CRISPR's Revolutionary Impact on Genetic Disorder Research in Animals

Researchers at ETH Zurich have developed a method to genetically modify individual cells in animals, allowing for the study of gene changes and their effects in a single experiment. Using adeno-associated virus (AAV)-mediated direct in vivo single-cell CRISPR screening, the researchers were able to switch off different genes in the cells of the mouse brain. This method provided new insights into a rare genetic disorder, 22q11.2 deletion syndrome, identifying three genes responsible for brain cell dysfunction. The technique allows for the study of genetic causes of diseases directly in fully grown animals, providing a more accurate representation of cell behavior in living organisms.