Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1

Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). It has been previously shown that ex vivo hepatocyte-directed gene therapy using an integrating lentiviral vector to replace the defective Fah gene can cure liver disea...

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發表在:Hum Gene Ther
Main Authors: VanLith, Caitlin, Guthman, Rebekah, Nicolas, Clara T., Allen, Kari, Du, Zeji, Joo, Dong Jin, Nyberg, Scott L., Lillegard, Joseph B., Hickey, Raymond D.
格式: Artigo
語言:Inglês
出版: Mary Ann Liebert, Inc., publishers 2018
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Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6247987/
https://ncbi.nlm.nih.gov/pubmed/29764210
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.252
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