Breakthrough Gene Editing Tool and Technique Developed by Weill Cornell Researchers for Investigating Cancer Mutations and Abnormal RNA Splicing

Researchers at Weill Cornell have developed a new gene editing tool that utilizes CRISPR-Cas9 technology to study cancer mutations in preclinical mouse models. The tool combines Cas9 and guide RNA with APOBEC, an enzyme that creates single base mutations in DNA. The team faced challenges with unwanted mutations and varying gene expression, but overcame them by integrating a single gene copy controlled by doxycycline. The tool has the potential to understand the effects of genetic changes on tumors, develop effective therapies, and study other disorders beyond cancer.