Credit: ETH Zurich
Researchers at ETH Zurich in Basel have developed a novel method using CRISPR-Cas gene scissors to simultaneously change several genes within the cells of a single animal. The method will make laboratory animal research faster and more efficient.
Using the new method, only one gene within a cell is altered. However, numerous cells within a given organ can be altered simultaneously enabling researchers to precisely analyze the ramifications of numerous gene changes in a single experiment.
In the study, published in Nature, researchers were able to use this method for the first time in live adult mice. The researchers utilized adeno-associated virus (AAV) to deliver the CRISPR-Cas gene scissors to specific genes within a cell, a method that can be used to target any organ. The mice were infected with a mixture of viruses carrying various instructions for gene destruction, allowing them to “switch off” a number of different cells within one organ.
During testing, the researchers obtained new information about a rare genetic disorder in humans called 22q11.2 deletion syndrome. It has long been known that a chromosomal region of 106 genes is responsible for the disease, however, the particular genes responsible for the disease have eluded researchers until now. In their study, the researchers aimed their focus at 29 genes in this region that are active in both mice and human brains. In the study, the researchers showed that three of these genes are most responsible for brain cell dysfunction.
By allowing researchers to conduct analysis in living organisms, the novel method introduced could have lasting impacts on the industry. “In many congenital diseases, multiple genes play a role, not just one,” said António Santinha - lead author of the publication. “This is also the case with mental illnesses such as schizophrenia. Our technique now lets us study such diseases and their genetic causes directly in fully grown animals.”