The FDA has approved a gene therapy for children with muscular dystrophy. This disease is caused by a mutation that rids the body of being able to make a protein it needs for healthy muscle function. Which can lead to potentially fatal health issues if the muscles that control the lungs, heart, etc decay. The purpose of this gene therapy is to package a protein with this function about one third the size of the original protein and deliver it via a harmless virus to muscle cells. Initial clinical trial results have shown that children have been able to produce the shortened protein with this therapy. However, further clinical trials are needed to confirm if this therapy is actually restoring muscle function. Nevertheless, this could potentially mean that a treatment for muscular dystrophy is on the horizon.
The implications of this gene therapy are incredibly profound. If researchers can replicate this therapy for other diseases, think of all of the other ailments that could potentially be cured. Hopefully the future results of these clinical trials prove that muscle function is being restored and muscular dystrophy will be a disease of the past.
https://www.sciencenews.org/article/first-gene-therapy-muscular-dystrophy-kids
https://www.mayoclinic.org/diseases-conditions/muscular-dystrophy/symptoms-causes/syc-20375388
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