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Platelet-Targeted Gene Therapy for Hemophilia

7 Feb 2018

Abstract excerpt

Gene therapy is an attractive approach for disease treatment. Since platelets are abundant cells circulating in blood with the distinctive abilities of storage and delivery and fundamental roles in hemostasis and immunity, they could be a unique target for gene therapy of diseases. Recent studies have demonstrated that ectopic expression of factor VIII (FVIII) in platelets under control of the platelet-specific...

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PMID: 29520365
DOI: 10.1016/j.omtm.2018.01.011

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