DMD affects about 1 in 5000 boys in the United States and results from a gene mutation that affects the production of dystrophin. The lack of dystrophin leads to muscle weakness and ultimately death early on in life.
The team developed a method to send micro-dystrophin to the affected muscles using a common virus that produces no symptoms in humans. This treatment was injected in the dogs when they began to show symptoms at about two to three months of age. As of now, the dogs are six to seven months old and are continuing to develop normally.
http://www.futurity.org/duchenne-muscular-dystrophy-dogs-1032682-2/
http://www.ncbi.nlm.nih.gov/pubmed/26264580
This could be a very promising form of treatment for the future. I would be very interested in seeing if DMD affects dogs in a more human-like manner compared to other animals like mice. I also wonder if any future complications will arise with the micro-dystrophin depleting over time and has to be administered at a later date and how often, so this would be an interesting project to revisit when the dogs are older!
ReplyDeleteThis is very interesting and hopefully can translate into humans! I am curious whether timing of the micro-dystrophin injected since DMD isn’t always recognized as early as 2-3 months. I am also interested to see how this will function in humans, maybe a baboon study of these injections are next, with varying concentrations and see how it affects muscle function over time, but definitely a promising treatment option.
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