Chicago, IL (PRWEB) February 27, 2015 Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA's 2015 Scientific Conference, to be held March 11-14 in Washington, D.C. Background: Gersbach and team are investigating a new genetic therapeutic technique that has the potential to treat more than half of patients fighting DMD and could be developed as a permanent, one-time treatment. Known as CRISPR-Cas9 genome editing, the strategy targets a large area of the dystrophin gene (DNA) and is designed to cause production of shorter-than-normal, but still functional, dystrophin protein in muscle tissue. If successful in humans, it could prolong function and increase longevity. Other promising experimental DMD treatments such as the experimental drugs eteplirsen and drisapersen are currently being tested in clinical trials, but because these other treatments target dystrophin RNA rather than DNA, they would not result in a permanent correction and would require repeated treatment over a patients lifetime. Ever since MDA-supported researchers identified flaws in the … Continue reading →

A LEADING North-East scientist has won a prestigious award for her pioneering work into neuromuscular diseases. Professor Kate Bushby, from Newcastle University, has received the 2015 EURORDIS Scientific Award, which recognises her outstanding research into rare inherited neuromuscular genetics. Nominations for this award were received from the public, giving an opportunity for everybody in the rare disease community and beyond to have their say on who should get the top accolade. Professor Bushby, who is an expert in neuromuscular genetics at Newcastle University, is director of the John Walton Muscular Dystrophy Research Centre, based at Newcastles International Centre for Life. She is also honorary consultant geneticist at Newcastle Hospitals NHS Foundation Trust. The research centre is at the forefront of developments in neuromuscular diseases as its team work with experts and patient organisations from around the globe to help improve diagnosis, care and treatment for patients. Professor Bushby said: I am delighted to have received this award: a real sign of recognition that the John Walton Muscular Dystrophy Research Centre is a world leader in the field of rare diseases. Genetic neuromuscular diseases are disorders that affect nerves that control the bodys voluntary movements, and a number of conditions are … Continue reading →