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Molecular biology and gene therapy for glycogen storage disease type Ib.
J Inherit Metab Dis. 2018 Nov;41(6):1007-1014. doi: 10.1007/s10545-018-0180-5. Epub 2018 Apr 16.
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Liver-directed gene therapy for murine glycogen storage disease type Ib.
Hum Mol Genet. 2017 Nov 15;26(22):4395-4405. doi: 10.1093/hmg/ddx325.

本文引用的文献

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors.
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Neutrophil stress and apoptosis underlie myeloid dysfunction in glycogen storage disease type Ib.
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Molecular basis and mechanisms of progression of non-alcoholic steatohepatitis.
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AAV vector integration sites in mouse hepatocellular carcinoma.
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