Researchers at The University of Texas at Austin have developed a new, precise gene editing method that can correct multiple disease-causing mutations in mammalian cells. This breakthrough technology uses genetic elements from bacteria to protect the microbes from viral infection and has shown promise in treating scoliosis and other diseases.
The current gene editing methods are limited to one or two mutations, leaving many people with rare genetic disorders behind. The new method can replace a large stretch of defective DNA with a healthy sequence, making it a more inclusive solution for patients. By developing an off-the-shelf tool that can cure multiple patients at once, the researchers hope to “democratize” gene therapy and make it more financially viable.
The team has already demonstrated its effectiveness in correcting scoliosis-causing mutations in zebrafish embryos and plans to apply this approach to develop gene therapies for cystic fibrosis. With over 1,000 mutations that can cause the disease, traditional gene editing technologies are not effective for all patients. The new method could impact a larger part of the CF population.
This research has the potential to revolutionize the treatment of rare genetic disorders and could lead to significant advancements in personalized medicine.
Source: https://www.eurekalert.org/news-releases/1102990