Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.

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第一作者： Qiushi,Liang

第一单位： Department of Hematology and Research Laboratory of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, P.R. China.;Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.

作者： Qiushi,Liang ^[1] ; Eva C,Vlaar ^[2] ; Fabio,Catalano ^[2] ; Joon M,Pijnenburg ^[2] ; Merel,Stok ^[3] ; Yvette,van Helsdingen ^[4] ; Arnold G,Vulto ^[5] ; Wendy W J,Unger ^[6] ; Ans T,van der Ploeg ^[7] ; W W M Pim,Pijnappel ^[2] ; Niek P,van Til ^[4]

作者单位： Department of Hematology and Research Laboratory of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, P.R. China.;Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[1] Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[2] Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Department of Hematology, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[3] Department of Hematology, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[4] Hospital Pharmacy, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[5] Laboratory of Pediatrics, Erasmus MC University Medical Center-Sophia Children's Hospital, 3015GE Rotterdam, the Netherlands. ^[6] Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.;Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands. ^[7]

关键词 Pompe disease acid alpha-glucosidase antibody formation immune response immune tolerance induction lentiviral gene therapy lysosomal storage disorders

DOI 10.1016/j.omtm.2022.04.016

PMID 35662813

发布时间 2022-07-16

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Molecular therapy. Methods & clinical development

2022年25卷

520-532页

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Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

相关期刊

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