Platelet Gene Therapy by Lentiviral Gene Delivery to Hematopoietic Stem Cells Restores Hemostasis and Induces Humoral Immune Tolerance in FIX(null) Mice

Here, we developed a clinically translatable platelet gene therapy approach for hemophilia B. Platelet-targeted FIX (2bF9) expression was introduced by transplantation of hematopoietic stem cells (HSCs) transduced with 2bF9 lentivirus (LV). Sustained therapeutic levels of platelet-FIX expression wer...

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Autors principals: Chen, Yingyu, Schroeder, Jocelyn A, Kuether, Erin L, Zhang, Guowei, Shi, Qizhen
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2014
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3978792/
https://ncbi.nlm.nih.gov/pubmed/24042561
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.197
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