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Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.
Liang, Qiushi; Vlaar, Eva C; Catalano, Fabio; Pijnenburg, Joon M; Stok, Merel; van Helsdingen, Yvette; Vulto, Arnold G; Unger, Wendy W J; van der Ploeg, Ans T; Pijnappel, W W M Pim; van Til, Niek P.
Affiliation
  • Liang Q; Department of Hematology and Research Laboratory of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, P.R. China.
  • Vlaar EC; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Catalano F; Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Pijnenburg JM; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Stok M; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • van Helsdingen Y; Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Vulto AG; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Unger WWJ; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • Pijnappel WWMP; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
  • van Til NP; Molecular Stem Cell Biology, Department of Clinical Genetics, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.
Mol Ther Methods Clin Dev ; 25: 520-532, 2022 Jun 09.
Article in En | MEDLINE | ID: mdl-35662813
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Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2022 Document type: Article
Fulltext
Print
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PubMed Links
Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: Mol Ther Methods Clin Dev Year: 2022 Document type: Article