Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
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| Title: | Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage. |
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| Authors: | Catalano F; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Vlaar EC; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Dammou Z; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Katsavelis D; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Huizer TF; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Zundo G; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Hoogeveen-Westerveld M; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Oussoren E; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., van den Hout HJMP; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Schaaf G; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands., van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Pijnappel WWMP; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands. |
| Source: | Human gene therapy [Hum Gene Ther] 2024 Apr; Vol. 35 (7-8), pp. 256-268. Date of Electronic Publication: 2024 Feb 02. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: M.A. Liebert Country of Publication: United States NLM ID: 9008950 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1557-7422 (Electronic) Linking ISSN: 10430342 NLM ISO Abbreviation: Hum Gene Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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