Gene editing will be more efficient owing to RNA-based drugs

CRISPR/Cas9, a way to use a naturally occurring bacterial system, has been found by researchers for several years to inactivate or correct specific genes in any organism. Though the CRISPR/Cas9 gene editing activity works continuously, it results in risk of additional editing at unwanted sites. Recently, scientists from University of California, San Diego School of Medicine, Ludwig Cancer Research and Isis Pharmaceuticals showed a way to use RNA to turn the CRISPR-Cas9 system on and off as wished: permanently editing a gene, but only temporarily activating CRISPR-Cas9. They published the new study in the National Academy of Sciences November 16. The study provides a platform for multiple therapeutic applications, especially for nervous system diseases, using successive application of designer CRISPR RNA drugs. They design a RNA which acts as a guide role to match the sequence of a specific target gene. The RNA guides the Cas9 enzyme to the desired site, where it cuts the DNA. The cell will repair the DNA break imprecisely, thus inactivating the gene. As an alternative, researchers can coax the cell into replacing the section adjacent to the cut with a healthier version of the gene. Now they are testing the CRISPR/Cas9 system in various applications to repair defective genes which cause disease. Above is the workflow of CRISPR/Cas9 system. This new way provides RNA-based, chemically modified drugs to transiently activate the CRISPR/Cas9 gene editing system. Though the activity is transient, the editing stops when the guiding RNA drug is cleared. An extension of the way can turn off the molecular scissors faster by addition of a second, chemically modified RNA drug that directs inactivation of the gene encoding the Cas9 enzyme. In conclusion, the RNA-based drugs shown in this study have so many advantages over the present CRISPR/Cas9 system in efficiency and selectivity. What's more, they can be synthesized in a short time on an industrial scale with strong commercial feasibility. Know more about CRISPR/Cas9 tools and other gene priducts here>>>http://www.cusabio.com/Recombinant-Protein/Recombinant-Homo-sapiens-Human-T-cell-surface-glycoprotein-CD8-alpha-chain-11090032.html