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Related Experiment Video

Updated: Jun 27, 2026

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Published on: June 21, 2013

Targeting the lung using siRNA and antisense based oligonucleotides.

Sterghios A Moschos¹, Karen Spinks, Andrew E Williams

¹Respiratory Research Group, Wythenshawe Hospital, School of Translational Medicine, University of Manchester, M23 9LT, UK.

Current Pharmaceutical Design

|December 17, 2008

Summary

The lung is a promising target for oligonucleotide therapeutics via inhalation. This review explores challenges in antisense and siRNA delivery, stability, and immune responses for effective lung-targeted treatments.

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Last Updated: Jun 27, 2026

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

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Published on: June 21, 2013

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

Area of Science:

Pharmacology
Molecular Biology
Respiratory Medicine

Background:

The lung's large surface area and accessibility via inhalation make it an attractive site for topical drug delivery.
Oligonucleotide-based therapeutics, including antisense and small interfering RNA (siRNA), show potential for treating lung diseases.

Purpose of the Study:

To critically review the challenges and opportunities for using antisense and siRNA therapeutics targeting the lung.
To evaluate key factors influencing the efficacy and safety of inhaled oligonucleotide therapies.

Main Methods:

Literature review focusing on in vivo target validation for antisense and siRNA approaches.
Analysis of factors including delivery, distribution, stability, off-target effects, and immunogenicity.
Discussion on the selection of optimal messenger RNA (mRNA) targets for therapeutic intervention.

Main Results:

Inhalation offers a viable route for topical lung delivery of oligonucleotide therapeutics.
Significant challenges remain in achieving effective delivery, distribution, and stability within lung tissues.
Off-target effects and unwanted immune responses are critical safety considerations for these therapies.

Conclusions:

Despite challenges, the lung presents a significant therapeutic target for oligonucleotide-based drugs.
Further research is needed to optimize delivery systems and overcome biological barriers for successful clinical application.
Careful target selection and mitigation of adverse effects are crucial for developing safe and effective lung oligonucleotide therapies.