Dogs given the treatment showed
Muscle wasting and breathing and heart problems follow, and patients rarely survive into their 40s.They injected the dogs with "microdystrophin" -- a compressed version of the dystrophin gene which is one of the largest in the human body with 2.The dogs&hybrid injection molding machine Factory39; symptoms remained in check two years after receiving a single injection, a team reported Tuesday in the journal Nature Communications.Boys affected by Duchenne muscular dystrophy develop muscle weakness in their first year of life, and generally become wheelchair-bound before the age of 15.The results, said the team, were "paving the way for human clinical trials.Successful treatment of a large mammal is a crucial step on the road to finding a treatment for human sufferers of DMD.. Duchenne muscular dystrophy is caused by genetic mutations that interfere with production of dystrophin, a protein # needed for healthy muscles.
"The disease has long been a target for gene therapy and it is only to be hoped that sufficient funds can be awarded for this research to reach its natural conclusion and go into full clinical trials," he said via the Science Media Centre.Given its size, it has been impossible to fit the distrophin gene into an emptied virus, which is used to deliver modified genes to patients.Geneticist Darren Griffin of the University of Kent commented that this was "really a very exciting study indeed". (Photo: Pixabay) Paris: Researchers have used gene editing to reverse symptoms in dogs of Duchenne muscular dystrophy (DMD), a muscle-wasting and life-shortening disease that affects one in 5,000 baby boys.
Dogs given the treatment showed "significant restoration of muscle function with a stabilisation of clinical symptoms for more than two years after injection," the researchers said in a statement.The international team developed a new type of gene transfer therapy to restore dystrophin expression in affected dogs."Duchenne muscular dystrophy is caused by genetic mutations that interfere with production of dystrophin, a protein needed for healthy muscles.3 million base pairs of coding "letters".Researchers have used revolutionary gene editing to resverse sumptoms of DMD in dogs and hope it paves way for human clinical trials
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