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The Johns Hopkins scientist who first showed that the absence of the protein myostatin leads to oversized muscles in mice and men has now found a second protein, follistatin, whose overproduction in mice lacking myostatin doubles the muscle-building effect.
The Recombinant DNA Advisory Committee (RAC), part of the U.S. National Institutes of Health (NIH) in Bethesda, Md., today voted that the first American study of gene therapy in boys with Duchenne muscular dystrophy (DMD) can move forward, the Muscular Dystrophy Association (MDA) announced.
The study reviewed by the RAC will test the safety of a laboratory-engineered gene for the muscle protein dystrophin tucked inside a modified adeno-associated virus. Dystrophin is a protein needed but missing in boys and young men with DMD.
In the MDA-funded trial, six boys with DMD, a devastating childhood muscle disease that affects some 30,000 boys in the United States and results in death from respiratory or heart failure in the 20s, will undergo injections of the gene into their biceps muscles. One biceps will receive gene injections, while the other receives sham injections.
After about six weeks, samples of muscle cells from each biceps will be examined for evidence of dystrophin production and signs of any damaging reactions, such as an unwanted immune response.
The study participants, who will receive injections and monitoring at Columbus Children’s Research Institute, part of Ohio State University, will undergo many other types of safety testing during and after the gene transfer.
The first gene therapy human trial in the United States for a form of muscular dystrophy is under way.
The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina at Chapel Hill's School of Medicine and the University of Pittsburgh.
The trial was launched March 28, at Columbus Children's Hospital in Ohio, an affiliate of Ohio State University's School of Medicine. In the trial, six boys with DMD will receive replacement genes for an essential muscle protein.
Each of the boys will receive replacement genes via injection into a bicep of one arm and a placebo in the other arm. Neither the investigators nor the participants will know which muscle got the genes. After several weeks, an analysis of the injected muscle tissue's microscopic appearance, as well as extensive testing of the health and strength of the trial participants, will reveal whether gene therapy for DMD is likely to be safe and whether it's likely to result in persistent production of the essential protein in muscle cells.
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