Research Topics
| Qizhen ShiSummaryAffiliation: Blood Research Institute Country: USA Publications
| Collaborators
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Detail Information
Publications
Intravascular recovery of VWF and FVIII following intraperitoneal injection and differences from intravenous and subcutaneous injection in miceQ Shi
Department of Pediatrics, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA
Haemophilia 18:639-46. 2012..These results demonstrate that both VWF and FVIII can be efficiently absorbed into the blood circulation following i.p., but not s.c. injection, indicating that i.p. administration could be an alternative route for VWF or FVIII infusion...
Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunityQizhen Shi
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, USA
Blood 112:2713-21. 2008....
Platelets as delivery systems for disease treatmentsQizhen Shi
Blood Research Institute, BloodCenter of Wisconsin, Departments of Pediatrics, Medical College of Wisconsin, Children s Hospital of Wisconsin, Milwaukee, USA
Adv Drug Deliv Rev 62:1196-203. 2010..This paper reviews the genetic therapy for inherited bleeding disorders utilizing platelets as delivery system, with a particular focus on platelet-derived FVIII for hemophilia A treatment...
Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia AQizhen Shi
Blood Research Institute, BloodCenter of Wisconsin, Milwaukee, WI, USA
Blood 116:3049-57. 2010....
Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia AQ Shi
Department of Pediatrics, Medical College of Wisconsin Milwaukee, WI 53226, USA
J Thromb Haemost 5:352-61. 2007....
Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodiesQizhen Shi
Blood Research Institute, BloodCenter of Wisconsin, Department of Pediatrics, Medical College of Wisconsin, and Children s Research Institute, Children s Hospital of Wisconsin, Milwaukee, Wisconsin 53226, USA
J Clin Invest 116:1974-82. 2006..Our results demonstrate that this approach to the targeted expression of FVIII in platelets has the potential to correct hemophilia A, even in the presence of inhibitory immune responses to infused FVIII...
Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B miceGuowei Zhang
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, USA
Blood 116:1235-43. 2010..Our studies suggest that targeting FIX expression to platelets could be a new gene therapy strategy for hemophilia B...
Correction of murine Bernard-Soulier syndrome by lentivirus-mediated gene therapySachiko Kanaji
Blood Research Institute, Blood Center of Wisconsin, Milwaukee, Wisconsin 53226, USA
Mol Ther 20:625-32. 2012..These results demonstrate that lentivirus-mediated gene transfer can provide sustained phenotypic correction of murine BSS, indicating that this approach may be a promising strategy for gene therapy of BSS patients...
Platelet and endothelial expression of clotting factors for the treatment of hemophiliaRobert R Montgomery
Blood Research Institute of BloodCenter of Wisconsin and Medical College of Wisconsin, Milwaukee, WI, USA
Thromb Res 129:S46-8. 2012..These approaches have been studied in murine models but will need further study before this approach can be attempted clinically...
Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIIID A Wilcox
Department of Pediatrics, Medical College of Wisconsin, and Children s Hospital of Wisconsin, Milwaukee, Wisconsin 53226, USA
J Thromb Haemost 1:2477-89. 2003..These results suggest feasibility for the development of a locally inducible secretory pool of FVIII in platelets of patients with hemophilia A...
Alternative strategies for gene therapy of hemophiliaRobert R Montgomery
Blood Research Institute, BloodCenter of Wisconsin, Milwaukee, WI, USA
Hematology Am Soc Hematol Educ Program 2010:197-202. 2010..A safe cure of hemophilia is still the desired goal, but many barriers must still be overcome...
Regulated release of VWF and FVIII and the biologic implicationsS L Haberichter
Department of Pediatrics, Medical College of Wisconsin and Blood Research Institute, Blood Center of Wisconsin, Milwaukee, Wisconsin 53201, USA
Pediatr Blood Cancer 46:547-53. 2006..The data presented indicate that regulated secretion of both proteins occurs only if there is endogenous synthesis of FVIII together with VWF...
Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIXQ Shi
Department of Pediatrics, Medical College of Wisconsin, MACC Fund Research Center MFRC, Milwaukee, WI 53226, USA
J Thromb Haemost 2:1989-97. 2004..This strategy could potentially be utilized for gene therapy of BSS...
Expression of human factor VIII under control of the platelet-specific alphaIIb promoter in megakaryocytic cell line as well as storage together with VWFQ Shi
Department of Pediatrics, Medical College of Wisconsin, MACC Fund Research Center (MFRC, 8701 Watertown Plank Road, Milwaukee 53226-0509, USA
Mol Genet Metab 79:25-33. 2003..The data indicate that the megakaryocytic compartment of hematopoietic cells may represent a potential target of gene therapy for hemophilia A-especially in those patients who have developed inhibitors to plasma FVIII...
Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatmentHelen V Yarovoi
The Children's Hospital of Philadelphia, 1 Civic Center, ARC, Rm 317, Philadelphia, PA 19104, USA
Blood 102:4006-13. 2003..Our studies also suggest that platelet-released factor VIII is at least as potent as an equivalent plasma level and perhaps even more potent in an arterial thrombosis model...
