DNA fragments: These insights are allowing us to improve the system and make the process more effective, paving the way for new strategies to treat RNA-based diseases. Novick, Timothy Strutzenberg and Patrick R. Photo courtesy Getty Images.
Their first work was around dementia treatment, and the team showed that the tool could be used to correct protein imbalances in cells of dementia patients. Over the past several years, CRISPR-Cas9 has seized the public imagination for its ability to edit genetic code in a way that may correct defects inside individual cells — potentially healing mutations and preventing the advent of many illnesses.
By identifying an enzyme that can target the mechanisms by which cells operate, rather than the overall plan for cellular function, scientists should be able to come up with even more highly refined treatments with fewer risks.
But these enzymes target DNA, which is the fundamental building block for the development of an organism, and there are growing concerns that using the enzyme to essentially reprogram the DNA of a cell may cause more harm than good. Once thought to just be the delivery mechanism for instructions encoded in DNA for cell operations, RNA is now known to carry out biochemical reactions like enzymes, and serve their own regulatory functions in cells.
Griffin of The Scripps Research Institute, according to a statement. Specifically, Cas9 enzymes act sort of like scissors, snipping away pieces of genetic code and swapping them out with a replacement. Cas9 protein: