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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.

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

LNP Delivery of Gene-Editing Machinery to Cure Hereditary Tyrosinemia Type 1g

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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