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
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	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
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ISSN:	0268-3369 1476-5365
DOI:	10.1038/s41409-017-0085-1