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Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells

Our previous studies have demonstrated that platelet FVIII (2bF8) gene therapy can improve hemostasis in hemophilia A mice, even in the presence of inhibitory antibodies, but none of our studies has targeted human cells. Here, we evaluated the feasibility for lentivirus (LV)-mediated human platelet...

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書誌詳細
主要な著者: Shi, Qizhen, Kuether, Erin L., Chen, Yingyu, Schroeder, Jocelyn A., Fahs, Scot A., Montgomery, Robert R.
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Hematology 2014
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC3894495/
https://ncbi.nlm.nih.gov/pubmed/24269957
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2013-08-520478
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