Muscular Dystrophy and Gene Therapy

Muscular Dystrophy and Gene Therapy

I found this study that the University of Missouri-Columbia performed regarding MD and gene therapy interesting and can have a huge impact on how physical therapy can be used more.  The link to the study can be found below:

https://www.sciencedaily.com/releases/2015/10/151022141722.htm





This study was successfully performed last year on dogs after having researched the method for 10 years. After successful animal trials, it is now possible that human trials could begin in the near future.  In simplest terms, the study tries to address the gene mutation aspect of MD, which causes the disruption of the production of the protein known as dystrophin.  This could then allow muscles to develop as they would in a health human being.



In order to do this type of treatment, scientists had to come up with a miniature version of the gene which is known as a "microgene."  This gene is then sent to all of the affected muscle of the test subjects, which were mice to begin with before progressing to dogs.

This is huge progress with the fight against MD, because the diseased dogs were shown to have normal muscle development just a few months after the gene was administered.  This would allow PTs and PTAs to deliver more therapy than what used to be possible due to lack of muscle mass.  If humans can have a similar response that the dogs have had, this could be life-changing for many people affected by MD. Patients could benefit form the overload and specificity principles of exercise and actually experience varying degrees of hypertrophy.