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Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

BACKGROUND: Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe and life-threatening bleeding. Lentiviral gene therapy (GT) for WAS has shown promising results in terms of immune reconstitutio...

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Detaylı Bibliyografya
Yayımlandı:J Allergy Clin Immunol
Asıl Yazarlar: Sereni, Lucia, Castiello, Maria Carmina, Di Silvestre, Dario, Della Valle, Patrizia, Brombin, Chiara, Ferrua, Francesca, Cicalese, Maria Pia, Pozzi, Loris, Migliavacca, Maddalena, Bernardo, Maria Ester, Pignata, Claudio, Farah, Roula, Notarangelo, Lucia Dora, Marcus, Nufar, Cattaneo, Lorella, Spinelli, Marco, Giannelli, Stefania, Bosticardo, Marita, van Rossem, Koen, D'Angelo, Armando, Aiuti, Alessandro, Mauri, Pierluigi, Villa, Anna
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mosby 2019
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6721834/
https://ncbi.nlm.nih.gov/pubmed/30926529
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jaci.2019.03.012
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