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Related Experiment Video

Updated: Jun 6, 2025

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses

Published on: May 5, 2014

Nucleic acid drugs: recent progress and future perspectives.

Xiaoyi Sun¹, Sarra Setrerrahmane², Chencheng Li¹

¹Jiangsu Province Engineering Research Center of Synthetic Peptide Drug Discovery and Evaluation, China Pharmaceutical University, Nanjing, 210009, China.

Signal Transduction and Targeted Therapy

|November 28, 2024

Summary

This summary is machine-generated.

Nucleic acid drugs (NADs) offer efficient gene editing but face delivery challenges. Advances in chemical modification and delivery strategies are improving their stability, targeting, and clinical application for various diseases.

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Area of Science:

Biotechnology
Pharmacology
Molecular Biology

Background:

Therapeutic agent development faces challenges with small molecules and biologics, particularly for 'undruggable' diseases.
Nucleic acid drugs (NADs) represent a promising new generation of gene-editing modalities with high efficiency and rapid development potential.

Purpose of the Study:

To review the development, classification, and delivery strategies of nucleic acid drugs (NADs).
To highlight the clinical successes, limitations, and future potential of NADs in gene therapy.

Main Methods:

Literature review of NADs development, chemical modification techniques, and various delivery approaches.
Analysis of clinical trial data and approved therapeutic applications of NADs.

Main Results:

Chemical modifications enhance NADs' physicochemical properties, but carriers are often needed for cellular uptake and intracellular targeting.
Multiple delivery strategies have been developed to improve NADs' in vivo bioavailability and intracellular delivery.
Several NADs have progressed to clinical trials and gained regulatory approval for therapeutic use.

Conclusions:

NADs show significant promise as gene therapy candidates, overcoming limitations of traditional therapeutics.
Continued advancements in delivery systems and chemical modifications are crucial for expanding NADs' clinical applications.
NADs are evolving rapidly, offering new avenues for treating a wide range of diseases.