Drug and gene targeting to the brain with molecular Trojan horses

Nat Rev Drug Discov. 2002 Feb;1(2):131-9. doi: 10.1038/nrd725.


Getting drugs and genes into the brain is a tall order. This is because the presence of the blood-brain barrier prevents many molecules from crossing into the brain. Overcoming this problem will have a profound effect on the treatment of many neurological disorders, allowing larger water-soluble molecules to pass into the brain. Transport vectors, such as endogenous peptides, modified proteins or peptidomimetic monoclonal antibodies, are one way of tricking the brain into allowing these molecules to pass. This article will review such molecular Trojan Horses, and the progress that has been made in the delivery of drugs and genes to the brain.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease / drug therapy
  • Animals
  • Biological Transport
  • Blood-Brain Barrier*
  • Brain / blood supply
  • Brain / metabolism*
  • Brain Diseases / therapy*
  • Brain Neoplasms / drug therapy
  • Gene Targeting
  • Genetic Therapy*
  • Humans
  • Neuroprotective Agents / pharmacokinetics
  • Oligonucleotides, Antisense / pharmacokinetics
  • Recombinant Fusion Proteins / therapeutic use


  • Neuroprotective Agents
  • Oligonucleotides, Antisense
  • Recombinant Fusion Proteins