Gene Therapy Treatment for Muscular Dystrophy Effective in Dogs

Duchenne muscular dystrophy (DMD) is a muscular disorder caused by a recessive mutation in dystrophin gene on the X chromosome that disrupts the production of the dystrophin protein. The disorder begins in the lower extremities and causes damaged muscle to be replaced with fibrous, fatty, or bony tissue. This leads to loss of muscle function and weakening of the muscles, while eventually causing a premature death. While being very prominent, as it affect 1 in 3500 males worldwide, scientists have searched for a way to successfully treat the dreadful disorder. 

The problem researchers have had with using gene therapy to repair the mutated dystrophin gene is that the gene is one of the largest in the human genome. However, after more then ten years of research, researchers at the University of Missouri (MU) School of Medicine have finally found a method of gene therapy that has successfully cured dogs with DMD and is now ready for human clinical trials.  Through previous research, the researchers at the MU School of Medicine have developed a miniature version of the dystrophin gene, called a microgene, which they can inject into an organism via a adeno-associated virus. In their testing, dogs were injected with the virus and the virus allowed for microgene to be delivered to all the muscles in the body of the diseased dog, effectively curing the dog of the disease. The dogs were injected with the virus when they were two to three months old and began showing signs of DMD, and are now six to seven months old and are developing normally. Since dogs inherit DMD naturally in a similar manner as humans this finding can be used to possibly cure the disease in human's. Based on how the dogs continue to develop the use of this gene therapy may be ready for human trial in a few years and can effectively lead to curing this disease. 

Overall, I find this article to be fascinating. It highlights the idea that genetics studies can be used for gene therapy and curing diseases. I think its amazing how we can combine so many aspects of genetics and biology and use it to cure diseases that have killed millions of people. 

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For more information on muscular dystrophy click here
For another article on the study directly from the MU website click here