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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...
Autores principales: | , , , , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mosby
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6721834/ https://www.ncbi.nlm.nih.gov/pubmed/30926529 http://dx.doi.org/10.1016/j.jaci.2019.03.012 |