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Related Experiment Video

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Modeling Myotonic Dystrophy 1 in C2C12 Myoblast Cells
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Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1.

JuYeon Lee1, Yugang Bai2,3,4, Ullas V Chembazhi5

  • 1Department of Chemistry, University of Illinois at Urbana-Champaign, Urbana, IL 61801.

Proceedings of the National Academy of Sciences of the United States of America
|April 13, 2019
PubMed
Summary

Researchers developed cell-penetrating multivalent ligands to treat myotonic dystrophy type 1 (DM1). These ligands target repeat expansions in DNA and RNA, showing efficacy in cellular and animal models with no observed toxicity.

Keywords:
DNA/RNA-targeting therapeuticscell-penetrating peptide mimicmultivalent ligandmyotonic dystrophy type 1trinucleotide repeat expansion diseases

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Drug Discovery

Background:

  • Developing multivalent ligands for therapeutic use faces challenges including poor cell permeability, delivery issues, and toxicity.
  • Myotonic dystrophy type 1 (DM1) is a repeat expansion disease affecting DNA and RNA, leading to disease progression.

Purpose of the Study:

  • To design and develop intrinsically cell-penetrating multivalent ligands targeting trinucleotide repeat expansions in DM1.
  • To evaluate the efficacy and safety of these novel ligands in cellular and animal models of DM1.

Main Methods:

  • Oligomeric ligands were designed mimicking cell-penetrating peptides, featuring alternating recognition moieties and bisamidinium groove binders.
  • Biological studies included assessing cell permeability, toxicity in cell cultures and mice, and therapeutic effects in DM1 cellular and animal models.
  • Phenotypic recovery was assessed in a DM1 Drosophila model.

Main Results:

  • The designed oligomers demonstrated intrinsic cell permeability and no apparent toxicity at effective concentrations in both cellular and animal models.
  • Significant reduction or elimination of key DM1 features was observed in DM1 cells and a DM1 liver mouse model.
  • Restoration of normal climbing behavior was noted in adult DM1 Drosophila.

Conclusions:

  • The developed multivalent ligands effectively target DM1 repeat expansions, offering a promising therapeutic strategy.
  • The amphiphilic, polycationic design strategy is applicable for treating DM1 and potentially other repeat expansion diseases.
  • This approach advances DNA/RNA-targeted therapeutics by overcoming common delivery and toxicity hurdles.