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The IMPACT project focuses on developing affordable gene correction therapies for rare genetic diseases, particularly Methylmalonic Acidemia. It aims to bridge the gap between scientific advancements in gene editing and practical applications in clinical settings.
Rare genetic diseases affect over 30 million people in Europe.
Although individually rare and very diverse, they are all caused by genetic mutations.
Recent progress in genetic technologies now holds the promise to correct the root cause of these diseases.
However, it remains difficult to develop these innovative technologies into affordable therapies for these diseases that only affect few patients, limiting commercial interest.IMPACT aims to overcome the translational gap between scientific ge…
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
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