Gene Editing to help manage Muscle disorder

Recently, Gene editing has seen growth with the use of the well-renowned Crspr-cas9 therapy. It includes using genes to replace damaged cells in DNA, helping cure the diseases. After a lot of luck and hope, researchers are trying to use it on a more intense disease, which affects boys at birth. Duchenne Muscular Dystrophy is deadly, can let boys loose muscle really fast and putting them in a wheelchair by the age of 10, it can result in heart failure and death.

Although it has not yet been tested on humans, three researchers reported to the Science journal that they used the therapy on mice. They used a virus that helps infect the mice while attaching with the DNA to cure the dystrophin gene. The defective stretch of DNA that affects the gene is known as anexon. With exon removed or not present, mice were able to regain muscle strength while shortening dystrophin protein helped mice retain health faster. The team comprised of Amy J. Wagers from Harvard University, Eric N. Olson from Univerity of Texas and Charles A. Gersbach from Duke University.

The affected genes mostly comprise of 79 exons or sections, but can also function if there are fewer than these exons present. Unless the two ends are intact, the protein will keep on functioning. Dr. Oslon thinks in order to help this work on humans, more clinical trials are needed and may take a few years before the issues related to permissions in regards to gene editing is provided. It needs to be safe, right and be standardized for humans in order to avert any damages. Although patients may receive e a single dose of treatment, Dr. Gersbach thinks it may take several before the protein is eliminated and the DNA becomes resistant. As current chemical drugs are not helping cure this disease, gene editing is seen as the future to ensure the safety of affected patients.


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