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Identification of Charcot-Marie-Tooth disease therapies through drug repurposing

Dr Barbaric and her team will use stem cells that carry a genetic change causing Charcot-Marie-Tooth-Disease Type 2A to identify potential treatments.
Principal Investigator
Dr Ivana Barbaric
University of Sheffield
Official title
A human stem cell-based model of axonal Charcot Marie Tooth disease Type 2A for identification of therapeutics
24 months
Total cost


Charcot-Marie-Tooth disease (CMT) is a group of progressive, genetic, muscle-wasting conditions that affect peripheral nerves – the nerves that connect the brain and spinal cord with the rest of the body. There is no treatment for CMT, and there are no animal models that faithfully represent the condition observed in humans. Dr Barbaric and her colleagues have managed to change human stem cells to represent the CMT condition, and these stem cells have grown into nerve cells that behave like the nerve cells of people with CMT.

What are the aims of the project?

The main objective of this research is to identify new treatments for CMT. The researchers will use the CMT nerve cells, which physically differ from healthy nerve cells, to test drugs that are already used for other conditions (also called drug repurposing). The hope of this is to find drugs that can also treat CMT symptoms, while at the same time being safe to use.

Why is this research important?

There is no treatment for CMT, and this research could help to identify currently used drugs as potential therapeutic approaches to managing CMT. This could greatly improve the quality of life of those with CMT and bring us one step closer to finding a cure.

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