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Related Concept Videos

Therapeutic Index01:13

Therapeutic Index

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The therapeutic index of a drug is a key parameter in pharmacology that quantifies the relative safety of a drug by calculating the ratio between the dose that causes toxicity in half the population (50%) to the dose that proves to be effective for half the population (50%). It provides a spectrum of doses for a particular drug ranging from effective to potentially toxic. To illustrate, consider an anticoagulant agent like warfarin. It possesses a narrow window within its therapeutic index to...
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Development of New Therapeutic Applications Using Microfluidics
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Bioconjugated Oligonucleotides: Recent Developments and Therapeutic Applications.

Sebastien Benizri1,2,3, Arnaud Gissot1,2,3, Andrew Martin4

  • 1Inserm U1212 , F-33076 Bordeaux , France.

Bioconjugate Chemistry
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Bioconjugated oligonucleotides, like antisense DNA (ASO) and RNA interference (RNAi), are promising future therapeutics. Novel conjugates enhance drug delivery and efficacy, advancing oligonucleotide-based treatments for various diseases.

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Area of Science:

  • Therapeutic oligonucleotide development
  • Chemical biology and drug delivery

Background:

  • Oligonucleotide-based agents represent a key future therapeutic class.
  • Bioconjugated oligonucleotides are transitioning from research to clinical application.

Purpose of the Study:

  • To review gene inhibition strategies using oligonucleotides (ASO and RNAi).
  • To analyze recent advancements in bioconjugated oligonucleotides and their clinical translation.
  • To discuss the future prospects and limitations of oligonucleotide therapeutics.

Main Methods:

  • Description of antisense DNA (ASO) and RNA interference (RNAi) gene inhibition.
  • Summary and analysis of various bioconjugated oligonucleotide modifications (e.g., GalNAc, peptides, lipids).
  • Review of approved and investigational bioconjugated oligonucleotides in clinical trials.

Main Results:

  • Novel conjugates enhance tissue targeting, cellular uptake, and nuclease resistance.
  • Several bioconjugated oligonucleotides are approved or in clinical trials.
  • Bioconjugation chemistry is central to therapeutic oligonucleotide advancements.

Conclusions:

  • Bioconjugated oligonucleotides offer significant potential for treating diverse diseases.
  • Opportunities exist in developing new chemistries and translating these agents to the clinic.
  • Further research in chemical biology and mechanistic studies is crucial.