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Improving the delivery of treatments for collagen VI-related muscular dystrophy

Professor Zhou and team is developing a new way for treatments for collagen VI-related muscular dystrophy to reach muscle cells, helping to treat the condition and potentially reduce side effects.
Details
Principal Investigator
Professor Haiyan Zhou
Institute
University College London
Official title
Preclinical development of peptide-conjugated antisense oligonucleotide therapy for COL6-related congenital muscular dystrophy
Duration
30 Months
Total cost
£179,774
Conditions
Congenital muscular dystrophies (CMD)
Year
2024

Background

Collagen VI-related muscular dystrophy is caused by genetic changes. These changes cause the collagen VI protein to not work as it should, leading to damage to muscles. Professor Zhou and team have already identified potential treatments which can help correct the two most common genetic changes. However, they have found it difficult to deliver these potential treatments to the right place in the body, where collagen VI protein is made in muscle. If the treatment isn’t delivered to the right place, it won’t work properly, and it may build up in unwanted areas of the body where it could do damage.

The team have recently identified molecules called peptides that specifically bind to muscle cells (muscle interstitial fibroblasts) where collagen VI is made. These peptides can be thought of as targeting systems, which only lock onto these specific muscle cells. Linking these peptides to the potential treatments could help deliver the treatments to the right cells and ultimately help treat collagen VI-related muscular dystrophy.

Project aims

This project aims to develop peptide-linked treatments for collagen VI-related muscular dystrophy. First, Professor Zhou and team will investigate the best way to link the peptides to the potential treatments. They will then test a range of peptide-linked treatments, in models of the condition, to measure how well they target the right cells and make the collagen VI protein work correctly. The best peptide-linked treatments will then be taken forward for additional testing, to gather more evidence on how well they work and how safe they are.

Why this research is important

This research is an important step in developing an effective treatment for collagen VI-related muscular dystrophy. Identifying the most promising peptide-linked treatments will move the development process to the next stage. While it is very early in the development process, the data collected in this project will help move treatments towards testing in people in clinical trials.

This project is also focussing on finding an effective treatment that is delivered only where it is needed. This could improve how well the treatment works and reduce any potential side effects.

Acknowledgements

This project is funded in collaboration with LifeArc.

Models

Meaning: To find effective treatments, researchers need to understand more about a condition, as well as test treatments and gather evidence that they could work in people. Models can be used to mimic the condition. Examples of models are animals and cells grown in a dish.

 

We’ve already made great progress.

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