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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...
Published: 7/10/2024   |   Inventor(s): Kiran Musunuru, Madelynn Whittaker, Xiao Wang, William Peranteau, Cara Berkowitz, Ana Dumitru
Keywords(s): Gene Editing, Genetic Disorders, Obesity, Metabolic Disease and Diabetes
Category(s): Technology Classifications > Therapeutics