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 Experiment Video

Updated: May 31, 2026

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia
10:41

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia

Published on: September 12, 2020

Myotonic dystrophy mouse models: towards rational therapy development.

Mário Gomes-Pereira1, Thomas A Cooper, Geneviève Gourdon

  • 1Inserm U781, Université Paris Descartes, Faculté de Medicine Necker Enfants Malades, Paris, France. mario.pereira@inserm.fr

Trends in Molecular Medicine
|July 5, 2011
PubMed
Summary
This summary is machine-generated.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Developing endpoints for the cardiac burden in myotonic dystrophy type 1: A workshop report.

Journal of neuromuscular diseases·2026
Same author

Progressive cardiac phenotypes and reduced reversibility from long-term CUGexp RNA expression in a DM1 mouse model.

JCI insight·2026
Same author

Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1.

American journal of human genetics·2026
Same author

Large-scale proteomics profiling of peripheral blood of DM1 patients identifies biomarkers for disease severity and functional capacity.

Journal of neuromuscular diseases·2026
Same author

Translational behavioral phenotypes in DMSXL mice for CNS manifestations of DM1.

Journal of neuromuscular diseases·2026
Same author

Pre- and postsynaptic upregulation of FasII synergistically underlies neuropathological and behavioral phenotypes in a Drosophila model of myotonic dystrophy.

Nature communications·2025
Same journal

Hyocholic acids: Third bile acids for neonatal health.

Trends in molecular medicine·2026
Same journal

Clonal hematopoiesis in Alzheimer's brain: Protective, pathogenic, and context-dependent?

Trends in molecular medicine·2026
Same journal

Targeting amino acid metabolism in hepatocellular carcinoma.

Trends in molecular medicine·2026
Same journal

Turning perfusion into repair through ferroptosis blockade.

Trends in molecular medicine·2026
Same journal

CaMKK2: A tumor stress-integration node.

Trends in molecular medicine·2026
Same journal

Precision gene editing: From proof-of-concept to curative therapies.

Trends in molecular medicine·2026
See all related articles

Mouse models of myotonic dystrophy reveal toxic RNA mechanisms and offer insights into therapeutic strategies. This review assesses their utility for preclinical drug and gene therapy testing.

Area of Science:

  • Molecular Biology
  • Genetics
  • Neurology

Background:

  • DNA repeat expansions cause toxic RNA production, a mechanism well-studied in myotonic dystrophy.
  • Over 20 mouse models have elucidated various aspects of this disease mechanism.

Purpose of the Study:

  • To provide an in-depth assessment of molecular and phenotypic features of myotonic dystrophy mouse models.
  • To critically evaluate the suitability and limitations of these models for preclinical therapeutic strategy testing.

Main Methods:

  • Review of existing literature on myotonic dystrophy mouse models.
  • Comparative analysis of molecular and phenotypic data.
  • Assessment of preclinical therapeutic strategy testing capabilities.

Main Results:

More Related Videos

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
09:39

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases
06:52

Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases

Published on: September 29, 2014

Related Experiment Videos

Last Updated: May 31, 2026

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia
10:41

Implantation of Osmotic Pumps and Induction of Stress to Establish a Symptomatic, Pharmacological Mouse Model for DYT/PARK-ATP1A3 Dystonia

Published on: September 12, 2020

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
09:39

Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells

Published on: July 29, 2016

Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases
06:52

Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases

Published on: September 29, 2014

  • Mouse models offer significant insights into toxic RNA-mediated disease mechanisms.
  • These models are valuable resources for testing pharmacological, anti-sense, and gene therapy approaches.
  • Critical analysis reveals both strengths and weaknesses of different transgenic lines for preclinical studies.

Conclusions:

  • Myotonic dystrophy mouse models are crucial for understanding disease pathogenesis.
  • Careful selection of appropriate models is essential for effective preclinical testing of novel therapies.
  • Further research using these models will accelerate the development of treatments for myotonic dystrophy.