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Article

Efficient production of Fah -null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer

14 Jun 2011

Abstract excerpt

UNLABELLED: 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 homologous recombination to target and disrupt the porcine Fah gene. AAV-DJ is an artificial chimeric AAV...

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PMID: 21674562
DOI: 10.1002/hep.24490

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