Scientists Getting Closer to Developing Effective Therapy for Muscle Degeneration
Duchenne muscular dystrophy (DMD) is a rare muscle disorder but is one of the most common genetic problems that primarily occur in men. For years, scientists have been working hard to find the best way to inhibit muscle degeneration and reverse the effects of the disease. But things have been very difficult, until today.
In a paper published yesterday in the journal Molecular Therapy, a team of researchers from University of Missouri showed how duchenne muscular dystrophy can be treated using gene therapy.
Muscular dystrophy takes place when damaged muscle tissues are replaced with fibrous, bony or fatty tissues. As the disease progresses, the muscles begin to lose their function and become permanently damaged. Previous studies reveal that boys who have DMD exhibit a gene mutation that hinders the production of a protein called dystrophin – a substance necessary for the smooth functioning and survival of muscles. The reduction in dystrophin levels is what causes the degeneration of muscle fibres.
Numerous studies and lab experiments have been carried out to know how dystrophin can be retained or increased. But being the largest gene in the human body, it is very difficult to deliver to the targeted area in the body. According to the researchers, there are approximately 4,000 amino acids in dystrophin. And in order for it to fit in a vehicle that will carry the gene to the affected area, one has to remove 70 percent of the gene.
In previous studies, scientists suggest that the presence of the micro-dystrophin in the affected muscles can effectively reverse the effects of the disease. In most rat experiments though, subjects demonstrated very minimal DMD symptoms (a condition that could not help predict what will happen in humans). Loss of dystrophin resulted to severe muscular dystrophy in canine models but previous trials that involved them have also failed.
Overcoming the Challenges
For the current study, MU researchers developed a new micro-dystrophin gene that carries an important functional region which according to them are absent in previous studies.
The team placed the new micro gene in a virus which was then injected to the dogs with severe muscular dystrophy. To their surprise, the new gene did not only reduce inflammation and fibrosis but has also improved the muscle strength.
Their findings open up a big door towards an entirely new gene therapy that could prevent muscle degeneration in human. Although there’s still a lot of work to be done, the researchers are very positive that their endeavour could lead to great outcomes.
The researchers are set to conduct clinical trials on humans soon.
Source of this article:
A quantum leap in gene therapy of Duchenne muscular dystrophy
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