نتائج البحث - "Marteau JB"

المؤلفون: Ferla R; Telethon Institute of Genetics and Medicine (TIGEM), Via Campi Flegrei 34, 80078 Pozzuoli, Italy.; AAVantgarde BIO Srl, 20123 Milan, Italy., Dell'Aquila F; Telethon Institute of Genetics and Medicine (TIGEM), Via Campi Flegrei 34, 80078 Pozzuoli, Italy., Doria M; Telethon Institute of Genetics and Medicine (TIGEM), Via Campi Flegrei 34, 80078 Pozzuoli, Italy., Ferraiuolo M; ReiThera Srl, 00128 Rome, Italy., Noto A; ReiThera Srl, 00128 Rome, Italy., Grazioli F; ReiThera Srl, 00128 Rome, Italy., Ammendola V; ReiThera Srl, 00128 Rome, Italy., Testa F; Eye Clinic, Multidisciplinary Department of Medical Surgical and Dental Sciences, University of Campania 'Luigi Vanvitelli', 80131 Naples, Italy., Melillo P; Eye Clinic, Multidisciplinary Department of Medical Surgical and Dental Sciences, University of Campania 'Luigi Vanvitelli', 80131 Naples, Italy., Iodice C; Telethon Institute of Genetics and Medicine (TIGEM), Via Campi Flegrei 34, 80078 Pozzuoli, Italy., Risca G; Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy., Tedesco N; Genethon, 91000 Evry, France.; Université Paris-Saclay, University Evry 91000, INSERM, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., le Brun PR; Genethon, 91000 Evry, France.; Université Paris-Saclay, University Evry 91000, INSERM, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Surace EM; Medical Genetics, Department of Translational Medicine, University of Naples 'Federico II', 80131 Naples, Italy., Simonelli F; Eye Clinic, Multidisciplinary Department of Medical Surgical and Dental Sciences, University of Campania 'Luigi Vanvitelli', 80131 Naples, Italy., Galimberti S; Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy., Valsecchi MG; Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milano-Bicocca, 20900 Monza, Italy., Marteau JB; Genosafe, 91000 Evry, France., Veron P; Genethon, 91000 Evry, France.; Université Paris-Saclay, University Evry 91000, INSERM, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Colloca S; ReiThera Srl, 00128 Rome, Italy., Auricchio A; Telethon Institute of Genetics and Medicine (TIGEM), Via Campi Flegrei 34, 80078 Pozzuoli, Italy.; AAVantgarde BIO Srl, 20123 Milan, Italy.; Department of Advanced Biomedical Sciences, 'Federico II' University, 80131 Naples, Italy.

المصدر: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Feb 11; Vol. 28, pp. 396-411. Date of Electronic Publication: 2023 Feb 11 (Print Publication: 2023).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

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دورية أكاديمية

Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.

المؤلفون: Brunetti-Pierri N; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Ferla R; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Ginocchio VM; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Rossi A; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Fecarotta S; Dipartimento ad Attivitá Integrata Materno Infantile, 'Federico II' University, Naples, Italy., Romano R; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Parenti G; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Yildiz Y; Division of Pediatric Metabolism, Ihsan Dogramaci Children's Hospital, Hacettepe University, Ankara, Turkey., Zancan S; Fondazione Telethon ETS, Rome, Italy., Pecorella V; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy., Dell'Anno M; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Graziano M; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy., Alliegro M; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy., Andria G; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Santamaria F; Department of Translational Medicine, 'Federico II' University, Naples, Italy., Brunetti-Pierri R; Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Università degli Studi della Campania 'Luigi Vanvitelli,' Naples, Italy., Simonelli F; Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Università degli Studi della Campania 'Luigi Vanvitelli,' Naples, Italy., Nigro V; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Precision Medicine, Università degli Studi della Campania 'Luigi Vanvitelli,' Naples, Italy., Vargas M; Department of Neurosciences, Reproductive and Odontostomatological Sciences, 'Federico II' University, Naples, Italy., Servillo G; Department of Neurosciences, Reproductive and Odontostomatological Sciences, 'Federico II' University, Naples, Italy., Borgia F; Department of Advanced Biomedical Sciences, Divisions of Cardiology and Cardiothoracic Surgery, 'Federico II' University, Naples, Italy., Soscia E; Institute of Biostructure and Bioimaging, National Research Council, Napoli, Italy., Gargaro M; Department of Medicine and Surgery, University of Perugia, Perugia, Italy., Funghini S; Newborn Screening, Clinical Chemistry and Pharmacology Laboratory, Meyer Children's University Hospital, Florence, Italy., Tedesco N; Genethon, Evry, France.; Integrare Research Unit UMR_S951 of Université Paris-Saclay, University Evry, INSERM, Evry, France., Le Brun PR; Genethon, Evry, France.; Integrare Research Unit UMR_S951 of Université Paris-Saclay, University Evry, INSERM, Evry, France., Rupar CA; London Health Science Centre, Western University, London, Ontario, Canada., Prasad C; London Health Science Centre, Western University, London, Ontario, Canada., O'Callaghan M; Servicio de Neurología, Unidad de Enfermedades Metabólicas, Clínica Rett, Hospital Sant Joan de Déu, Barcelona, Spain., Mitchell JJ; Division of Medical Genetics, Montreal Children's Hospital, McGill University Health Centre, Montreal, Quebec, Canada.; Division of Pediatric Endocrinology, Montreal Children's Hospital, McGill University Health Centre, Montreal, Quebec, Canada., Danos O; REGENXBIO Inc, Rockville, MD., Marteau JB; Genosafe SAS, Evry, France., Galimberti S; Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy., Valsecchi MG; Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy., Veron P; Genethon, Evry, France.; Integrare Research Unit UMR_S951 of Université Paris-Saclay, University Evry, INSERM, Evry, France., Mingozzi F; Genethon, Evry, France., Fallarino F; Department of Medicine and Surgery, University of Perugia, Perugia, Italy., la Marca G; Newborn Screening, Clinical Chemistry and Pharmacology Laboratory, Meyer Children's University Hospital, Florence, Italy.; Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy., Sivri HS; Division of Pediatric Metabolism, Ihsan Dogramaci Children's Hospital, Hacettepe University, Ankara, Turkey., Auricchio A; Telethon Institute of Genetics and Medicine, Pozzuoli, Naples, Italy.; Department of Advanced Biomedical Sciences, 'Federico II' University, Naples, Italy.

المصدر: NEJM evidence [NEJM Evid] 2022 Jul; Vol. 1 (7), pp. EVIDoa2200052. Date of Electronic Publication: 2022 Jun 06.

نوع المنشور: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I

بيانات الدورية: Publisher: NEJM Group, a division of the Massachusetts Medical Society Country of Publication: United States NLM ID: 9918317485806676 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2766-5526 (Electronic) Linking ISSN: 27665526 NLM ISO Abbreviation: NEJM Evid Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy*/methods , Mucopolysaccharidosis VI*/therapy , Mucopolysaccharidosis VI*/genetics , Mucopolysaccharidosis VI*/urine , Dependovirus*/genetics , N-Acetylgalactosamine-4-Sulfatase*/genetics, Humans ; Male ; Female ; Child ; Adolescent ; Child, Preschool ; Adult ; Young Adult ; Genetic Vectors/administration & dosage ; Liver/metabolism ; Liver/pathology ; Enzyme Replacement Therapy/methods

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