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New research funding brings new hope for Duchenne Muscular Dystrophy

Barts Charity and Action Duchenne have combined forces to support research into new stem cell therapies for Duchenne Muscular Dystrophy. With a combined award of £276,000 (£250,000 from Barts Charity and £26,000 from Action Duchenne) they will be supporting the research of Dr Yung-Yao Lin, lecturer at the Barts and The London School of Medicine and Dentistry, Queen Mary University of London.

9 May 2018

 

Dr Lin’s lab has developed novel gene-corrected muscle stem cells, generated from the patient’s own skin, that offer a potential therapeutic agent for Duchenne Muscular Dystrophy (DMD). This award will fund a feasibility study to produce the data needed to start clinical research. Muscle stem cells (myogenic progenitors) can regenerate muscle fibres and replenish the muscle. The project will explore whether transplanting DMD gene-corrected muscle stem cells generated from patients’ own skin cells, will reduce the risk of immunological rejection and the need for life-long immunosuppression, bringing new hope for people affected by this devastating disease.

Muscular dystrophies are debilitating genetic diseases characterised by progressive weakness and wasting of skeletal muscle, which is responsible for voluntary movements and breathing. Gene mutations lead to loss of muscle fibres and their replacement with fat and connective tissue. Current standards of care can delay loss of ambulation, cardiac and respiratory problems, but patients develop progressive weakness leading to immobility. Some muscular dystrophies cause premature death.

Dr Francesca Gliubich, Barts Charity’s Director of Grants said “We are excited at the prospects this research project offers to people affected worldwide.  Muscular dystrophies are genetic disorders with major impact both on life expectancy and quality. At present, there are few effective therapies, and patient-derived muscle stem cells have the potential to provide an effective treatment. This project will provide an essential milestone for progression to clinical trials.”

Diana Ribeiro, Chief Executive Officer and Director of Research, Action Duchenne said: “We are delighted to co-fund this project with the Barts Charity. This was an integral part of Action Duchenne’s updated research strategy released last year, supporting promising new techniques that will lead to further advances for Duchenne and Becker Muscular Dystrophy.”

Dr Yung-Yao Lin, Queen Mary University of London added: “We are grateful to receive the joint award from Action Duchenne and Barts Charity. This award will allow us to further advance the development of cell therapy for treating muscular dystrophies. Our study will pave the way for a first-in-human safety clinical trial. Our collaborative team is very excited to work with both charities on this innovative project.”   

 

 

 

 

 

 

 

 

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