Debunking The Duchenne Muscular Dystrophy: Can Exonics’ CRISPR Be The Answer?

By Cyril , Feb 28, 2017 10:46 PM EST

Long before, a significant number of experts have regarded the Duchenne muscular dystrophy (DMD) as a genetic disorder characterized by progressive muscle degeneration and weakness which happens to be one of the nine types of muscular dystrophy. Now, DMD patients might have just regained their hope after a foundation representing boys dying from muscular dystrophy has recently revealed that they will be trying to cure the disease using CRISPR, a breakthrough method of correcting DNA. CureDuchenne, a patient charity based in Newport Beach, California, says that it will allegedly spend $5 million to finance a new startup company, Exonics Therapeutics, based on their research wherein Exonics scientists were able to cure mice having muscular dystrophy by altering the DNA letters inside their cells.

The CRISPR Technology

According to reports revealed by Fierce Biotech, the new CRISPR/Cas9 technology, which has been put to human testing in China last year, is yet to be tested in the clinic the U.S this year. Despite not being tested in the US, authorities have claimed that it has already proven a major scientific draw for biotech companies including Editas, CRISPR Therapeutics and Intellia, Caribou Biosciences, as well as academic work coming out of the University of Pennsylvania. Additionally, experts have explained that a protein called Cas9 then attaches to the DNA and essentially cuts it, all of which either gets rid of, completely removes or replaces a gene with a better strand of DNA.

In one of his statements reported by MIT Technology Review, Jak Knowles, a doctor who serves as an acting CEO of the new startup company said that they have been looking for a home run, with nothing less than correction of the DNA error. Knowles continues to explain that the company hopes to move as quickly as possible toward a test of CRISPR in boys with Duchenne since CRISPR technology is a novel and potent way to precisely rewrite DNA. By being delivered into the muscles of these affected boys, authorities hope that it will enable the repair of the genetic error that causes the fatal condition, afflicting about one in 3,500 male births.

The Duchenne Muscular Dystrophy

Meanwhile, the Duchenne muscular dystrophy is said to be the most common severe form of childhood muscular dystrophy that hits young boys, with both their skeletal and heart muscles affected. As of the press time, it was found that the genetic disorder has already been affecting around 15,000 boys in the U.S. and around 300,000 globally. Ultimately, Knowles has also claimed that Exonics is focused on generating robust preclinical data that supports the development of a safe and efficacious therapy and advancing the company's Duchenne program thoughtfully and expeditiously to the clinic.


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