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In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy

Our previous studies have demonstrated that platelet-targeted FIX gene therapy can introduce sustained platelet-FIX expression in hemophilia B (FIX(null)) mice. In this study, we aimed to enhance platelet-FIX expression in FIX(null) mice with MGMT-mediated in vivo drug selection of transduced cells...

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Bibliografiske detaljer
Udgivet i:J Cell Physiol
Main Authors: Chen, Yingyu, Schroeder, Jocelyn A., Gao, Chunyan, Li, Jing, Hu, Jianda, Shi, Qizhen
Format: Artigo
Sprog:Inglês
Udgivet: 2020
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7722216/
https://ncbi.nlm.nih.gov/pubmed/32510630
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jcp.29861
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