Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.

Autor: 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.

Publikováno v: Human gene therapy [Hum Gene Ther] 2024 Apr; Vol. 35 (7-8), pp. 256-268. Date of Electronic Publication: 2024 Feb 02.

Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.

Autor: Catalano F; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Vlaar EC; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Katsavelis D; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Dammou Z; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Huizer TF; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van den Bosch JC; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Hoogeveen-Westerveld M; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van den Hout HJMP; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Oussoren E; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Ruijter GJG; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Schaaf G; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden 2333ZA, the Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden 2333ZA, the Netherlands.; Department of Pediatrics, Leiden University Medical Center, Leiden 2333ZA, the Netherlands., van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pijnappel WWMP; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Nov 02; Vol. 31, pp. 101149. Date of Electronic Publication: 2023 Nov 02 (Print Publication: 2023).

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

Autor: Liang Q; 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., Vlaar EC; 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., Catalano F; 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., Pijnenburg JM; 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., Stok M; 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., van Helsdingen Y; Department of Hematology, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands., Vulto AG; Hospital Pharmacy, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands., Unger WWJ; Laboratory of Pediatrics, Erasmus MC University Medical Center-Sophia Children's Hospital, 3015GE Rotterdam, the Netherlands., van der Ploeg AT; 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., Pijnappel WWMP; 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., van Til NP; Department of Hematology, Erasmus MC University Medical Center, 3015GE Rotterdam, the Netherlands.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 May 04; Vol. 25, pp. 520-532. Date of Electronic Publication: 2022 May 04 (Print Publication: 2022).