Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells.

Autor: Bulcaen M; Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000 Leuven, Belgium; Department of Chronic Diseases and Metabolism, KU Leuven, 3000 Leuven, Belgium. Electronic address: mattijs.bulcaen@kuleuven.be., Kortleven P; Department of Chronic Diseases and Metabolism, KU Leuven, 3000 Leuven, Belgium., Liu RB; Department of Biosystems, KU Leuven, 3000 Leuven, Belgium; School of Engineering, University of Edinburgh, EH9 3JL Edinburgh, UK., Maule G; Department of CIBIO, University of Trento, 38123 Povo-Trento, Italy., Dreano E; INSERM, CNRS, Institut Necker Enfants Malades, 75015 Paris, France; Université Paris-Cité, 75015 Paris, France., Kelly M; INSERM, CNRS, Institut Necker Enfants Malades, 75015 Paris, France; Université Paris-Cité, 75015 Paris, France., Ensinck MM; Department of Chronic Diseases and Metabolism, KU Leuven, 3000 Leuven, Belgium., Thierie S; Department of Chronic Diseases and Metabolism, KU Leuven, 3000 Leuven, Belgium., Smits M; Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000 Leuven, Belgium; Leuven Viral Vector Core, KU Leuven, 3000 Leuven, Belgium., Ciciani M; Department of CIBIO, University of Trento, 38123 Povo-Trento, Italy., Hatton A; INSERM, CNRS, Institut Necker Enfants Malades, 75015 Paris, France; Université Paris-Cité, 75015 Paris, France., Chevalier B; INSERM, CNRS, Institut Necker Enfants Malades, 75015 Paris, France; Université Paris-Cité, 75015 Paris, France., Ramalho AS; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium., Casadevall I Solvas X; Department of Biosystems, KU Leuven, 3000 Leuven, Belgium., Debyser Z; Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000 Leuven, Belgium; Leuven Viral Vector Core, KU Leuven, 3000 Leuven, Belgium., Vermeulen F; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium; Department of Pediatrics, UZ Leuven, 3000 Leuven, Belgium., Gijsbers R; Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, 3000 Leuven, Belgium; Leuven Viral Vector Core, KU Leuven, 3000 Leuven, Belgium., Sermet-Gaudelus I; INSERM, CNRS, Institut Necker Enfants Malades, 75015 Paris, France; Université Paris-Cité, 75015 Paris, France; Cystic Fibrosis National Pediatric Reference Center, Pneumo-Allergologie Pédiatrique, Hôpital Necker Enfants Malades, Assistance Publique Hôpitaux de Paris (AP-HP), 75015 Paris, France; European Reference Network, ERN-Lung CF, 60596 Frankfurt am Mein, Germany., Cereseto A; Department of CIBIO, University of Trento, 38123 Povo-Trento, Italy., Carlon MS; Department of Chronic Diseases and Metabolism, KU Leuven, 3000 Leuven, Belgium; Leuven Viral Vector Core, KU Leuven, 3000 Leuven, Belgium. Electronic address: marianne.carlon@kuleuven.be.

Publikováno v: Cell reports. Medicine [Cell Rep Med] 2024 May 21; Vol. 5 (5), pp. 101544. Date of Electronic Publication: 2024 May 01.

Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases.

Autor: Medaer L; Laboratory of Molecular Virology and Gene Therapy, Department of Pharmacological and Pharmaceutical Sciences, Faculty of Medicine., Veys K; Laboratory of Paediatric Nephrology, Department of Development and Regeneration, Faculty of Medicine., Gijsbers R; Laboratory of Molecular Virology and Gene Therapy, Department of Pharmacological and Pharmaceutical Sciences, Faculty of Medicine.; Leuven Viral Vector Core, Faculty of Medicine; KU Leuven, Leuven, Belgium.

Publikováno v: Human gene therapy [Hum Gene Ther] 2024 Mar; Vol. 35 (5-6), pp. 139-150.

Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.

Autor: Bondue T; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium., Berlingerio SP; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium., Siegerist F; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany., Sendino-Garví E; Division Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands., Schindler M; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany., Baelde HJ; Department of Pathology, Leiden University Medical Center, Leiden, The Netherlands., Cairoli S; Laboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Goffredo BM; Laboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Arcolino FO; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; Department of Pediatric Nephrology, Emma Children's Hospital and Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands., Dieker J; Mercurna B.V., Oss, The Netherlands., Janssen MJ; Division Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands., Endlich N; Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany., Brock R; Department of Medical Biosciences, Radboud University Medical Center, Nijmegen, The Netherlands.; Department of Medical Biochemistry, College of Medicine and Medical Sciences, Arabian Gulf University, Manama, Kingdom of Bahrain., Gijsbers R; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.; Leuven Viral Vector Core (LVVC), KU Leuven, Leuven, Belgium., van den Heuvel L; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.; Department of Pediatric Nephrology, Radboud University Medical Center, Nijmegen, The Netherlands., Levtchenko E; Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium. e.n.levtchenko@amsterdamumc.nl.; Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, H7-234, Meibergdreef 9, 1105AZ, Amsterdam, The Netherlands. e.n.levtchenko@amsterdamumc.nl.

Publikováno v: Scientific reports [Sci Rep] 2023 Nov 28; Vol. 13 (1), pp. 20961. Date of Electronic Publication: 2023 Nov 28.

Nanoblades allow high-level genome editing in murine and human organoids.

Autor: Tiroille V; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.; Equipe labélisée Ligue National Contre le Cancer, Basel, Switzerland., Krug A; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France., Bokobza E; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.; Equipe labélisée Ligue National Contre le Cancer, Basel, Switzerland., Kahi M; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.; Equipe labélisée Ligue National Contre le Cancer, Basel, Switzerland., Bulcaen M; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven, Leuven, Belgium.; Laboratory of Respiratory Diseases and Thoracic Surgery (BREATHE), Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium., Ensinck MM; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven, Leuven, Belgium.; Laboratory of Respiratory Diseases and Thoracic Surgery (BREATHE), Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium., Geurts MH; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Hendriks D; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Vermeulen F; UZ Leuven, Department of Pediatrics, Leuven, Belgium., Larbret F; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France., Gutierrez-Guerrero A; CIRI - International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, F-69007 Lyon, France., Chen Y; Human Oncology and Pathogenesis Program, Department of Medicine, Memorial Sloan; Department of Medicine, Weill Cornell Medical College, New York, NY 10065, USA., Van Zundert I; Synthetic Biology Group, Department of Biomedical Engineering, Eindhoven University of Technology, P.O. Box 513, 5600 MB Eindhoven, the Netherlands., Rocha S; Molecular Imaging and Photonics, Department of Chemistry, KU Leuven, Celestijnenlaan 200F, 3001 Heverlee, Belgium., Rios AC; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands.; Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands., Medaer L; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven, Leuven, Belgium., Gijsbers R; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven, Leuven, Belgium., Mangeot PE; CIRI - International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, F-69007 Lyon, France., Clevers H; Hubrecht Institute, Royal Netherlands Academy of Arts and Sciences (KNAW) and University Medical Center Utrecht, Utrecht, the Netherlands.; Oncode Institute, Hubrecht Institute, Utrecht, the Netherlands., Carlon MS; Laboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, Faculty of Medicine, KU Leuven, Leuven, Belgium.; Laboratory of Respiratory Diseases and Thoracic Surgery (BREATHE), Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium., Bost F; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.; Equipe labélisée Ligue National Contre le Cancer, Basel, Switzerland., Verhoeyen E; Université Côte d'Azur, INSERM, C3M, 06204 Nice, France.; CIRI - International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, F-69007 Lyon, France.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2023 Jun 08; Vol. 33, pp. 57-74. Date of Electronic Publication: 2023 Jun 08 (Print Publication: 2023).

Case report: Myocarditis in congenital STAT1 gain-of function.

Autor: Staels F; Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium.; Department of Microbiology, Immunology and Transplantation, Laboratory of Adaptive Immunology, KU Leuven, Leuven, Belgium., Roosens W; Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium., Giovannozzi S; Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium.; Department of Pharmaceutical and Pharmacological Sciences, Laboratory of Molecular Virology and Gene Therapy, KU Leuven, Leuven, Belgium., Moens L; Department of Microbiology, Immunology and Transplantation, Laboratory of Inborn Errors of Immunity, KU Leuven, Leuven, Belgium., Bogaert J; Department of Imaging and Pathology, Translational MRI, KU Leuven, Leuven, Belgium., Iglesias-Herrero C; Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium.; Department of Pharmaceutical and Pharmacological Sciences, Laboratory of Molecular Virology and Gene Therapy, KU Leuven, Leuven, Belgium., Gijsbers R; Department of Pharmaceutical and Pharmacological Sciences, Laboratory of Molecular Virology and Gene Therapy, KU Leuven, Leuven, Belgium., Bossuyt X; Department of Microbiology, Immunology and Transplantation, Experimental Laboratory Immunology, KU Leuven, Leuven, Belgium., Frans G; Department of Microbiology, Immunology and Transplantation, Experimental Laboratory Immunology, KU Leuven, Leuven, Belgium., Liston A; Department of Microbiology, Immunology and Transplantation, Laboratory of Adaptive Immunology, KU Leuven, Leuven, Belgium.; Laboratory of Lymphocyte Signaling and Development, Babraham Institute, Cambridge, United Kingdom., Humblet-Baron S; Department of Microbiology, Immunology and Transplantation, Laboratory of Adaptive Immunology, KU Leuven, Leuven, Belgium., Meyts I; Department of Microbiology, Immunology and Transplantation, Laboratory of Inborn Errors of Immunity, KU Leuven, Leuven, Belgium.; Department of Pediatrics, University Hospitals Leuven, Leuven, Belgium., Van Aelst L; Department of Cardiovascular Diseases, University Hospitals Leuven, Leuven, Belgium., Schrijvers R; Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium.

Publikováno v: Frontiers in immunology [Front Immunol] 2023 Mar 20; Vol. 14, pp. 1095595. Date of Electronic Publication: 2023 Mar 20 (Print Publication: 2023).