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Cellular uptake of antisense oligonucleotides

A M Tari1, G Lopez-Berestein

  • 1Department of Bioimmunotherapy, The University of Texas MD Anderson Cancer Center, Houston 77030, USA. atari@mdanderson.org

Current Opinion in Investigational Drugs (London, England : 2000)
|March 14, 2002
PubMed

Insights

Antisense oligonucleotides (AS ONs) block protein production by targeting mRNA. This review examines cell receptors involved in AS ON uptake and their bioavailability after in vivo administration.

Area of Science:

  • Molecular Biology
  • Cell Biology
  • Pharmacology

Background:

  • Antisense oligonucleotides (AS ONs) are short nucleic acid sequences designed to inhibit gene expression by binding to target messenger RNA (mRNA).
  • Cellular uptake of AS ONs is primarily mediated by endocytosis, a process involving specific cell surface receptors.
  • The efficiency and specificity of AS ON delivery depend heavily on the type and expression levels of these receptors, which vary significantly across different cell types.

Purpose of the Study:

  • To review the characteristics of various cell surface receptors involved in the in vitro uptake of antisense oligonucleotides.
  • To discuss the mechanisms of receptor-mediated endocytosis for AS ONs.
  • To explore the bioavailability and cellular uptake of AS ONs following in vivo administration.

Main Methods:

  • Literature review of in vitro studies characterizing receptors involved in AS ON cellular uptake.
  • Analysis of data on receptor expression patterns in different cell types.
  • Review of studies investigating AS ON bioavailability and tissue distribution in vivo.

Main Results:

  • Identification and characterization of diverse cell surface receptors mediating AS ON endocytosis.
  • Demonstration that receptor characteristics and expression levels are cell-type specific, influencing AS ON uptake.
  • Overview of factors affecting AS ON bioavailability and cellular penetration in vivo.

Conclusions:

  • Receptor-mediated endocytosis is a critical pathway for cellular delivery of antisense oligonucleotides.
  • Understanding receptor characteristics is essential for optimizing AS ON delivery and therapeutic efficacy.
  • Further research into receptor-ligand interactions can enhance the development of targeted AS ON therapies.

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