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Related Experiment Videos

Oligonucleotide therapy.

S T Crooke1

  • 1Isis Pharmaceuticals, Carlsbad, California 92008.

Current Opinion in Biotechnology
|December 1, 1992
PubMed
Summary
This summary is machine-generated.

Oligonucleotide therapeutics show rapid advancement, with new insights into medicinal chemistry and pharmacodynamics. First-generation analogs, including phosphorothioates, are progressing, with one entering clinical trials.

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Area of Science:

  • Medicinal Chemistry
  • Pharmacology
  • Biotechnology

Background:

  • Oligonucleotide therapeutics represent a rapidly advancing field.
  • Established programs are demonstrating increasing productivity.
  • Recent progress builds upon foundational work from the past four years.

Purpose of the Study:

  • To summarize recent advances in oligonucleotide therapeutics.
  • To highlight progress in medicinal chemistry and understanding of oligonucleotide properties.
  • To report on the development and clinical progression of oligonucleotide analogs.

Main Methods:

  • Review of recent scientific literature and program updates.
  • Analysis of advances in oligonucleotide medicinal chemistry.
  • Evaluation of pharmacokinetic, pharmacodynamic, and toxicologic studies.

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  • Assessment of combinatorial approaches for target identification.

    • Main Results:

    • Significant advances in oligonucleotide medicinal chemistry and pharmacodynamics.
    • Improved understanding of pharmacokinetic and toxicologic properties of first-generation analogs, particularly phosphorothioates.
    • One oligonucleotide, ISIS 2105, has entered clinical trials.
    • Development of combinatorial methods for identifying novel oligonucleotide binders.

    Conclusions:

    • The field of oligonucleotide therapeutics is experiencing substantial growth and productivity.
    • Key areas of advancement include drug design, property understanding, and clinical translation.
    • Emerging technologies like combinatorial approaches promise further expansion of therapeutic targets.