Blood-brain barrier delivery of protein and non-viral gene therapeutics with molecular Trojan horses.
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.
Pardridge
William M
WM
Department of Medicine, UCLA, Warren Hall 13-164, 900 Veteran Ave., Los Angeles, CA 90024, USA. wpardridge@mednet.ucla.edu
D000818:Animals; D000911:Antibodies, Monoclonal / Q000737:chemistry*; D001665:Binding Sites; D001812:Blood-Brain Barrier / Q000378:metabolism*; D004247:DNA / Q000008:administration & dosage* / Q000493:pharmacokinetics; D004337:Drug Carriers / Q000737:chemistry*; D006801:Humans; D008081:Liposomes; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage* / Q000493:pharmacokinetics; D013045:Species Specificity
D000911:Antibodies, Monoclonal; D004337:Drug Carriers; D008081:Liposomes; D011993:Recombinant Fusion Proteins; D004247:DNA