A Study on Preventing Aortic Dissections in Marfan Patients by Base Editing Smooth Muscle Cells
CWSF · 2026 Disease & Illness
Overview
Marfan syndrome (MFS), a connective tissue disorder caused by FBN1 gene mutations, affects 1 in 5000 individuals. Current interventions for MFS are insufficient; they only mitigate symptoms and must be continuously maintained. In this project, we synthesized scientific research to identify technologies for eliminating the severest MFS symptom: aortic aneurysm and dissection. We propose a novel genetic modification tool, base editing, for repairing aortic cells by changing individual letters in the genetic code to correct missense mutations (the leading cause of MFS). Base editors have rescued mouse models with similar aortic dilation and restored MFS stem cells to normal morphology and karyotype. This treatment should be administered pediatrically, leveraging sequencing to identify the mutations’ loci and using adeno-associated virus 9 as the vector. The foremost challenge will be to attain the critical mass of edited cells to create a positive feedback loop, significantly improving cellular environment regulation and patient outcomes.
Awards (1)
- Selected for CWSF 2026
Competition history
- CWSF 2026
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