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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.

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第一作者： Estera,Rintz

第一单位： Department of Molecular Biology, Faculty of Biology, University of Gdansk, Wita Stwosza, 59, 80-308 Gdansk, Poland.;Nemours/Alfred I. duPont Hospital for Children, 1600 Rockland Road, Wilmington, DE 19803, USA.

作者： Estera,Rintz ^[1] ; Takashi,Higuchi ^[2] ; Hiroshi,Kobayashi ^[2] ; Deni S,Galileo ^[3] ; Grzegorz,Wegrzyn ^[4] ; Shunji,Tomatsu ^[5]

作者单位： Department of Molecular Biology, Faculty of Biology, University of Gdansk, Wita Stwosza, 59, 80-308 Gdansk, Poland.;Nemours/Alfred I. duPont Hospital for Children, 1600 Rockland Road, Wilmington, DE 19803, USA. ^[1] Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, 3 Chome-25-8 Nishishinbashi, Minato City, Tokyo 105-8461, Japan. ^[2] Department of Biological Sciences, University of Delaware, 118 Wolf Hall, Newark, DE 19716, USA. ^[3] Department of Molecular Biology, Faculty of Biology, University of Gdansk, Wita Stwosza, 59, 80-308 Gdansk, Poland. ^[4] Nemours/Alfred I. duPont Hospital for Children, 1600 Rockland Road, Wilmington, DE 19803, USA.;Department of Biological Sciences, University of Delaware, 118 Wolf Hall, Newark, DE 19716, USA.;Department of Pediatrics, Gifu University, Gifu, Yanagido 501-1193, Japan.;Department of Pediatrics, Thomas Jefferson University, 901 Walnut Street, Philadelphia, PA 19107, USA. ^[5]

关键词 gene promoter gene therapy lentivirus lysosomal storage disease stem cell therapy

DOI 10.1016/j.omtm.2021.11.007

PMID 34977274

发布时间 2024-04-04

基金项目

P30 GM114736/GM/NIGMS NIH HHS/United States R01 HD065767/HD/NICHD NIH HHS/United States R01 HD102545/HD/NICHD NIH HHS/United States

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Molecular therapy. Methods & clinical development

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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.

Molecular therapy. Methods & clinical development

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Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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