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Related Concept Videos

Nucleic acids02:43

Nucleic acids

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Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and carry instructions for its functioning.
DNA and RNA
The two main types of nucleic acids are deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA is the genetic material in all living organisms, ranging from single-celled bacteria to multicellular mammals. It is in the nucleus of eukaryotes and in the organelles, chloroplasts, and mitochondria. In prokaryotes,...
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Drug Delivery: Overview01:16

Drug Delivery: Overview

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The selection of a drug's delivery route depends upon its physicochemical properties, including lipid or water solubility and ionization, as well as the therapeutic requirement, such as immediate or sustained effect. These routes can be divided into three primary categories: enteral, parenteral, and topical.
Enteral delivery involves administering drugs directly through swallowing, sublingual placement, or buccal application. Orally administered drugs predominantly navigate the...
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Drug Delivery Systems: Different Types01:27

Drug Delivery Systems: Different Types

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Conventional oral drug products, termed immediate-release (IR) formulations, are engineered to promptly release their active pharmaceutical ingredient (API) upon ingestion, typically in tablets or capsules. This rapid release often results in swift drug absorption and consequent pharmacodynamic effects, although the timing and intensity can vary depending on the drug's properties. Prodrugs within these formulations require metabolic conversion to activate their pharmacodynamic effects,...
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Modified-Release Drug Delivery Systems: Bioavailability01:30

Modified-Release Drug Delivery Systems: Bioavailability

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Modified-release (MR) dosage forms are designed to extend drug release over time, thereby maintaining stable plasma concentrations and reducing dosing frequency. However, their bioavailability is typically below 100% due to incomplete drug release and presystemic metabolism, and limitations in drug permeability across the gastrointestinal epithelium, all of which can restrict the fraction of the drug reaching systemic circulation. Consequently, studying the in vivo bioavailability of MR...
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Modified-Release Drug Delivery Systems: Site-Targeted01:24

Modified-Release Drug Delivery Systems: Site-Targeted

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Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
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Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

Site-Targeted Drug Delivery Systems: Polymeric Carriers

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Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...
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Related Experiment Video

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Combining QD-FRET and Microfluidics to Monitor DNA Nanocomplex Self-Assembly in Real-Time
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Development of bioconjugate-based delivery systems for nucleic acids.

Aniket Wahane1, Vishal Kasina1, Mounika Pathuri1

  • 1Department of Pharmaceutical Sciences, University of Connecticut, Storrs, Connecticut 06269, USA.

RNA (New York, N.Y.)
|October 30, 2024
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Nucleic acid drugs offer potential for genetic diseases by modulating gene expression. Targeted delivery, using strategies like GalNAc conjugation, is key to their clinical success.

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Area of Science:

  • Biotechnology
  • Pharmacology
  • Molecular Biology

Background:

  • Nucleic acids modulate gene expression via RNAi, mRNA degradation, splice modulation, and translation blocking.
  • They offer potential for treating genetic and rare diseases by targeting aberrant gene expression.
  • Targeted delivery is a significant challenge hindering clinical adoption of nucleic acid therapeutics.

Purpose of the Study:

  • To review nucleic acid drug classes and their mechanisms of action.
  • To discuss challenges in nucleic acid drug delivery.
  • To elaborate on advances in bioconjugation strategies for targeted delivery.

Main Methods:

  • Review of existing literature on nucleic acid therapeutics.
  • Analysis of chemical modifications and bioconjugation strategies for delivery.
  • Examination of ligand development for organ and cell-specific targeting.

Main Results:

  • N-acetylgalactosamine (GalNAc) conjugation enables liver targeting, leading to FDA-approved nucleic acid drugs.
  • Various bioconjugation strategies are being explored for enhanced organ and cell targeting.
  • Diverse ligands are being developed to improve nucleic acid drug delivery.

Conclusions:

  • Bioconjugation strategies are crucial for overcoming nucleic acid drug delivery challenges.
  • Targeted delivery enhances the therapeutic potential of nucleic acid drugs.
  • Continued advances in ligand development promise broader clinical applications for nucleic acid therapeutics.