Treating Muscle Disease Arumainayagam

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Title: CRISPR Gene Editing Shows Promise for Treating a Fatal Muscle Disease

Author: Dina Fine Maron

Source: Scientific American

Publication Date: November, 2018


Scientists at the University of Texas Southwestern Medical Center have been able to treat Duchenne muscular dystrophy in dogs by editing their genes, which may lead to treatment for the same disease in humans. Duchenne muscular dystrophy causes the breakdown of muscles in early childhood and occurs mostly in males, affecting 1 in 3,500 boys. At the moment, there is no cure for the disease, and it usually causes an early death. The disease is caused by a genetic mutation that inhibits the production of the protein dystrophin, which is used in muscle cells to strengthen and protect muscles over time. In a recent study, the research group edited the muscle cells in dogs with Duchenne using CRISPR/Cas9 to remove a protein-coding segment of DNA that inhibits protein production, which is found in both dogs and humans. Their results showed that the mutations in the dogs increased their production of dystrophin, especially in cardiac muscle. They did not detect any unintended side-effects that sometimes occur because of gene-editing. Although it is still unknown whether this technique can be used to cure Duchenne in humans, the study showed promising results.


For someone suffering from Duchenne, knowing whether this disease can be cured by gene-editing is something they would want to know. Because the genetic mutations in this study were done on young dogs, if the same kind of modification could be done on children, there would be no need to genetically modify human embryos that have this disease. This would avoid the type of ethical backlash that accompanies human germline editing. Knowing that gene-editing may hold the key for curing Duchenne in children who already suffer from it would make an immense difference, and it’s likely that most people suffering from this disease would want to undergo this genetic modification if they could.