CORDIS Project
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This project focuses on developing a cost-effective therapy for Duchenne muscular dystrophy by genetically engineering mesoangioblasts to correct dystrophin gene mutations. The approach aims to create immune-privileged cells that can be expanded and used for transplantation, potentially revolutionizing treatment for th…
Duchenne muscular dystrophy (DMD) is a devastating incurable disease, affecting thousands with heavy burden on health systems.
This project combines the development of a safe, immune-privileged cell with genetic engineering to correct many dystrophin gene mutations for an efficacious and cost affordable therapy.
The applicant pioneered systemic intra-arterial transplantation of mesoangioblasts (blood vessel-derived progenitors) that proved safe in DMD patients and is being implemented for effica…
OSPEDALE SAN RAFFAELE SRL
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