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Improving muscle function by reducing scarring and restoring dystrophin in Duchenne muscular dystrophy

This grant by Professor Linda Popplewell aims to assess if molecular patches, also known as antisense oligonucleotides (ASOs), designed to reduce muscle scarring can improve the efficacy of therapies that promote dystrophin restoration.
Details
Principal Investigator
Professor Linda Popplewell
Institute
Royal Holloway, University of London
Official title
Proof-of-concept demonstration that combined dystrophin restoration and periostin manipulation synergistically improve skeletal muscle pathology
Duration
12 months
Total cost
£29,843

Background

Duchenne muscular dystrophy is caused by genetic changes in the dystrophin gene, which halt the production of dystrophin protein. In the absence of dystrophin, muscle cells break down and muscle tissue gets replaced by scar tissue, resulting in the weakening of the muscle. While therapies mainly aim to restore dystrophin in muscles, it is important to consider combining such therapies with treatments that reduce scaring of the tissue, often using molecular patches.

What are the aims of the project?

By using animal models of Duchenne, this project aims to:

  1. Understand whether using molecular patches to change a protein associated with muscle scaring in Duchenne would reduce muscle scaring in animal models of Duchenne.
  2. Understand if the combination of molecular patches and therapies that restore dystrophin production improves the efficacy of the dystrophin-restoring treatments.

Why is this research important?

This research can help improve future therapies, as well as provide evidence that a combination of treatments can prove an effective therapy for Duchenne. Notably, severe muscle scaring is often seen in adults living with Duchenne, and this research will be of particular importance to improve their symptoms and quality of life if scarring can be reduced.

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