Efficient production of Fah-null heteroz
โ
Raymond D. Hickey; Joseph B. Lillegard; James E. Fisher; Travis J. McKenzie; Sea
๐
Article
๐
2011
๐
John Wiley and Sons
๐
English
โ 466 KB
Hereditary tyrosinemia type I (HT1) results in hepatic failure, cirrhosis, and hepatocellular carcinoma (HCC) early in childhood and is caused by a deficiency in the enzyme fumarylacetoacetate hydrolase (FAH). In a novel approach we used the chimeric adeno-associated virus DJ serotype (AAV-DJ) and h