The researchers in the Duke University announced that with the use of high-end CRISPR/Cas9 technology, they have successfully edited the gene, which resulted in the reduction in the levels of blood cholesterol in mice.
The study published in an online journal, Nature Communications, has asserted that the researchers have succeeded to silence the gene, which will lead to the reduction of blood cholesterol. This repression will last for 6 months after the one-time treatment. CRISPR/Cas9 method is built on an antiviral resistance system in bacteria in which the Cas9 enzyme distinguishes the viral DNA orders of earlier diseases, and trims up the attacking DNA during reinfection. The CRISPR/Cas9 system not only locates and cuts the particular order of DNA but also turns on and off the expression of targeted genes, and does not change the DNA-coding order permanently.
Charles Gersbach, Associate professor at Duke University, asserted that they previously used the similar types of tools which turned the genes on or off in the cells which are cultured, and they wanted to check if they could also deliver the same to the animal category that is related with the gene therapy. The test was successful and the researchers also have observed liver enzymes in the blood.
Pratiksha Thakore, the Ph.D. student, confirmed the reduction in the levels of the target gene, Pcsk9, after the injection. Also, she added that there was an increase in the expansion of immune cells indicating that the immune cells were entering the liver after the delivery of Cas9 in mice. Further, she asserted that obtaining better insights of the immune response and the modulation of the same will be an important part while using the Cas9 techniques for therapies.
Recently, a CNIO study has explained a powerful and versatile mouse pattern helpful for improving the cancer research. This study will accelerate the premedical testing of the targeted therapies.
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