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Research

Gene Therapy Success in IBM

Scientists at five U.S. institutions have successfully used gene therapy to improve muscle function in a single human subject with a hereditary form of  inclusion-body myositis (IBM)  caused by mutations of the GNE gene. Study results were published online March 30, in the Journal of Gene Medicine.

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SMN Gene Transfer Benefits Mice

A research group from the University of Sheffield in the United Kingdom has found that mice with a disease mimicking human  spinal muscular atrophy (SMA)  benefited significantly from intravenous transfer of the gene for the SMN (survival of motor neurons) protein. The mice lived significantly longer than untreated mice of the same type.

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Tadalafil Trial in Becker MD

Update (Oct. 8, 2012):  This story has been updated to reflect that the tadalafil trial in BMD has been slightly revised and is now open. In June 2012, it had temporarily closed to new participants. The investigators say the revised trial design was necessary because they were unable to obtain access to the MRI machine for the time periods that were necessary in the first design.

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DMD Research: Potential Heart Saver

A synthetic compound that seals cellular membranes has been found to stop the progression of heart-muscle destruction in dogs with a disease closely resembling human  Duchenne muscular dystrophy (DMD), MDA-supported researchers have found.MDA research grantee DeWayne Townsend at the University of Minnesota-Minneapolis and colleagues found poloxamer 188 (p188), infused into a vein in golden retrievers with a DMD-like disease, sealed fragile cardiac muscle-cell membranes and prevented the loss of cardiac muscle function (cardiomyopathy) that occurred in an untreated control group of comparable dogs.

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Building New Muscle Requires Controlled Damage

Remodeling a building usually requires some degree of dismantling before new construction can begin. That principle, it now seems, also may apply to the remodeling of the body's cells. Scientists at Ottawa Hospital Research Institute and the University of Ottawa have found recently that it applies to muscle cells, which can only develop from a stemlike state into mature muscle fibers after a certain amount of their DNA has been disassembled and then rebuilt. 

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New Muscle Stem Cell Found in Mice

MDA-supported scientists in France have identified a previously unknown type of muscle stem cell located in the spaces between muscle fibers in mice. They say the new cells, dubbed "PICs," may play at least as important a role in muscle regeneration and repair as satellite cells, which have been recognized as stemlike cells in muscle since the 1960s. As such, they could have implications for treatment of  muscular dystrophies.

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ALS Research: Poison Dirt?

New findings suggest a possible link between dust-dwelling bacterial toxins and an elevated incidence of  ALS (amyotrophic lateral sclerosis)  in Gulf War veterans.The study blames cyanobacteria, microorganisms that live in desert sands and which can be inhaled when they’re kicked up in dust, such as when a convoy of military vehicles rumbles by. Cyanobacteria are common throughout the world in salt water, fresh water and soil.

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Follistatin Genes Strengthen Muscles in Monkeys

Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength with no adverse effects, say researchers at Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University.

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Research Updates Summer 2009

Story includes research about: Becker muscular dystrophy, Duchenne muscular dystrophy and oculopharyngeal muscular dystrophy. Scientists at Children’s National Medical Center in Washington, Carolinas Medical Center in Charlotte, N.C., and the National Center of Neurology and Psychiatry in Tokyo, have successfully treated dogs with a disease closely resembling  Duchenne muscular dystrophy (DMD), using a molecular treatment strategy called exon skipping.

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ALS: A Vicious Cycle

A vicious cycle in which damage to nerve cells (neurons) in the spinal cord results in the loss of an important mechanism to protect neurons, causing more neuron loss, has been identified as a possible contributor to  ALS (amyotrophic lateral sclerosis). Identification of this pathway opens the door to targeting it with therapeutic agents.

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