Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2018-07, Vol.53 (7), p.913-917 |
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| container_title | Bone marrow transplantation (Basingstoke) |
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| creator | 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 |
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| doi_str_mv | 10.1038/s41409-017-0085-1 |
| format | Article |
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| identifier | ISSN: 0268-3369 |
| ispartof | Bone marrow transplantation (Basingstoke), 2018-07, Vol.53 (7), p.913-917 |
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| source | MEDLINE; Springer Nature - Complete Springer Journals; Nature; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals |
| subjects | 38 45/43 692/499 692/700/1720 Cardiovascular diseases Cell Biology Correspondence Endothelium Gene therapy Genetic Therapy - methods Hematology Hematopoietic stem cell transplantation Hematopoietic Stem Cell Transplantation - methods Hematopoietic stem cells Humans Injury prevention Internal Medicine Leukodystrophy Leukodystrophy, Metachromatic - drug therapy Leukodystrophy, Metachromatic - pathology Leukodystrophy, Metachromatic - therapy Male Medicine Medicine & Public Health Platelet Aggregation Inhibitors - pharmacology Platelet Aggregation Inhibitors - therapeutic use Polydeoxyribonucleotides - pharmacology Polydeoxyribonucleotides - therapeutic use Public Health Sphingolipidoses Stem cell transplantation Stem Cells Transplantation Conditioning - methods Twins |
| title | Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy |
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