Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.

Published

Journal Article

Antisense technology constitutes development of sequence-specific DNA or RNA analogs that can block the activity of selected single-stranded genetic sequences and offer the potential of high specificity lacking in many current drug treatments. The sequencing of the human genome has greatly increased the potential of this approach. Antisense oligonucleotides, the most commonly used antisense approach, are unmodified or chemically modified single stranded RNA or DNA molecules specifically designed to hybridize to corresponding RNA by Watson-Crick binding. Phosphorodiamidate Morpholino oligomers (PMO) are a novel class of non-ionic antisense agents that inhibit gene expression by binding to RNA and sterically blocking processing or translation. PMOs have shown excellent efficiency and safety profile via various routes of administration in multiple animal and human studies. This review will summarize the preclinical studies with PMOs on the road to their development as therapeutic agents with particular emphasis on in vivo biodistribution and pharmacokinetics.

Full Text

Duke Authors

Cited Authors

  • Arora, V; Devi, GR; Iversen, PL

Published Date

  • October 2004

Published In

Volume / Issue

  • 5 / 5

Start / End Page

  • 431 - 439

PubMed ID

  • 15544491

Pubmed Central ID

  • 15544491

International Standard Serial Number (ISSN)

  • 1389-2010

Language

  • eng

Conference Location

  • Netherlands