Novel genome editing strategy to treat genetic disorders
![](https://www.starfishinnovations.com/wp-admin/admin-ajax.php?action=kernel&p=image&src=%7B%22file%22%3A%22wp-content%2Fuploads%2F2020%2F12%2Fbigstock-221731816.jpg%22%2C%22thumbnail%22%3A%221200%2C520%2C%22%7D&hash=d0618a98)
Start date project: 2023
Involved institutes: LUMC
Stage: Preclinical
Status: Ongoing
Description: For the genetic disorder Duchenne Muscular Dystrophy (DMD) there is currently no universal therapy available that stops the muscle deterioration. The aim of this project is to test a novel and precise gene editing approach for DMD patients with a variety of underlying mutations in the dystrophin gene. This technology could be applied in gene therapies for other genetic diseases and metabolic disorders as well.