Towards effective gene therapy for more metabolic disorders
What innovative techniques can we use to treat rare metabolic disorders? “We often know the genetic cause of rare metabolic disorders, but we are not yet able to cure them,” says Sabine Fuchs, Professor of Metabolic Disorders and Innovative Therapies.
In the AT-PRIME project, Sabine Fuchs and her team are developing a new method designed to correct genetic mutations that are currently beyond the scope of existing technologies. The project builds on years of research into why current gene therapy works well for some mutations, but remains inadequate or unpredictable for many others.
A major challenge is to demonstrate that ‘prime editing’, an advanced form of CRISPR-Cas9, can be applied safely and effectively to various disease-causing mutations.
Congratulations to Sabine and the team on securing an ERC Proof of Concept grant to support this project.
About Regenerative Medicine Utrecht
Regenerative Medicine Utrecht(RMU) is a world-renowned collaboration of excellent knowledge institutes and start-ups, located at the Utrecht Science Park. We bring together researchers from UMC Utrecht, Utrecht University (UU) and the Hubrecht Institute. Our focus is on restoring health and vitality instead of symptom control. By repairing, replacing, or regenerating cells, tissues, and organs, we aim to cure patients.