in the efficient delivery of therapeutic proteins using tissue engineered stem cell-based gene therapies.
URI: 
ISSN: 1525-0016
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Molecular and Vascular Biology
ESAT - STADIUS, Stadius Centre for Dynamical Systems, Signal Processing and Data Analytics
Biomedical Sciences Group Management - miscellaneous
× corresponding author
# (joint) last author

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