BioCentury
ARTICLE | Distillery Therapeutics

Therapeutics: Fumarylacetoacetate hydrolase (FAH)

August 18, 2016 7:00 AM UTC

Mouse and pig studies suggest ex vivo FAH gene therapy could help treat hereditary tyrosinemia type 1, which is caused by FAH deficiency. In a mouse model of the disease, transplants of mouse hepatocy...