Blood?brain barrier delivery of protein and non-viral gene therapeutics with molecular Trojan horses

Blood?brain barrier delivery of protein and non-viral gene therapeutics with molecular Trojan horses
Received 23 February 2007; accepted 2 April 2007. Available online 6 April 2007
William M. Pardridge, a
Journal of Controlled Release
Article in Press
Copyright ? 2007 Elsevier B.V. All rights reserved.
aDepartment of Medicine, UCLA, Warren Hall 13-164, 900 Veteran Ave., Los Angeles, CA 90024, USA
The products of biotechnology, recombinant proteins, monoclonal antibodies, antisense, RNA interference, or non-viral gene transfer, cannot be developed as pharmaceuticals for the brain, unless these molecules are re-formulated to enable transport across the blood?brain barrier (BBB). Large molecule drugs, and plasmid DNA, can be delivered across the BBB with receptor-specific molecular Trojan horses. Trojan horse BBB delivery systems, coupled with one of 3 different technology platforms (fusion proteins, avidin?biotin, or Trojan horse liposomes), can enable the BBB transport of virtually any large molecule drug or plasmid DNA.
Keywords: Blood?brain barrier; Drug targeting; Insulin receptor; Fusion protein
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