CRISPR -Cas9 to Emerge as Novel Therapeutic Treatment for Myotonic Dystrophy Disease

Release Date: 27-Nov-2020

One of the most common type of disease that is found in young children is Myotoinc dystrophy I. The symptoms that are usually observed in the patients suffering from the disease are progressive muscle wasting and weakness and a wide variety of other debilitating symptoms. According to the researchers involved in the respective clinical research study, the novel technology CRISPR -Cas9 is estimated to reverse the symptoms of the disease in the patients. In an older study, a group of researchers have proved that CRISPR-Cas9 could be effectively used in eliminating 95% of the aberrant RNA that is directly linked with the disease i.e. myotonic dystrophy type 1 and type 2.


Therefore, the current research study conducted by the researchers is focused towards advancing the use of CRISPR-Cas9 for reversing the complete symptoms of the disease in mouse model. The approach that the researchers will be using in the respective clinical research study will be gene therapy. The group of researchers used CRISPR-Cas9 in a non-infectious virus, which indeed delivered RNA-chewing enzyme inside cells. It was found that the CRISPR-Cas9 technique was able to reduced aberrant RNA repeats by more than 50%, further leading to the development of indistinguishable characteristics when compared with the healthy mice.


Further suppression of the complete immune system of the mice in the respective clinical trial research study led to the prevention of immune system reaction, clearance, longer CRISPR-Cas9 persistence as well as better response against the diseased cells. It also led to increase in the activity of genes involved in new muscle formation. The arrival of such clinical research studies in the pharmaceutical industry has led to the opening of several avenues for the disease treatment which has now become a growing problem in the entire world. Researchers inclined towards the respective study will accelerate the development of the therapy treatment by pushing the study into preclinical and clinical trials.

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