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Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models
Wiskott–Aldrich Syndrome (WAS) is a life-threatening X-linked disease characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancies. Gene therapy could represent a therapeutic option for patients lacking a suitable bone marrow (BM) donor. In this study, we analyzed the long-ter...
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| Main Authors: | , , , , , , , , , , , , , , , , , , , |
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| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
Nature Publishing Group
2009
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| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2835187/ https://ncbi.nlm.nih.gov/pubmed/19259069 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.31 |
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