Pharmacokinetic and pharmacodynamic considerations in gene therapy

Hiroyuki Kamiya, Hidetaka Akita, Hideyoshi Harashima

Research output: Contribution to journalReview articlepeer-review

111 Citations (Scopus)


During gene therapy the concentration of plasmid DNA or oligonucleotides in the plasma can be quite different from their concentrations in the nucleus or cytosol where they exert their actions. For a better understanding of the apparent discrepancies between pharmacokinetics (PK) and pharmacodynamics (PD), a new concept for intracellular PK with an emphasis on the final efficacy of gene transcription is needed. Here, the conventional PK and intracellular PK and PD of non-viral gene delivery systems are discussed, together with a new concept, referred to as controlled intracellular disposition, which integrates these factors to gain a better understanding of gene expression in the nucleus. The importance of optimizing the system from a transcriptional point of view in the nucleus is also discussed. These new concepts must be integrated to develop an optimized non-viral gene delivery system.

Original languageEnglish
Pages (from-to)990-996
Number of pages7
JournalDrug Discovery Today
Issue number21
Publication statusPublished - 2003 Nov 1
Externally publishedYes


  • Biochemistry
  • Cell biology
  • Controlled nuclear disposition
  • Drug Discovery
  • Gene delivery system
  • Intracellular pharmacokinetics
  • Molecular Medicine
  • Non-viral gene delivery
  • Pharmaceutical Science
  • Pharmacodynamics
  • Pharmacokinetics

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery


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