Generalizable in vivo genome editing therapeutics for Ehlers-Danlos Syndrome and other dominant genetic disorders

• Applicant: Dr. Johannes Karl Dohr
• Subject Area: Cardiology, Angiology
• Term: since 2024
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 555837174

This project aims to develop a specific gene therapy for genetically caused aortic diseases, using vascular Ehlers-Danlos syndrome as an example. The first objective is the specific correction of the causative mutation using base editing/prime editing. The second objective is the allele-specific disruption of the mutated allele and the upregulation of the expression of the healthy allele. The second strategy will also be tested for human mutations and thus serve as a basis for a clinical strategy that could be applied to patients.

• DFG Programme: WBP Fellowship
• International Connection: USA
• Host: Professor Dr. Gregory Arthur Newby