CORDIS Project
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This project aims to develop a gene-editing therapy for methylmalonic acidemia, a severe genetic metabolic disease. By utilizing a novel technique called prime editing, the project focuses on correcting genetic mutations in liver cells, with the goal of improving patient outcomes through personalized treatment strategi…
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…
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
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