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

Publication ,  Journal Article
Arora, V; Devi, GR; Iversen, PL
Published in: Curr Pharm Biotechnol
October 2004

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.

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Published In

Curr Pharm Biotechnol

DOI

ISSN

1389-2010

Publication Date

October 2004

Volume

5

Issue

5

Start / End Page

431 / 439

Location

Netherlands

Related Subject Headings

  • Oligonucleotides, Antisense
  • Humans
  • Genetic Therapy
  • Drug Delivery Systems
  • Carbohydrate Sequence
  • Biotechnology
  • Animals
  • 3214 Pharmacology and pharmaceutical sciences
  • 3206 Medical biotechnology
  • 1115 Pharmacology and Pharmaceutical Sciences
 

Citation

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Arora, V., Devi, G. R., & Iversen, P. L. (2004). Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol, 5(5), 431–439. https://doi.org/10.2174/1389201043376706
Arora, Vikram, Gayathri R. Devi, and Patrick L. Iversen. “Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.Curr Pharm Biotechnol 5, no. 5 (October 2004): 431–39. https://doi.org/10.2174/1389201043376706.
Arora V, Devi GR, Iversen PL. Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol. 2004 Oct;5(5):431–9.
Arora, Vikram, et al. “Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.Curr Pharm Biotechnol, vol. 5, no. 5, Oct. 2004, pp. 431–39. Pubmed, doi:10.2174/1389201043376706.
Arora V, Devi GR, Iversen PL. Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol. 2004 Oct;5(5):431–439.

Published In

Curr Pharm Biotechnol

DOI

ISSN

1389-2010

Publication Date

October 2004

Volume

5

Issue

5

Start / End Page

431 / 439

Location

Netherlands

Related Subject Headings

  • Oligonucleotides, Antisense
  • Humans
  • Genetic Therapy
  • Drug Delivery Systems
  • Carbohydrate Sequence
  • Biotechnology
  • Animals
  • 3214 Pharmacology and pharmaceutical sciences
  • 3206 Medical biotechnology
  • 1115 Pharmacology and Pharmaceutical Sciences