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Current Projects

We fund pioneering research for better treatments to improve people’s lives today, and to transform those of future generations.

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Understanding the biology underlying a form of congenital muscular dystrophy
This PhD studentship, to be supervised by Dr Laura Swan at the University of Liverpool, will investigate the structure and function of INPP5K, a protein that is important in congenital muscular dystrophy.
Read Understanding the biology underlying a form of congenital muscular dystrophy
Characterising pre-clinical models and testing gene therapy for X-linked spinal muscular atrophy
Professor Thomas Gillingwater and colleagues previously developed gene therapy to treat X-linked spinal muscular atrophy (XL-SMA); however, they were unable to test this due to a lack of animal models. In this project, the researchers will characterise a newly generated mouse model, which is much needed to test new treatments for XL-SMA.
Read Characterising pre-clinical models and testing gene therapy for X-linked spinal muscular atrophy
Improving accessibility of bone density scanning for wheelchair users living with muscle wasting conditions
Dr Jarod Wong will lead a study involving people living with muscle wasting conditions and healthcare workers to improve the accessibility and performance of bone density scanning to make monitoring weak bones more straightforward.
Read Improving accessibility of bone density scanning for wheelchair users living with muscle wasting conditions
Understanding the link between the shape of nuclei and DNA organisation in muscle cells from people living with laminopathy
Professor Francesco Saverio Tedesco and his PhD student at University College London aim to understand if changes in the shape of nuclei in muscle cells of people with laminopathies cause the loss of organisation of DNA.
Read Understanding the link between the shape of nuclei and DNA organisation in muscle cells from people living with laminopathy
Understanding the molecular processes that cause the progression of FSHD
Professor Giorgio Tasca and colleagues from Newcastle University will study muscle cells from people living with Facioscapulohumeral muscular dystrophy (FSHD) to see whether specific genes are switched on or off and where in the cells this happens.
Read Understanding the molecular processes that cause the progression of FSHD
Developing a mouse model and treatment for SORD neuropathy
Dr James Sleigh and colleagues from University College London will use a mouse model of SORD neuropathy to test potential gene therapies for this condition.
Read Developing a mouse model and treatment for SORD neuropathy
Developing a gene editing technique as a potential treatment for Duchenne muscular dystrophy
Professor Linda Popplewell and her PhD student at Teesside University will develop a new type of gene editing therapy for people living with Duchenne muscular dystrophy (DMD). This potential therapy could be suitable for many people with DMD unlike other potential treatments that target specific regions of the DMD gene.
Read Developing a gene editing technique as a potential treatment for Duchenne muscular dystrophy
Understanding genetic variations in different tissues of people with DM1
Dr Ami Ketley from the University of Nottingham will use a new technique and computer analysis to look at different tissues in mice that have the same genetic information as people with myotonic dystrophy type 1 (DM1) to understand what is happening in the heart, brain and muscles.
Read Understanding genetic variations in different tissues of people with DM1
Developing a gene therapy for desminopathy
Dr Michael Keogh from Newcastle University will be developing a new gene therapy for a type of myofibrillar myopathy called desminopathy. He’ll be looking to see if it’s harmful for muscle cells in laboratory settings.
Read Developing a gene therapy for desminopathy
Developing a new gene therapy for periodic paralysis
Dr Roope Mannikko and his PhD student at University College London (UCL) aim to develop a new gene therapy to help prevent muscle weakening in people with periodic paralysis.
Read Developing a new gene therapy for periodic paralysis
Using state-of-the-art genetic sequencing techniques to find genes that cause limb-girdle muscular dystrophies
Professor Henry Houlden from University College London (UCL), and colleagues from UCL and Newcastle University, will use state-of-the-art DNA sequencing techniques to identify genetic causes of limb-girdle muscular dystrophies in families who have no genetic diagnosis.
Read Using state-of-the-art genetic sequencing techniques to find genes that cause limb-girdle muscular dystrophies
Dietary supplements as a potential new treatment for mitochondrial myopathies
Professor Rita Horvath and her team from the University of Cambridge will test the use of a dietary supplement as a potential new treatment for people with mitochondrial myopathies.
Read Dietary supplements as a potential new treatment for mitochondrial myopathies

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