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10.1016/j.omtm.2018.01.011 http://scihub22266oqcxt.onion/10.1016/j.omtm.2018.01.011 C5842292!5842292!29520365     free     free     free       Mol+Ther+Methods+Clin+Dev 2018 ; 9 (ä): 100-8 Nephropedia Template TP {{tp|p=29520365|t=2018. Platelet-Targeted Gene Therapy for Hemophilia |pdf=|usr=}}{{29520365}} gab.com Text Platelet-Targeted Gene Therapy for Hemophilia JO: Mol Ther Methods Clin Dev http://www.kidney.de/pget.php?&tw=1&pmid=29520365 #FOAvip 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 promoter results in FVIII storage together with its carrier protein von Willebrand factor (VWF) in ?-granules and the phenotypic correction of hemophilia A. Importantly, the storage and sequestration of FVIII in platelets appears to effectively restore hemostasis even in the presence of functional-blocking inhibitory antibodies. This review summarizes studies on platelet-specific gene therapy of hemophilia A as well as hemophilia B. Twit Text FOAVip Platelet-Targeted Gene Therapy for Hemophilia ????Mol Ther Methods Clin Dev http://www.kidney.de/pget.php?&tw=1&pmid=29520365 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=29520365 #FOAvip English Wikipedia <ref>{{Cite pmid|29520365}}</ref> Platelet-Targeted Gene Therapy for Hemophilia #MMPMID29520365 Shi Q Mol Ther Methods Clin Dev 2018[Jun]; 9 (ä): 100-8 PMID29520365show ga 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 promoter results in FVIII storage together with its carrier protein von Willebrand factor (VWF) in ?-granules and the phenotypic correction of hemophilia A. Importantly, the storage and sequestration of FVIII in platelets appears to effectively restore hemostasis even in the presence of functional-blocking inhibitory antibodies. This review summarizes studies on platelet-specific gene therapy of hemophilia A as well as hemophilia B. äPlatelet-Targeted Gene Therapy for Hemophilia (epmc).pdf DeepDyve Pubget Overpricing