Within reWRITE, we seek to improve the function of stem cells by editing their genetic information.

Rare genetic diseases affect around five percent of the global population. Although much progress has been made in identifying the rare genetic mutations that cause these diseases, there are still thousands of diseases where the gene defect is known but for which there are no available treatments.

Research within reWRITE focuses on integrating stem cell technologies and new approaches to gene therapy. reWRITE’s scientists are now able to alter the genetic information of a stem cell – its genome – enabling the repair of an inherited genetic defect or even to deliver a new cellular function.

reWRITE research lies at the intersection of gene and cell therapy. Being able to edit genes of stem cells to correct genetic mutations potentially means that a new generation of stem cell-based therapies for rare diseases is within reach.

In this way, we hope to revolutionize the treatment of rare and untreatable genetically inherited diseases. These include disorders of the immune system, muscle disorders present from birth that worsen over time, and neurological conditions.

reWRITE’s researchers focus on pushing new gene modifying technologies to reach clinical trials as quickly as possible. Our Leiden teams are already involved in first-in-man clinical trials for severe combined immunodeficiency syndrome (SCID), a rare genetic disorder that causes life-threatening problems with the immune system, through gene editing.