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  1. The First Curative Treatment of Phenylketonuria Using Genetic Engineering Approaches

    The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects. Problem: Phenylketonuria (PKU) is a severe disorder affecting approximately 1 in 10,000 U.S. newborns. PKU is marked by a genetic mutation in the phenylalanine hydroxylase enzyme (PAH), resulting in phenylalanine (Phe)...
  2. LNP Delivery of Gene-Editing Machinery to Cure Hereditary Tyrosinemia Type 1g

    Problem: Hereditary tyrosinemia type 1 (HT1) is a genetic metabolic disease caused by the inability to break down tyrosine, an amino acid present in many meats, dairy products, fruits, and nuts. This disease can be fatal within the first few months of life and increases the risk of liver cancer. The current treatment involves a strict drug regimen and...
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