European Society for Immunodeficiencies guidelines for the management of patients with congenital athymia.

Autor: Kreins AY; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom; Infection Immunity and Inflammation Research and Teaching Department, University College London Great Ormond Street Institute of Child Health, London, United Kingdom. Electronic address: a.kreins@ucl.ac.uk., Dhalla F; Department of Paediatrics and Institute of Developmental and Regenerative Medicine, University of Oxford, Oxford, United Kingdom; Department of Clinical Immunology, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom., Flinn AM; Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom; Paediatric Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom; Department of Paediatric Immunology, Children's Health Ireland at Crumlin, Crumlin, Ireland., Howley E; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Ekwall O; Department of Pediatrics, Institute of Clinical Sciences, The Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden; Department of Rheumatology and Inflammation Research, Institute of Medicine, The Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden., Villa A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Hospital, Milan, Italy; Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale Delle Ricerche (IRGB-CNR), Milan, Italy., Staal FJT; Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Willem-Alexander Children's Hospital, Leiden, The Netherlands; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Anderson G; Institute of Immunology and Immunotherapy, Medical School, University of Birmingham, Birmingham, United Kingdom., Gennery AR; Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom; Paediatric Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom., Holländer GA; Department of Paediatrics and Institute of Developmental and Regenerative Medicine, University of Oxford, Oxford, United Kingdom; Paediatric Immunology, Department of Biomedicine, University of Basel and University Children's Hospital Basel, Basel, Switzerland; Department of Biosystems Science and Engineering, ETH Zurich, Basel, Switzerland., Davies EG; Department of Immunology and Gene Therapy, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom; Infection Immunity and Inflammation Research and Teaching Department, University College London Great Ormond Street Institute of Child Health, London, United Kingdom.

Publikováno v: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2024 Dec; Vol. 154 (6), pp. 1391-1408. Date of Electronic Publication: 2024 Sep 18.

Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells.

Autor: Sorel N; Human Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France., Díaz-Pascual F; ProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany., Bessot B; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France., Sadek H; Human Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France., Mollet C; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France., Chouteau M; Human Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France., Zahn M; ProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany., Gil-Farina I; ProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany., Tajer P; Department of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands., van Eggermond M; Department of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands., Berghuis D; Department of Pediatrics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands., Lankester AC; Department of Pediatrics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands., André I; Human Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France., Gabriel R; ProtaGene CGT GmbH, Im Neuenheimer Feld 582, 69120 Heidelberg, Germany., Cavazzana M; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France.; Biotherapy Department, Necker-Enfants Malades Hospital, AP-HP, 75015 Paris, France.; Imagine Institute UMR1163, Université Paris Cité, Sorbonne Paris Cité, 75015 Paris, France., Pike-Overzet K; Biotherapy Department, Necker-Enfants Malades Hospital, AP-HP, 75015 Paris, France., Staal FJT; Department of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands.; Department of Pediatrics, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands., Lagresle-Peyrou C; Human Lymphohematopoiesis Laboratory, Université Paris Cité, Imagine Institute, INSERM UMR 1163, 75015 Paris, France.; Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM, 75015 Paris, France.

Publikováno v: Biomedicines [Biomedicines] 2024 Jul 05; Vol. 12 (7). Date of Electronic Publication: 2024 Jul 05.

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.

Advances in gene therapy for inborn errors of immunity.

Autor: Ott de Bruin LM; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology.; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Lankester AC; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology., Staal FJT; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program and Laboratory for Pediatric Immunology.; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.

Publikováno v: Current opinion in allergy and clinical immunology [Curr Opin Allergy Clin Immunol] 2023 Dec 01; Vol. 23 (6), pp. 467-477. Date of Electronic Publication: 2023 Oct 13.

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).