The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.

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第一作者： Whitney S,Thompson

第一单位： Department of Pediatrics, Mayo Clinic, Rochester, MN, USA.

作者： Whitney S,Thompson ^[1] ; Gourish,Mondal ^[2] ; Caitlin J,Vanlith ^[3] ; Robert A,Kaiser ^[4] ; Joseph B,Lillegard ^[5]

作者单位： Department of Pediatrics, Mayo Clinic, Rochester, MN, USA. ^[1] Department of Surgery, Research Scientist, Mayo Clinic, Rochester, MN, USA. ^[2] MD/PhD student, Mayo Clinic, Rochester, MN, USA. ^[3] Department of Surgery, Research Scientist, Mayo Clinic, Rochester, MN, USA.;Midwest Fetal Care Center, Childrens Hospital of Minnesota, MN, USA. ^[4] Midwest Fetal Care Center, Childrens Hospital of Minnesota, MN, USA.;Assistant Professor of Surgery, Mayo Clinic, Rochester, MN, USA. ^[5]

关键词 Adeno-associated viral vector gene therapy hereditary tyrosinemia type 1 inborn errors of metabolism lentiviral vector

DOI 10.1080/21678707.2020.1791082

PMID 33224636

发布时间 2022-04-18

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The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.

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The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.

Expert opinion on orphan drugs

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Expert opinion on orphan drugs

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