ERC Advanced Grant · 2019
Immune-privileged, immortal, myogenic stem cells for gene therapy of Muscular Dystrophy.
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 efficacy. However, this personalised approach would prove prohibitively expensive for healthcare systems, as pricing of successful gene therapies is showing. We made the striking observation that human…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.