المؤلفون: Francesco Morena, Serena Acquati, Chiara Argentati, Sabata Martino, Stephen DeWall, Fiona Kelly, Stefano Zancan, Alessandra Biffi, Valeria Calbi, Alessandro Aiuti, Francesca Fumagalli

المساهمون: Morena, Francesco, Argentati, Chiara, Acquati, Serena, Dewall, Stephen, Kelly, Fiona, Calbi, Valeria, Fumagalli, Francesca, Zancan, Stefano, Biffi, Alessandra, Aiuti, Alessandro, Martino, Sabata

المصدر: The journal of applied laboratory medicine. 6(2)

مصطلحات موضوعية: 0301 basic medicine, Adult, Arylsulfatase A, medicine.medical_specialty, lysosomal enzymes, Lysosomal storage disorders, Gastroenterology, 03 medical and health sciences, 0302 clinical medicine, Cerebrospinal fluid, Internal medicine, Medicine, Humans, Child, Cerebroside-Sulfatase, Total protein, business.industry, Leukodystrophy, biochemical assays, General Medicine, Genetic Therapy, Leukodystrophy, Metachromatic, medicine.disease, Reference intervals, Clinical Practice, Metachromatic leukodystrophy, 030104 developmental biology, business, 030217 neurology & neurosurgery

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::567c9008524ec75e098f6b56978e3e7b
https://pubmed.ncbi.nlm.nih.gov/32910180

المؤلفون: Maria Grazia Natali Sora, Vanessa Attanasio, Facchini Marcella, Robert Chiesa, Marina Sarzana, Francesca Ferrua, Giovanna Chidini, Francesco Morena, Gian Luigi Ardissino, Sabata Martino, Federica Barzaghi, Gigliola Antonioli, Serena Acquati, Paolo Silvani, Fabio Ciceri, Daniela Redaelli, Maria Ester Bernardo, Matteo Zoccolillo, Valeria Calbi, Francesca Ciotti, Cristina Baldoli, Maria Pia Cicalese, Maddalena Migliavacca, Giulia Consiglieri, Rachele Penati, Francesca Fumagalli, Luigi Naldini, Alessandro Aiuti, Andrea Assanelli, Ruchi Arora

المساهمون: Calbi, Valeria, Fumagalli, Francesca, Consiglieri, Giulia, Penati, Rachele, Acquati, Serena, Redaelli, Daniela, Attanasio, Vanessa, Marcella, Facchini, Cicalese, Maria Pia, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Assanelli, Andrea, Silvani, Paolo, Zoccolillo, Matteo, Chidini, Giovanna, Chiesa, Robert, Arora, Ruchi, Ciotti, Francesca, Sarzana, Marina, Antonioli, Gigliola, Baldoli, Cristina, Morena, Francesco, Martino, Sabata, Ardissino, Gian Luigi, Sora, Maria Grazia Natali, Naldini, Luigi, Ciceri, Fabio, Aiuti, Alessandro, Bernardo, Maria Ester

المصدر: Bone Marrow Transplantation

مصطلحات موضوعية: 0301 basic medicine, Male, medicine.medical_specialty, Transplantation Conditioning, medicine.medical_treatment, Genetic enhancement, Twins, Hematopoietic stem cell transplantation, Hematology, Transplantation, ARSA, Gene Therapy, Defibrotide, 03 medical and health sciences, 0302 clinical medicine, Polydeoxyribonucleotides, Internal medicine, Correspondence, medicine, Humans, Transplantation, business.industry, Hematopoietic Stem Cell Transplantation, Hematopoietic stem cell, Genetic Therapy, Leukodystrophy, Metachromatic, Gene Therapy, medicine.disease, Metachromatic leukodystrophy, 030104 developmental biology, medicine.anatomical_structure, 030220 oncology & carcinogenesis, Immunology, Platelet aggregation inhibitor, ARSA, business, Platelet Aggregation Inhibitors, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1644782068f8ef760b1e0820c59421b1
https://hdl.handle.net/11391/1423195

المؤلفون: Alessandro Aiuti, Rachele Penati, Maria Ester Bernardo, Francesca Fumagalli, Valeria Calbi

المساهمون: Penati, Rachele, Fumagalli, Francesca, Calbi, Valeria, Bernardo, Maria Ester, Aiuti, Alessandro

المصدر: Journal of Inherited Metabolic Disease

مصطلحات موضوعية: 0301 basic medicine, Genetic enhancement, Mucopolysaccharidosis I, Genetic Vectors, Lysosomal storage disease, Biology, Viral vector, Ssiem 2016, 03 medical and health sciences, Mucopolysaccharidosis type I, Gene therapy, Genetic, Genetics, medicine, Animals, Humans, Enzyme Replacement Therapy, Genetics (clinical), Transplantation, Gene Transfer Techniques, Hematopoietic Stem Cell Transplantation, Enzyme replacement therapy, Genetic Therapy, Leukodystrophy, Metachromatic, medicine.disease, Metachromatic leukodystrophy, Lysosomal Storage Diseases, Haematopoiesis, 030104 developmental biology, Treatment Outcome, Immunology, Viruses, Cancer research, Stem cell

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8b9c5f4d44b9aa89a98839236b3ceecf
https://pubmed.ncbi.nlm.nih.gov/28560469

المؤلفون: Chiara Dallatomasina, Elisa Riboni, Carlo Alberto Forcellini, Lucia Dora Notarangelo, Raisa Jofra Hernandez, Pietro Luigi Poliani, Letizia Leocani, Antonella Tabucchi, Fabio Minicucci, Giancarlo la Marca, Federica Barzaghi, Nicola Carriglio, Francesca Fumagalli, Alessandro Aiuti, Letterio S. Politi, Stefania Medaglini, Sabrina Canale, Giancarlo Comi, Chiara Azzari, Miriam Casiraghi, Veronica Bianchi, Patrizia D'Adamo, Francesca Ferrua, Maria Sessa, Filippo Carlucci, Aisha V. Sauer, Manuela Cominelli, Cristina Baldoli

المساهمون: Sauer, Aisha V., Hernandez, Raisa Jofra, Fumagalli, Francesca, Bianchi, Veronica, Poliani, Pietro L., Dallatomasina, Chiara, Riboni, Elisa, Politi, Letterio S., Tabucchi, Antonella, Carlucci, Filippo, Casiraghi, Miriam, Carriglio, Nicola, Cominelli, Manuela, Forcellini, Carlo Alberto, Barzaghi, Federica, Ferrua, Francesca, Minicucci, Fabio, Medaglini, Stefania, Leocani, ANNUNZIATA MARIA LETIZIA, La Marca, Giancarlo, Notarangelo, Lucia D., Azzari, Chiara, Comi, Giancarlo, Baldoli, Cristina, Canale, Sabrina, Sessa, Maria, D'Adamo, Patrizia, Aiuti, Alessandro

المصدر: Scientific Reports

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, Adenosine, Adenosine Deaminase, Genetic enhancement, Article, Mice, 03 medical and health sciences, 0302 clinical medicine, Adenosine deaminase, immune system diseases, Multidisciplinary, ADA, Internal medicine, medicine, Animals, Humans, Behavior, Severe combined immunodeficiency, Multidisciplinary, Behavior, Animal, biology, business.industry, Brain, hemic and immune systems, Enzyme replacement therapy, medicine.disease, Adenosine receptor, 3. Good health, Adenosine deaminase deficiency, Transplantation, 030104 developmental biology, Endocrinology, biology.protein, Nervous System Diseases, business, 030217 neurology & neurosurgery, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d88ee227fe397b8492c5d40e0259e3a6
https://doi.org/10.1038/srep40136

المؤلفون: Maria Grazia Natali Sora, Sabrina Canale, Clelia Di Serio, Sabata Martino, Andrea Assanelli, Angelo Quattrini, Luigi Naldini, Fabrizio Benedicenti, Alessandra Biffi, Francesco Morena, Francesca Fumagalli, Ubaldo Del Carro, Maria Grazia Roncarolo, Ignazio Diego Lopez, Paolo Silvani, Daniela Redaelli, Laura Lorioli, Eugenio Montini, Paola M.V. Rancoita, Andrea Calabria, Michela Gabaldo, Cristina Baldoli, Serena Acquati, Gigliola Antonioli, Maria Sessa, Rossana Fiori, Alessandro Aiuti, Maria Pia Cicalese, Fabio Ciceri

المساهمون: Sessa, Mario, Lorioli, Laura, Fumagalli, Francesca, Acquati, Serena, Redaelli, Daniela, Baldoli, Cristina, Canale, Sabrina, Lopez, Ignazio D., Morena, Francesco, Calabria, Andrea, Fiori, Rossano, Silvani, Paolo, Rancoita, Paola Maria Vittoria, Gabaldo, Michela, Benedicenti, Fabrizio, Antonioli, Gigliola, Assanelli, Andrea, Cicalese, Maria Pia, del Carro, Ubaldo, Sora, Maria Grazia Natali, Martino, Sabato, Quattrini, Angelo, Montini, Eugenio, Di Serio, Clelia, Ciceri, Fabio, Roncarolo, Maria Grazia, Aiuti, Alessandro, Naldini, Luigi, Biffi, Alessandra

مصطلحات موضوعية: 0301 basic medicine, Male, medicine.medical_specialty, Arylsulfatase A, Adolescent, medicine.medical_treatment, Hematopoietic stem cell transplantation, Neutropenia, lysosomal storage disorders, 03 medical and health sciences, ARSA activity in CSF, Internal medicine, medicine, Humans, Age of Onset, Child, Preschool, Cytopenia, business.industry, Medicine (all), Leukodystrophy, Lentivirus, Hematopoietic Stem Cell Transplantation, Infant, biochemical assays, General Medicine, Leukodystrophy, Metachromatic, Genetic Therapy, Metachromatic, medicine.disease, gene therapy, Child, Preschool, Female, Follow-Up Studies, Italy, Treatment Outcome, Surgery, Clinical trial, Metachromatic leukodystrophy, 030104 developmental biology, gene therapy, lysosomal storage disorders, Age of onset, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d6919efb04ca92443e1d89c4bee4e4b8