| Abstract |
1 in 5000 people live with Marfan syndrome, a connective tissue disorder affecting the FBN1 gene, increasing the risk of aortic dissections. Our project proposes a novel genetic engineering technology, base editing, to prevent these life-threatening complications by repairing aortic cells, saving lives and improving life quality. It makes single-stranded breaks in DNA to modify missense errors in genes, having shown promise in pre-clinical trials in stem cells from a Marfan patient and in mouse models with a similar aortic disorder. |
