(Mirror Daily, United States) – The people behind the technology that received the journal Science’s award for “Breakthrough of the Year” are busy once again proving its enormous potential, this time by having CRISPR treating Duchenne Muscular Dystrophy in mice.
For the time in history, CRISPR was used to successfully and fully cure a genetic disease affecting a fully grown living mammal, all the while using a technology and strategy that can be easily applied to a human suffering from the same illness.
A team of researchers led by associate professor of biomedical engineering Charles Gersbach, from the Duke University, ran the test, and the results were published over the course of three studies, all published in Thursday’s edition of the journal Science.
The studies focus on the team of researchers attempting and succeeding in curing an adult mouse suffering from the infamous Duchenne muscular dystrophy, efforts rewarded due to their use of the CRISPR technology – clustered regularly interspaced short palindromic repeats.
Duchenne muscular dystrophy is a disease that affects roughly 5000 male babies every year, and it manifests by causing an increasingly debilitating weakness in the patient via genetic mutations that interfere with the proper development of muscle tissue.
This is achieved by interfering with the production of dystrophin, the protein responsible with the creation of healthy muscle tissue.
The team of researchers from the Duke University managed to cure the mouse of the disorder by making use of CRISPR-Cas9’s groundbreaking technology.
Hailed for its enormous potential for both good and evil, the CRISPR technology can fix genetic mutations in subjects by creating very subtle changes in the DNA of the patient, by employing the use of non-pathogenic viruses to deliver the alterations.
By working and refining the CRISPR-Cas9 technique so that it becomes applicable in cases such as Duchenne muscular dystrophy, Charles Gersbach modified the procedure so that it a modified bacterial defense system targets and cuts through the DNA sequences of a number of known viruses.
By applying the modified technique directly to the leg muscle of the affected animal, they were able to observe a series of corrections taking place throughout the entire mouse’s body.
Not only was its affected leg healed, but so were a number of the mouse’s other muscles, including its heart.
Even though there is still a lot of work to do in order to ensure the safety and applicability of the treatment for human patients, the team of researchers is quite excited about the results, hardly being able to wait for the next round of tests to begin.
Image source: Wikimedia
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