Targeted delivery of protein and gene medicines through the blood-brain barrier

Clin Pharmacol Ther. 2015 Apr;97(4):347-61. doi: 10.1002/cpt.18. Epub 2014 Dec 15.


The development of biologic drugs (recombinant proteins, therapeutic antibodies, peptides, nucleic acid therapeutics) as new treatments of brain disorders has been difficult, and a major reason is the lack of transport through the blood-brain barrier (BBB) of these large molecule pharmaceuticals. Biologic drugs can be re-engineered as brain-penetrating neuropharmaceuticals using BBB molecular Trojan horse technology. Certain peptidomimetic monoclonal antibodies that target endogenous receptors on the BBB, such as the insulin or transferrin receptor, enable the re-engineering of biologic drugs that cross the BBB.

Publication types

  • Review

MeSH terms

  • Animals
  • Biological Products / therapeutic use
  • Blood-Brain Barrier / drug effects*
  • Blood-Brain Barrier / metabolism
  • Drug Delivery Systems / methods*
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Humans
  • Recombinant Proteins / pharmacokinetics
  • Recombinant Proteins / therapeutic use*


  • Biological Products
  • Recombinant Proteins