Scientists have developed a retron-based gene-editing tool that corrects multiple genetic mutations at once with unprecedented efficiency. It could transform gene therapy for disorders like cystic fibrosis by offering broad, affordable, and scalable treatment options. Credit: Shutterstock
Some inherited diseases, including cystic fibrosis, hemophilia, and Tay Sachs disease, involve multiple genetic mutations within a person’s DNA. Even two individuals with the same condition may have different sets of mutations. Because of this complexity, creating gene therapies that work broadly across all patients with a given disorder has been extremely difficult.
A New and More Efficient Gene Editing Breakthrough
Scientists at The University of Texas at Austin have developed a more precise and efficient gene-editing approach that can correct several disease-causing mutations at once in mammalian cells. The technique also successfully repaired mutations linked to scoliosis in zebrafish embryos.