Adenine base editing in an adult mouse model of tyrosinaemia
نویسندگان
چکیده
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15 صفحه اولHomologous recombination mediates stable Fah gene integration and phenotypic correction in tyrosinaemia mouse-model
AIM To stably correct tyrosinaemia in proliferating livers of fumarylacetoacetate-hydrolase knockout (Fah-/-) mice by homologous-recombination-mediated targeted addition of the Fah gene. METHODS C57BL/6 Fah∆exon5 mice served as an animal model for human tyrosinaemia type 1 in our study. The vector was created by amplifying human Fah cDNA including the TTR promoter from a lentivirus plasmid as...
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ژورنال
عنوان ژورنال: Nature Biomedical Engineering
سال: 2019
ISSN: 2157-846X
DOI: 10.1038/s41551-019-0357-8