يعرض 1 - 10 نتائج من 14 نتيجة بحث عن '"Fabiola De Mattia"', وقت الاستعلام: 1.01s تنقيح النتائج
  1. 1
    دورية أكاديمية
    P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up

    المؤلفون: Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti

    المصدر: Genetics in Medicine Open, Vol 2, Iss , Pp 100882- (2024)

    مصطلحات موضوعية: Genetics, QH426-470, Medicine

    وصف الملف: electronic resource

    Relation: http://www.sciencedirect.com/science/article/pii/S2949774424000281; https://doaj.org/toc/2949-7744

    URL الوصول: https://doaj.org/article/754fbeb348e443f3bc199e31def8215a

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  2. 2
    دورية أكاديمية
    Biological and functional characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency

    المؤلفون: Nadia Starc, Daniela Ingo, Antonella Conforti, Valeria Rossella, Luigi Tomao, Angela Pitisci, Fabiola De Mattia, Immacolata Brigida, Mattia Algeri, Mauro Montanari, Giuseppe Palumbo, Pietro Merli, Paolo Rossi, Alessandro Aiuti, Franco Locatelli, Maria Ester Bernardo

    المصدر: Scientific Reports, Vol 7, Iss 1, Pp 1-13 (2017)

    مصطلحات موضوعية: Medicine, Science

    وصف الملف: electronic resource

    Relation: https://doaj.org/toc/2045-2322

    URL الوصول: https://doaj.org/article/6a8d074e533241b8af7e7686f9f524fc

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  3. 3
    Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use

    المؤلفون: Valeria Calbi, Francesca Fumagalli, Vera Gallo, Salvatore Recupero, Fabiola De Mattia, Elena Sophia Fratini, Ambra Corti, Giulia Tarantola, Marina Sarzana, Alessia Ippolito, Stefano Scarparo, Francesca Ciotti, Maddalena Fraschini, Margherita Levi, Mara Sangalli, Francesco Morena, Sabata Martino, Caroline Sevin, Vinodh Narayanan, Etai Adam, Lisa Emrick, Roberto Giugliani, Alessandro Aiuti

    المصدر: Molecular Genetics and Metabolism. 138:107044

    مصطلحات موضوعية: Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::2a354d438c53ad77b6c584626d9c478a
    https://doi.org/10.1016/j.ymgme.2022.107044

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  4. 4
    Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up

    المؤلفون: Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto A. Zambon, Vera Gallo, Salvatore Recupero, Cristina Baldoli, Elena Sophia Fratini, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Andrea Calabria, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Angelica Claros, Arpeat Kaviya, Sean Moro, Jean Brooks, Alan Richardson, Alessandro Aiuti

    المصدر: Molecular Genetics and Metabolism. 138:107108

    مصطلحات موضوعية: Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::fb5de78ef77ef84f4816c6e6c14f6a95
    https://doi.org/10.1016/j.ymgme.2022.107108

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  5. 5
    Blood sulfatides as disease biomarker for metachromatic leukodystrophy: Disease characterization, early diagnosis, and response to treatment

    المؤلفون: Valeria Calbi, Francesca Fumagalli, Elena Sophia Fratini, Andrea Palasciano, Salvatore Recupero, Vera Gallo, Fabiola De Mattia, Ambra Corti, Maria Della Bona, Sabrina Malvagia, Giulia Forni, Giancarlo La Marca, Alessandro Aiuti

    المصدر: Molecular Genetics and Metabolism. 138:107043

    مصطلحات موضوعية: Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::2ceac9574a6e64528a85b16ac40f1d32
    https://doi.org/10.1016/j.ymgme.2022.107043

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  6. 6
    Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients

    المؤلفون: Maddalena Migliavacca, Maria Ester Bernardo, Michela Gabaldo, Elena Fratini, Maria Grazia Natali Sora, Francesco Morena, Jesus Segovia, Giada Farinelli, Alessandra Biffi, Gerald Downey, Clelia Di Serio, Federica Cugnata, Luigi Naldini, Cristina Baldoli, Sabata Martino, Francesca Tucci, Mirko Essing, Francesca Ferrua, Federica Barzaghi, Eugenio Montini, Stefano Scarparo, Andrea Calabria, Maria Pia Cicalese, Mara Sangalli, Paolo Silvani, Maddalena Fraschini, Alberto A. Zambon, Marina Sarzana, Laetitia C. Schwab, Alessandra G. Clerici, Sara Locatelli, Alessandro Aiuti, Salvatore Recupero, Stefano Zancan, Francesca Ciotti, Paola M.V. Rancoita, Fabiola De Mattia, Gigliola Antonioli, Maria Sessa, Massimo Filippi, Fabio Ciceri, Vera Gallo

    المصدر: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics.

    مصطلحات موضوعية: Metachromatic leukodystrophy, Haematopoiesis, business.industry, Genetic enhancement, medicine, Cancer research, Progenitor cell, medicine.disease, business

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::1f3c98d7c741137af7a7d58dd91889ba
    https://doi.org/10.1055/s-0041-1739647

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  7. 7
    Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry

    المؤلفون: Maria L. Beltran-Quintero, Xinying Hong, Alan Finglas, Michael H. Gelb, Omar Sherbini, Giancarlo la Marca, Joanne Kurtzberg, Fabiola De Mattia, Maria L. Escolar, Arun Kumar, Francesca Fumagalli, Roberta Damiano, Adeline Vanderver, Martin Sadilek, Alessandro Aiuti, Maria Luisa Della Bona, Teryn R. Suhr, Jessica Daiker, Amy Waldman, Valeria Calbi, Sarah Woidill, Amber Olsen, Laura Adang, Fan Yi

    المساهمون: Hong, Xinying, Kumar, Arun Babu, Daiker, Jessica, Yi, Fan, Sadilek, Martin, De Mattia, Fabiola, Fumagalli, Francesca, Calbi, Valeria, Damiano, Roberta, Bona, Maria Della, la Marca, Giancarlo, Vanderver, Adeline L, Waldman, Amy T, Adang, Laura, Sherbini, Omar, Woidill, Sarah, Suhr, Teryn, Kurtzberg, Joanne, Beltran-Quintero, Maria L, Escolar, Maria, Aiuti, Alessandro, Gelb, Michael H

    المصدر: Anal Chem

    مصطلحات موضوعية: Arylsulfatase A, Multiple Sulfatase Deficiency Disease, 010402 general chemistry, Tandem mass spectrometry, 01 natural sciences, Article, Analytical Chemistry, Multiple sulfatase deficiency, Tandem Mass Spectrometry, medicine, Leukocytes, Humans, Cerebroside-Sulfatase, chemistry.chemical_classification, Newborn screening, Sulfoglycosphingolipids, biology, Molecular Structure, 010401 analytical chemistry, Leukodystrophy, Metachromatic, medicine.disease, Molecular biology, 0104 chemical sciences, Dried blood spot, Metachromatic leukodystrophy, Enzyme, chemistry, biology.protein, Dried Blood Spot Testing, Antibody, Chromatography, Liquid

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::71f19bd69462c70a8a3b19c8909f9aaf
    https://europepmc.org/articles/PMC7203001/

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  8. 8
    Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy

    المؤلفون: Raisa Jofra Hernandez, Paola M.V. Rancoita, Bernhard Gentner, Ilaria Visigalli, Maryam Omrani, Luca Basso-Ricci, Patrizia Cristofori, Maddalena Migliavacca, Francesca Sanvito, Paola Albertini, Maura De Simone, Serena Scala, Fabiola De Mattia, Luigi Naldini, Nicola Carriglio, Clelia Di Serio, Fabrizio Benedicenti, Francesca Cecere, Rossana Norata, Giada Farinelli, Eugenio Montini, Andrea Calabria, Alessandra Mortellaro, Alessandro Aiuti, Michela Vezzoli

    المساهمون: Jofra Hernandez, R., Calabria, A., Sanvito, F., De Mattia, F., Farinelli, G., Scala, S., Visigalli, I., Carriglio, N., De Simone, M., Vezzoli, M., Cecere, F., Migliavacca, M., Basso-Ricci, L., Omrani, M., Benedicenti, F., Norata, R., Rancoita, P. M. V., Di Serio, C., Albertini, P., Cristofori, P., Naldini, L., Gentner, B., Montini, E., Aiuti, A., Mortellaro, A.

    المصدر: Molecular Therapy

    مصطلحات موضوعية: Time Factors, Genetic enhancement, mouse model, Genetic Vectors, GLP, lentiviral vectors, medicine.disease_cause, Granulomatous Disease, Chronic, Viral vector, 03 medical and health sciences, Mice, 0302 clinical medicine, Chronic granulomatous disease, Drug Discovery, Genetics, medicine, Animals, Humans, Progenitor cell, Molecular Biology, 030304 developmental biology, Pharmacology, 0303 health sciences, NADPH oxidase, biology, business.industry, Lentivirus, Myeloid leukemia, Genetic Therapy, medicine.disease, gene therapy, Good Laboratory Practice, myelodysplastic syndrome, Haematopoiesis, Disease Models, Animal, Treatment Outcome, inflammation, 030220 oncology & carcinogenesis, Hematologic Neoplasms, NADPH Oxidase 2, X-linked chronic granulomatosis disease, biology.protein, Cancer research, Molecular Medicine, Original Article, Carcinogenesis, business

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9ef52c93830efdd78f0cc248deafd234

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  9. 9
    Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200

    المؤلفون: Federica Barzaghi, Francesco Morena, Maddalena Migliavacca, Jesus Segovia, Maria Ester Bernardo, Vera Gallo, Mara Sangalli, Marina Sarzana, Francesca Fumagalli, Laetitia C. Schwab, Maddalena Fraschini, Gerald Downey, Luigi Naldini, Elena Fratini, Francesca Ferrua, Maria Pia Cicalese, Sabata Martino, Francesca Tucci, Sara Locatelli, Alessandro Aiuti, Fabio Ciceri, Valeria Calbi, Francesca Ciotti, Fabiola De Mattia

    المصدر: Molecular Genetics and Metabolism. 132:S21-S22

    مصطلحات موضوعية: business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, medicine.disease, Biochemistry, Cryopreservation, Metachromatic leukodystrophy, Haematopoiesis, Endocrinology, Genetics, Cancer research, Medicine, Stem cell, business, Molecular Biology

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::c6610d8814bcd31dd8a0d9788faa0bef
    https://doi.org/10.1016/j.ymgme.2020.12.031

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  10. 10
    Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

    المؤلفون: Vera Gallo, Maria Ester Bernardo, Clelia De Serio, Paula M.V. Rancoita, Gerald Downey, Stefano Zancan, Serena Acquati, Francesca Tucci, John Sharpe, Francesca Fumagalli, Cristina Baldoli, Laetitia C. Schwab, Sabata Martino, Valeria Calbi, Luigi Naldini, Alessandro Aiuti, Fabiola De Mattia, Maria Grazia Natali Sora, Maria Sessa, Alessandra Biffi, Massimo Filippi, Fabio Ciceri, Alberto A. Zambon, Michela Gabaldo, Eugenio Montini

    المصدر: Molecular Genetics and Metabolism. 129:S59

    مصطلحات موضوعية: business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, medicine.disease, Biochemistry, Metachromatic leukodystrophy, Haematopoiesis, Endocrinology, Genetics, Cancer research, Medicine, Progenitor cell, business, Molecular Biology

    URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1084bfb180ff4ade95b475d019ce2e84
    https://doi.org/10.1016/j.ymgme.2019.11.135

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