Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

Site-Targeted Drug Delivery Systems: Polymeric Carriers

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...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Systemic Inflammation Modulates Clearance and Drives Extra-Hepatic Distribution of Extracellular Vesicles.

Journal of extracellular vesicles·2026
Same author

Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression.

Journal of cachexia, sarcopenia and muscle·2026
Same author

Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.

Molecular therapy. Nucleic acids·2026
Same author

Biophysical and biological properties of splice-switching oligonucleotides and click conjugates containing LNA-phosphothiotriester linkages.

Nucleic acids research·2025
Same author

Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides.

Angewandte Chemie (International ed. in English)·2025
Same author

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.

Gene therapy·2025

Related Experiment Video

Updated: May 23, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
09:04

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

Published on: September 21, 2017

Optimizing tissue-specific antisense oligonucleotide-peptide conjugates.

Corinne A Betts1, Suzan M Hammond, Hai-Fang Yin

  • 1Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.

Methods in Molecular Biology (Clifton, N.J.)
|March 29, 2012
PubMed
Summary
This summary is machine-generated.

Next-generation antisense oligonucleotide (AON) therapies utilize cell-targeting peptides for improved delivery. These peptide-conjugated AONs show promise for treating diseases like Duchenne muscular dystrophy (DMD) by enhancing exon skipping and protein expression.

More Related Videos

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?
14:20

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?

Published on: June 13, 2014

Related Experiment Videos

Last Updated: May 23, 2026

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
09:04

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

Published on: September 21, 2017

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?
14:20

Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?

Published on: June 13, 2014

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Antisense oligonucleotides (AONs) are a promising therapeutic modality for mRNA-misregulated diseases.
  • Cell-targeting peptides enhance the delivery and efficacy of AONs.
  • Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene.

Purpose of the Study:

  • To describe methods for analyzing cell-targeting peptide conjugated AONs.
  • To evaluate the efficacy of these conjugates in vitro and in vivo for Duchenne muscular dystrophy (DMD) treatment.
  • To provide a framework for fast-tracking candidate peptides for clinical trials.

Main Methods:

  • Analysis of peptide-conjugated AONs in primary cell cultures.
  • Assessment of local and systemic delivery in mouse models of DMD.
  • Quantification of exon skipping and dystrophin protein expression.
  • In vitro screening followed by in vivo administration of candidate peptides.

Main Results:

  • Chimeric and novel cell-penetrating peptides demonstrate high levels of exon skipping and dystrophin protein expression.
  • Effective body-wide expression achieved at very low AON doses.
  • Physiological and functional correction of dystrophin protein confirmed.

Conclusions:

  • Cell-targeting peptide-conjugated AONs represent a next-generation therapy for Duchenne muscular dystrophy (DMD).
  • Systematic analysis and screening methods facilitate the development of effective AON conjugates.
  • These approaches enable the rapid advancement of promising peptide-AON candidates towards clinical application.