Thanks to a successful trial on the man's bestfriend.
Duchenne muscular dystrophy is a genetic disorder on which the production of dystrophin--needed for muscles to properly function--is disrupted. This causes the muscles to progressively degenerate and weaken.
This is a very serious disease for those who suffer have an average life expectancy of 26 years. It affects one in 5000 males and can begin as early as age 3 which vastly advances that it disables sufferers from walking by the age 12!
But people, take heart.
An international team of researches was able to restore muscle strength in dogs that suffers the same hereditary condition with their new gene therapy. This rapidly improved their ability to walk, run and even jump! Some were even undistinguishable from those healthy dogs.
They injected them with the gene microdystrophin--a smaller version of the dystrophin gene-- to replace the same faulty gene. And after just one injection, these dogs had a “significant restoration of muscle function with a stabilisation of clinical symptoms for more than two years after injection,” said the team.
In other words, the symptoms were absent even two years after the injection! Two years!
“This is tremendously exciting progress towards a gene therapy for Duchenne muscular dystrophy," Professor George Dickson, who led the trial at Royal Holloway, said. “The studies in dogs have been spectacular and exceeded our expectations.” And hopefully, this would soon be readily available for humans as well.
“Duchenne muscular dystrophy is a horrible, wasting, life-ending disease of young people," Prof Darren Griffin from the University of Kent, said. “By making use of the canine model and showing genuine improvement in the animals treated, then real hope is present for the prospect for disease treatment in humans. 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.”
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