المؤلفون: Ferla, Rita, Dell’Aquila, Fabio, Doria, Monica, Ferraiuolo, Maria, Noto, Alessia, Grazioli, Fabiana, Ammendola, Virginia, Testa, Francesco, Melillo, Paolo, Iodice, Carolina, Risca, Giulia, Tedesco, Novella, le Brun, Pierre Romain, Surace, Enrico Maria, Simonelli, Francesca, Galimberti, Stefania, Valsecchi, Maria Grazia, Marteau, Jean-Brice, Veron, Philippe, Colloca, Stefano, Auricchio, Alberto

المصدر: In Molecular Therapy - Methods & Clinical Development 9 March 2023 28:396-411

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المؤلفون: Nicola Brunetti-Pierri, M. D. 1 2, Rita Ferla, Ph. D. 1, 2, Virginia Maria Ginocchio, M. D., Alessandro Rossi, Ph. D. 2, Simona Fecarotta, Ph. D. 3, Roberta Romano, M. D. 2, Giancarlo Parenti, Yilmaz Yildiz, Ph. D. 4, Stefano Zancan, M. Sc. 5, Valentina Pecorella, M. Sc. 1, Margherita Dell’Anno, Mafalda Graziano, Marialuisa Alliegro, Generoso Andria, Francesca Santamaria, Raffaella Brunetti-Pierri, Ph. D. 6, Francesca Simonelli, M. D. 6, Vincenzo Nigro, M. D. 1 7, Maria Vargas, M. D. 8, Giuseppe Servillo, Francesco Borgia, M. D. 9, Ernesto Soscia, M. D. 10, Marco Gargaro, Ph. D. 11, Silvia Funghini, Ph. D. 12, Novella Tedesco, M. Sc. 13, Pierre Romain Le Brun, Charles A. Rupar, Ph. D., F. C. C. M. G. 15, Chitra Prasad, M. D. F. R. C. P., Mar O’Callaghan, Ph. D. 16, John J. Mitchell, F. R. C. P. 17, Olivier Danos, Ph. D. 19, Jean-Brice Marteau, Ph. D. 20, Stefania Galimberti, Ph. D. 21, Maria Grazia Valsecchi, Philippe Veron, Ph. D. 13, Federico Mingozzi, Francesca Fallarino, Giancarlo la Marca, Pharm. Sc. 12, H. Serap Sivri, M. D. 4, and Alberto Auricchio, M. D. 1

المساهمون: BRUNETTI PIERRI, Nicola, 1 2, M. D., Ferla, Rita, h. D. 1., P, Ginocchio, virginia maria, D., M., Rossi, Alessandro, h. D. 2., P, Fecarotta, Simona, h. D. 3., P, Romano, Roberta, D. 2., M., Parenti, Giancarlo, Yildiz, Yilmaz, h. D. 4., P, Zancan, Stefano, Sc. 5., M., Pecorella, Valentina, Sc. 1., M., Dell’Anno, Margherita, Graziano, Mafalda, Alliegro, Marialuisa, Andria, Generoso, Santamaria, Francesca, Brunetti-Pierri, Raffaella, h. D. 6., P, Simonelli, Francesca, D. 6., M., Nigro, Vincenzo, 1 7, M. D., Vargas, Maria, D. 8., M., Servillo, Giuseppe, Borgia, Francesco, D. 9., M., Soscia, Ernesto, 10, M. D., Gargaro, Marco, 11, Ph. D., Funghini, Silvia, 12, Ph. D., Tedesco, Novella, 13, M. Sc., Romain Le Brun, Pierre, Rupar, Charles A., h. D., P, 15, F. C. C. M. G., Prasad, Chitra, D. F. R. C. P., M., O’Callaghan, Mar, 16, Ph. D., Mitchell, John J., 17, F. R. C. P., Danos, Olivier, 19, Ph. D., Marteau, Jean-Brice, 20, Ph. D., Galimberti, Stefania, 21, Ph. D., Grazia Valsecchi, Maria, Veron, Philippe, 13, Ph. D., Mingozzi, Federico, Fallarino, Francesca, la Marca, Giancarlo, 12, Pharm. Sc., Serap Sivri, H., D. 4., M., Alberto Auricchio, And, D. 1., M.

URL الوصول: https://explore.openaire.eu/search/publication?articleId=od______3730::5738f09a90c8a5d20b8239b9aff172c3
https://hdl.handle.net/11588/912900

المؤلفون: Nicolina C. Sorrentino, Vincenzo Cacace, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Francesca Barone, Maria laura Bacci, Yan Huang, Nan Liu, Susan Kalled, Vivian Choi, Alessandro Fraldi

المساهمون: Nicolina C. Sorrentino, Vincenzo Cacace, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Francesca Barone, Maria laura Bacci, Yan Huang, Nan Liu, Susan Kalled, Vivian Choi, Alessandro Fraldi, Sorrentino, Nicolina C., Cacace, Vincenzo, Maffia, Veronica, Strollo, Sandra, Tedesco, Novella, Nusco, Edoardo, Romagnoli, Noemi, Ventrella, Domenico, Barone, Francesca, laura Bacci, Maria, Huang, Yan, Liu, Nan, Kalled, Susan, Choi, Vivian, Fraldi, Alessandro, Nicolina C. Sorrentino, Vincenzo Cacace, Veronica Maffia, Sandra Strollo, Novella Tedesco, Edoardo Nusco, Noemi Romagnoli, Domenico Ventrella, Francesca Barone, Maria laura Bacci, Yan Huang, Nan Liu, Susan Kalled, Vivian Choi, Alessandro Fraldi

مصطلحات موضوعية: Gene Therapy, MPS IIIA, Mice, Intra-CSF injection

وصف الملف: ELETTRONICO

URL الوصول: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::b52b00c9a0fcffbf18aac2f0b0ee7b8e
http://hdl.handle.net/11588/756658

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