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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)...
Published: 10/28/2024   |   Inventor(s): Kiran Musunuru, Xiao Wang, Dominique Brooks
Keywords(s): Gene Editing, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology
Category(s): Technology Classifications > Therapeutics