Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

Satellite Stem Cells and Muscular Dystrophy

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...

Targeted Cancer Therapies

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...

You might also read

Related Articles

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

Sort by

Same author

Topological rules and anomalies in intramolecular G-quadruplex folding: a comprehensive study.

Nucleic acids research·2026

Same author

The Epstein-Barr virus EBNA1 protein binds to G-quadruplexes in its own mRNA hence controlling its expression and immune evasion of the virus.

Nucleic acids research·2025

Same author

Conformational Change in a Four-Tetrad DNA G-Quadruplex upon Intercalation of a Small-Molecule Ligand PyDH2.

Angewandte Chemie (International ed. in English)·2025

Same author

iMab antibody binds single-stranded cytosine-rich sequences and unfolds DNA i-motifs.

Nucleic acids research·2024

Same author

Structural Optimization of Azacryptands for Targeting Three-Way DNA Junctions.

Angewandte Chemie (International ed. in English)·2024

Same author

Correction: Harnessing an emissive guanine surrogate to design small-molecule fluorescent chemosensors of <i>O</i><sup>6</sup>-methylguanine-DNA-methyltransferase (MGMT).

Organic & biomolecular chemistry·2024

Search research articles

Related Experiment Video

Updated: Mar 10, 2026

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

Targeting Expanded CUG and CTG Repeats as a Therapeutic Approach for Myotonic Dystrophy Type 1 (DM1).

Camille Richagneux^1,2, Anton Granzhan²

¹CMBC, CNRS UMR9187, INSERM U1196, Institut Curie, Université Paris Saclay, Orsay, France.

|March 9, 2026

Summary

Researchers are developing new drugs to treat myotonic dystrophy type 1 (DM1). These therapies target toxic RNA repeats by using small molecules and other compounds to block MBNL1 sequestration, offering hope for DM1 patients.

Keywords:

CTG repeats CUG repeats MBNL1 RNA degraders RNA ligands myotonic dystrophy type 1

More Related Videos

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

Related Experiment Videos

Last Updated: Mar 10, 2026

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

Area of Science:

Molecular Biology
Genetics
Drug Discovery

Background:

Myotonic dystrophy type 1 (DM1) stems from expanded CTG repeats in DNA.
These repeats transcribe into toxic RNA (CUG) that sequesters the MBNL1 protein, causing its dysfunction.

Purpose of the Study:

This review synthesizes 18 years of research on CUG and CTG ligands as potential DM1 therapeutics.
It focuses on compounds designed to inhibit MBNL1 sequestration.

Main Methods:

The review analyzes small molecules, oligomers, peptides, engineered proteins, and synthetic oligonucleotides.
It examines their chemical structures, design, binding modes (RNA/DNA), affinities, specificities, and mechanisms of action.

Main Results:

Various ligands have been identified or designed to interact with CUG repeats or CTG repeats.
These compounds demonstrate varying degrees of in vitro affinity, specificity, and biological activity in DM1 models.

Conclusions:

Inhibiting MBNL1 sequestration through targeted ligands is a promising therapeutic strategy for DM1.
Continued research into CUG/CTG ligands is crucial for developing effective DM1 drug candidates.