Testing the efficacy of new gene therapies more efficiently
Better Cell Model Developed Thanks to 'Gene Scissors' Recently, a research team headed by Janine Reichenbach, a UZH professor and Co-Head of the Division of Immunology at the University Children's Hospital Zurich, has developed a new cellular model that enables to test the efficacy of new gene therapies much more efficiently. "We used Crispr/Cas9 technology to change a human cell line so that the blood cells show the genetic change typical of a specific form of Chronic Granulomatous Disease," explains the pediatrician and immunologist. In this way, the modified cells reflect the disease genetically and functionally. Until now, scientists had to rely on using patients' skin cells that they had reprogrammed into stem cells in the lab. This approach is laborious, and requires considerable time and money. "With our new testing system, this process is faster and cheaper, enabling us to develop new gene therapies for affected patients more efficiently,...