Gene Editing in Muscular Dystrophy

There are over 6,000 different genetic diseases known, with very few cures. One of the more common types of genetic disorders is Muscular Dystrophy, which does not have a cure, but it does have certain treatments that can slow down the advancement of the disease. Muscular Dystrophy causes the muscles to gradually weaken and lose muscle mass; this loss of muscle mass affects the heart and body movements, which in some patients can lead to life in a wheelchair by the time they become teenagers. This degeneration of the muscles is caused by more than 30 genetic conditions on the DNA. The mutations that cause this disease only occur in a single gene (that code for dystrophin, which controls muscle cell structure) so doctors have been trying to find a cure, without success.

Researchers working for National Institutes of Health (NIH), are doing a study of the genes that affect Muscular Dystrophy in mice. Although mice are not humans, the disease runs the course parallel to the way it runs its course in humans. Recently, researchers discovered that about ⅘ of the genes that affect this particular protein expression are on a section of DNA that is not necessary for survival. This could mean during RNA processing (the step before translation where proteins are assembled), the nucleotides that code for the dystrophin could be removed, so the cell structure and growth in a patient with Muscular Dystrophy could return to normal. Since the mice have been treated with a gene that is programmed to remove the certain nucleotide sequences, their health has seemed to improve. Both the structure and function of the mice seem to be working much better after the treatment than before.

Because DNA Replication is so complicated, this research still has a long way to go, but they are making more and more advances every day. If the researchers can master this technique on mice, the next step would be to work with humans. Gene therapies are very controversial, so this could become controversial as well, but if they can find a way to remove certain RNA nucleotides that serve for harmful proteins, this breakthrough could change the lives of many victims. This article really stood out to me because we recently learned about DNA Replication and gene expression in this class, so I understood what the article was saying (mostly). DNA Replication was one of my favorite units because it amazes me what the human body can do in such small cells. Some parts of this article went a little over my head, but I was able to use the information from class to piece most of the information together. Also reading about up and coming medicine / cures always interests me because these repetitive trials can lead to something extraordinary.

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Biology Teacher

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