ERC Starting Grant · 2021
Treatment options are insufficient for the majority of genetic metabolic diseases. Methylmalonic acidemia (MMA) is one such severe metabolic disease. With current therapeutic strategies, patients still develop metabolic decompensations, brain damage, kidney failure, and have a lifelong risk to acutely become blind. Repair of the genetic cause of disease would revolutionize outcomes for these patients, especially with increasing detection in neonatal screening programs. This makes it possible to correct the cause of disease prior to the onset of irreversible damage. However, efficiency, safety, versatility and delivery of precise gene editing is not yet sufficient for in vivo gene-correction…
From the public funding record at EU CORDIS. Describes the funded project, not the reviews below.