ERC Starting Grant · 2024
Next generation versatile and effective AAV-mediated large gene delivery
Despite 6 products on the market and dozens in advanced phases of clinical development, in vivo gene therapy with vectors derived from adeno-associated viruses (AAV) still faces the major challenge of the limited vector cargo capacity (< 5kb). This prevents AAV application for treatment of inherited diseases caused by mutations in large genes. To address this, we have recently developed an AAV-based strategy relying on the use of short protein elements called inteins. These, when fused at the extremities of fragments of a large protein delivered to cells through AAV, can mediate joining of the fragments in a traceless manner, resulting in the reconstitution of the intact target protein.…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.