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Understanding how Duchene muscular dystrophy affects brain function 

Professor Patrizia Ferretti and her team will look at brain cells from people with Duchene muscular dystrophy (DMD) to improve our understanding of how brain function is affected in people with this condition.
Details
Principal Investigator
Professor Patrizia Ferretti
Institute
UCL Hospitals NHS Foundation Trust and University College London
Official title
Addressing Astrocyte Dysfunction in Duchene Muscular Dystrophy
Duration
24 months
Total cost
£149,843
Conditions
Duchenne muscular dystrophy
Year
Duchene muscular dystrophy

Background:

DMD is a progressive muscle wasting condition caused by genetic changes in the dystrophin gene, which prevent the production of the dystrophin protein. As well as being important in muscles, the dystrophin protein also plays a role in the brain. People with DMD are known to be more likely to have conditions like autism, ADHD, learning difficulties, anxiety, depression and OCD. However, how DMD affects brain function is not well understood and current knowledge and treatments for DMD do not address this.  

What are the aims of the project?

A type of brain cells, called astrocytes, have been shown to not work properly in some people with DMD. The aim of this project is to look at how changes in the dystrophin gene affect the function of these cells. The researchers will also look at how these changes in astrocytes affect the function of another type of brain cell called neurons.  

Why is this research important?

This research will increase our understanding of the causes of changes in how the brain works in some people with DMD. Findings from it could potentially lead to future treatment developments to improve psychological symptoms in people living with DMD 

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