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Novel genome editing strategy to treat genetic disorders

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.