Testing the Efficacy of New Gene Therapies More Efficiently

Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested faster and cost-effectively in the lab for their efficacy. A team of researchers from the University of Zurich and the Children’s Hospital Zurich successfully achieved this using the ‘gene-scissor’ CRISPR/Cas9 technology. The aim is to treat severely affected patients in the near future using novel approaches. (Mehr in: Pressemitteilungen – idw – Informationsdienst Wissenschaft)