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Correction of Murine Bernard–Soulier Syndrome by Lentivirus-mediated Gene Therapy

Bernard–Soulier syndrome (BSS) is an inherited bleeding disorder caused by a defect in the platelet glycoprotein (GP) Ib-IX-V complex. The main treatment for BSS is platelet transfusion but it is often limited to severe bleeding episodes or surgical interventions due to the risk of alloimmunization....

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Autores principales: Kanaji, Sachiko, Kuether, Erin L, Fahs, Scot A, Schroeder, Jocelyn A, Ware, Jerry, Montgomery, Robert R, Shi, Qizhen
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2012
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3293608/
https://ncbi.nlm.nih.gov/pubmed/22044935
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.231
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