Versiti - Qizhen Shi, MD, PhD | Versiti Blood Research Institute

Research Interests

Gene Therapy of Hemophilia A

Hemophilia a is a recessive, X-linked bleeding disorder resulting from factor VIII (FVIII) deficiency. Although protein replacement therapy is effective for hemophilia A treatment, up to 35% of patients may develop inhibitory antibodies (referred to as “inhibitors”) that neutralize FVIII activity. Introducing FVIII expression via genetic therapy is an attractive alternative treatment for hemophilia A patients. However, the potential to develop inhibitors to the transgene protein remains a significant concern.

To apply this gene therapy to a clinically translatable protocol, we use lentiviral gene delivery to hematopoietic stem cells (HSCs) to introduce FVIII expression in platelets. We found that 2bF8 lentiviral gene delivery to HSCs can not only restore hemostasis, but also induce antigen-specific immune tolerance in hemophilia A mice. Our current work aims to 1) further optimize this approach; 2) dissect the potential underlying mechanisms by which immune tolerance is induced after platelet gene therapy; and 3) translate findings made in laboratory animals to human patients.

Gene Therapy of Hemophilia B

Hemophilia B is a genetic bleeding disorder that results from factor IX (FIX) deficiency. Although the incidence of anti-FIX inhibitor development is lower (5%) in hemophilia B patients after protein replacement therapy, anaphylaxis is a daunting problem in inhibitor patients, which limits the use of FIX infusion and increases the risk of morbidity and mortality. Our studies show that FIX can be ectopically expressed and stored in platelet a-granules when it is driven under the same aIIb promoter that we use for platelet-FVIII expression. Platelet-derived FIX, which is fully g-carboxylated, can rescue bleeding diathesis in hemophilia B mice, but the clinical efficacy is limited in the presence of anti-FIX inhibitors.

Although platelet-FIX does not maintain clinical efficacy in the face of inhibitors, targeting FIX expression to platelets is still an attractive potential strategy for gene therapy of hemophilia B. Indeed, our studies demonstrate that 2bF9 gene delivery to HSCs not only restores hemostasis, but also induces FIX-specific immune tolerance in hemophilia B mice. Our current work aims to optimize the FIX expression and develop a protocol for gene therapy of hemophilia B with pre-existing immunity.

FVIII Immune Responses in Hemophilia A

The development of inhibitors against FVIII is not only a significant complication in protein replacement therapy, but also a hurdle in gene therapy of hemophilia A. Understanding how FVIII immune responses occur may open a new therapeutic approach to limiting immune responses. Currently, we are dissecting the functional properties of recently identified subsets of immune cells, including T follicular help (Tfh) cells and T follicular help regulatory (Tfr) cells in FVIII immune responses. This will help us better understand the cellular and molecular mechanisms of FVIII inhibitor development and assist us in identifying potential novel targets for therapeutic intervention to prevent or reverse FVIII inhibitor development.

In addition, we want to know how VWF impacts FVIII immune responses in hemophilia A. Our studies demonstrate that VWF is essential for maintaining platelet-FVIII efficacy in hemophilia A with inhibitors. VWF reduces inhibitor activation of FVIII, exerting a protective effect both in vitro and in vivo. VWF/FVIII association, plus the apparent ability of VWF to delay the time-dependent inactivation of FVIII by inhibitors, provides mechanisms by which platelet-derived FVIII maintains function even in the presence of inhibitors when FVIII is targeted to platelets for gene therapy of hemophilia A with inhibitors. Our current work aims to investigate the roles of VWF in FVIII immunity in hemophilia A.

Lab Team

Saurabh Kumar, PhD

Postdoctoral Fellow

Email: SKumar@Versiti.org

Mikayla Couch

Animal Technician

Email: mettmayer@Versiti.org

Jocelyn Schroeder, PhD

Research Associate

Email: JSchroeder@Versiti.org

He Yang, MS

Research Technologist

Email: hyang@versiti.org

Hongyin Yu, MS

IDP Graduate Student

Email: HYu@Versiti.org

Grant Support

R01 HL102035, “Platelet-derived FVIII gene therapy of hemophilia A”, Role: PI (2010-2028).
R01 HL142791, “A phase I clinical trial testing Feasibility of hematopoietic stem cell gene therapy using platelet FVIII to safely improve hemostasis for severe hemophilia A with inhibitory antibodies to FVIII,” Role: Co-I (2019-2027)
R01 HL166382, “Understanding and Controlling the Contribution of Fibrinolysis to Bleeding Using a Long-Acting Antifibrinolytic RNA Therapy”, Role: Co-I (2023-2027)
MACC FUND, “FVIII immune responses in hemophilia A”, Role: PI.
Joan Cox Gill CCBD/MSI Clinical & Translational Pilot Award, “High-plex cytokine and humoral profiling to define immune signatures associated with inhibitor development and immune tolerance induction in hemophilia A”, Role: PI (2025-2026)

Selected Publications

Yu, H., Schroeder, J. A., Matterson, J. G., Gao, C., & Shi, Q. (2026). The impact of anti-platelet antagonists on the hemostatic efficacy of platelet-targeted FVIII gene therapy in hemophilia A mice. Blood Advances, 10(6), 1883–1896. https://doi.org/10.1182/bloodadvances.2025017921
Chen, Y., Xue, F., Kumar, S., Schroeder, J. A., Jing, W., Ni, H., & Shi, Q. (2026). FVIII-containing platelets modulate immune responses and attenuate inhibitor development in hemophilia A mice. Haematologica. https://doi.org/10.3324/haematol.2025.289331
Ferraresso, F., Skaer, C. W., Wei, Z., Paul, M., Hur, W. S., Yu, H., Seadler, M., Chen, T. H. Y., Dai, W., Lapointe, C., Ketelboeter, L. M., Lund, H., Rodriguez, G. G., Juang, L. J., Strilchuk, A. W., Zhang, Y., Cullis, P. R., Dyer, M. R., Gerras, A. L., ... Kastrup, C. J. (2026). Age-associated increases in PAI-1 silenced with siRNA-lipid nanoparticles reduces thrombosis and prolongs lifespan. Blood. https://doi.org/10.1182/blood.2025029834
Shi, Q. (2026). A Padua moment for factor VIII gene therapy. Blood, 147(4), 322–323. https://doi.org/10.1182/blood.2025032121
Shi, Q., Mattson, J. G., Morateck, P. A., Christopherson, P. A., Fahs, S. A., Rapten, J., Schulte, M. L., Weiler, H., Zhu, J., Haberichter, S. L., Flood, V. H., & Montgomery, R. R. (2025). The pivotal role of VWF binding to platelet integrin αIIbβ3 in stabilizing the formation of a platelet plug at sites of vascular injury. Haematologica, 110(10), 2343–2355. https://doi.org/10.3324/haematol.2025.287685
Jing, W., Schroeder, J. A., Kumar, S., Chen, J., Cai, Y., Malec, L. M., Dent, A. L., Cui, W., & Shi, Q. (2025). The T follicular helper/T follicular helper regulatory pathway in FVIII immune responses in mice. Blood, 146(8), 998–1010. https://doi.org/10.1182/blood.2025029470
Kaminski, T. W., Zhang, H., Katoch, O., Shi, Q., Kato, G. J., Sundd, P., & Pradhan-Sundd, T. (2025). Small molecule inhibitor screen to identify mechanisms controlling selective clearance of sickle hemoglobin by liver endothelial cells. Blood Vessels, Thrombosis & Hemostasis, 2(2), 100045. https://doi.org/10.1016/j.bvth.2025.100045
Eapen, M., Malec, L. M., Armant, M. A., Johnson, B. D., Shi, Q., Xu, H., Du, L. M., Jerkins, J. H., Duffy, L. J., Bushman, F. D., Lee, C., Petrichenko, A., Hematti, P., Brazauskas, R., Jobe, S. M., Hari, P. N., & Wilcox, D. A. (2025). Platelet-targeted gene therapy for hemophilia A with inhibitor history. New England Journal of Medicine, 392(4), 412–414. https://doi.org/10.1056/NEJMc2415164
Chen, Z., Zheng, Q., Wang, Y., An, X., Yirga, S. K., Lin, D., Shi, Q., Huang, M., & Chen, Y. (2024). CXCL13/CXCR5 axis facilitates TFH expansion and correlates with disease severity in adults with immune thrombocytopenia. Thrombosis Research, 244, 109196. https://doi.org/10.1016/j.thromres.2024.109196
Zheng, Q., Lin, K., Zhang, N., Shi, Q., Wu, Y., & Chen, Y. (2024). Anti-mCD20 in combination with α-mCXCL13 monoclonal antibody inhibits anti-FVIII antibody development in hemophilia A mice. International Immunopharmacology, 139, 112735. https://doi.org/10.1016/j.intimp.2024.112735
Chen, Y., Jing, L., Schroeder, J. A., Jing, W., & Shi, Q. (2024). Clinical translatable preconditioning for platelet gene therapy in murine hemophilia A with inhibitors. Journal of Thrombosis and Haemostasis, 22(11), 3035–3047. https://doi.org/10.1016/j.jtha.2024.07.023
Luo, L., An, X., Wang, Y., Zheng, Q., Shi, Q., & Chen, Y. (2024). Correlation of chemokine CXCL13 with anti-FVIII inhibitor development in hemophilia A patients and murine models. International Immunopharmacology, 143(Pt 2), 113472. https://doi.org/10.1016/j.intimp.2024.113472
Strilchuk, A. W., Hur, W. S., Batty, P., Sang, Y., Abrahams, S. R., Yong, A. S. M., Leung, J., Silva, L. M., Schroeder, J. A., Nesbitt, K., de Laat, B., Moutsopoulos, N. M., Bugge, T. H., Shi, Q., Cullis, P. R., Merricks, E. P., Wolberg, A. S., Flick, M. J., Lillicrap, D., ... Kastrup, C. J. (2024). Lipid nanoparticles and RNAi targeting plasminogen provide lasting inhibition of fibrinolysis in mouse and dog models of hemophilia A. Science Translational Medicine, 16(735), eadh0027. https://doi.org/10.1126/scitranslmed.adh0027
Kumar, S., Schroeder, J. A., & Shi, Q. (2024). Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond. Journal of Thrombosis and Haemostasis, 22(1), 23–34. https://doi.org/10.1016/j.jtha.2023.07.025
Biswas, R., Boyd, E., Steenackers, A., Schulte, M. L., Eaton, N., Reusswig, F., Yu, H., Shi, Q., Plomann, M., Hoffmeister, K. M., & Falet, H. (2023). Mice lacking PACSIN2 display severe thrombus formation defects due to hyperactive platelet integrin β1. Journal of Thrombosis and Haemostasis, 21(12), 3619–3632. https://doi.org/10.1016/j.jtha.2023.08.026
Li, J., Karakas, D., Xue, F., Chen, Y., Zhu, G., Yucel, Y. H., MacParland, S. A., Zhang, H., Semple, J. W., Freedman, J., Shi, Q., & Ni, H. (2023). Desialylated platelet clearance in the liver is a novel mechanism of systemic immunosuppression. SJP Research, 6, 0236. https://doi.org/10.34133/research.0236
Jing, W., Baumgartner, C. K., Xue, F., Schroeder, J. A., & Shi, Q. (2023). Pre-existing anti-FVIII immunity alters therapeutic platelet-targeted FVIII engraftment in the system preconditioned with busulfan alone through cytotoxic CD8 T cells. Journal of Thrombosis and Haemostasis, 21(3), 488–498. https://doi.org/10.1016/j.jtha.2022.10.006
Luo, L., Zheng, Q., Chen, Z., Huang, M., Fu, L., Hu, J., Shi, Q., & Chen, Y. (2022). Hemophilia A patients with inhibitors: Mechanistic insights and novel therapeutic implications. Frontiers in Immunology, 13, 1019275. https://doi.org/10.3389/fimmu.2022.1019275
Cai, Y., Schroeder, J. A., Jing, W., Yu, H., Gurski, C., Williams, C. B., Wang, S., Dittel, B. N., & Shi, Q. (2022). Targeting transmembrane-domain-less MOG expression to platelets prevents disease development in experimental autoimmune encephalomyelitis. Frontiers in Immunology, 13, 1029356. https://doi.org/10.3389/fimmu.2022.1029356
Shi, Q., & Weiler, H. (2022). Blocking hemophilic arthropathy. Blood, 139(18), 2735. https://doi.org/10.1182/blood.2022015984
Chen, Y., Luo, L., Zheng, Y., Zheng, Q., Zhang, N., Gan, D., Yirga, S. K., Lin, Z., Shi, Q., Fu, L., Hu, J., & Chen, Y. (2022). Association of platelet desialylation and circulating follicular helper T cells in patients with thrombocytopenia. Frontiers in Immunology, 13, 810620. https://doi.org/10.3389/fimmu.2022.810620
Shi, Q., Fahs, S. A., Matterson, J. G., Yu, H., Perry, C., Morateck, P. A., Schroeder, J. A., Rapten, J., Weiler, H., & Montgomery, R. R. (2022). A novel mouse model of type 2N VWD recapitulates human VWD and suggests dysfunctional VWF may inhibit alternative hemostatic pathways. Blood Advances, 6(9), 2778–2790. https://doi.org/10.1182/bloodadvances.2021006481
Schroeder, J. A., Kuether, E. A., Chen, J., Jing, W., Weiler, H., Wilcox, D. A., Montgomery, R. R., & Shi, Q. (2021). Thromboelastometry assessment of hemostatic properties in various murine models with coagulopathy and the effect of factor VIII therapies. Journal of Thrombosis and Haemostasis, 19(10), 2417–2427. https://doi.org/10.1111/jth.15456
Schroeder, J. A., Chen, J., Chen, Y., Cai, Y., Yu, H., Mattson, J. G., Monahan, P. E., & Shi, Q. (2021). Platelet-targeted hyperfunctional factor IX gene therapy for hemophilia B mice even with pre-existing anti-FIX immunity. Blood Advances, 5(5), 1224–1238. https://doi.org/10.1182/bloodadvances.2020004071
Li, J., Chen, J., Schroeder, J. A., Hu, J., Williams, C. B., & Shi, Q. (2021). Platelet gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific T cells. Molecular Therapy: Nucleic Acids, 23, 719–730. https://doi.org/10.1016/j.omtn.2020.12.026
Chen, Y., Schroeder, J. A., Li, J., Hu, J., & Shi, Q. (2021). In vivo enriching genetically engineered platelets for gene therapy of hemophilia B mice. Journal of Cellular Physiology, 236(1), 354–365. https://doi.org/10.1002/jcp.29861
Cai, Y., & Shi, Q. (2020). Platelet-targeted FVIII gene therapy restores hemostasis and induces immune tolerance for hemophilia A. Frontiers in Immunology, 11, 964. https://doi.org/10.3389/fimmu.2020.00964
Shi, Q., Carman, C. V., Chen, Y., Sage, P. T., Xue, F., Liang, X. M., & Gilbert, G. E. (2020). Unexpected enhancement of FVIII immunogenicity by endothelial expression in lentivirus-transduced and transgenic mice. Blood Advances, 4(10), 2272–2285. https://doi.org/10.1182/bloodadvances.2020001468
Shi, Q., Mattson, J. G., Fahs, S. A., Geurts, A. M., Weiler, H., & Montgomery, R. R. (2020). The severe spontaneous bleeding phenotype in a novel hemophilia A rat model is rescued by platelet FVIII expression. Blood Advances, 4(1), 55–65. https://doi.org/10.1182/bloodadvances.2019000944
Garcia, J., Flood, V. H., Haberichter, S. L., Fahs, S. A., Mattson, J. G., Geurts, A. M., Zogg, M., Weiler, H., Shi, Q., & Montgomery, R. R. (2020). A rat model of severe VWD by elimination of the VWF gene using CRISPR/Cas9. Research and Practice in Thrombosis and Haemostasis, 4(1), 64–71. https://doi.org/10.1002/rth2.12280
Wang, D., Zhang, G., Gu, J., Shao, X., Dai, Y., Li, J., Pan, X., Yao, S., Jin, Y., Huang, J., Shi, Q., Chen, Z., & Chen, S. (2020). Platelet-targeted gene therapy of murine hemophilia A using HSPCs derived from genome edited iPSCs. Haematologica, 105(4), e175–e179. https://doi.org/10.3324/haematol.2019.219089
Jing, W., Chen, J., Cai, Y., Chen, Y., Schroeder, J. A., Cui, W., Johnson, B. D., & Shi, Q. (2019). Induction of activated T follicular helper cells is critical for anti-FVIII inhibitor development in hemophilia A mice. Blood Advances, 3(20), 3099–3110. https://doi.org/10.1182/bloodadvances.2019000650
Gao, C., Schroeder, J. A., Xue, F., Jing, W., Cai, Y., Subramaniam, S., Rao, S., Weiler, H., Czechowicz, A., & Shi, Q. (2019). Immunotoxin-mediated non-genotoxic preconditioning for platelet gene therapy of hemophilia A mice. Blood Advances, 3(18), 2700–2711. https://doi.org/10.1182/bloodadvances.2019000516
Chen, J., Schroeder, J. A., Luo, X., Montgomery, R. R., & Shi, Q. (2019). The impact of GPIbα on platelet-targeted FVIII gene therapy in hemophilia A with pre-existing anti-FVIII immunity. Journal of Thrombosis and Haemostasis, 17(3), 449–459. https://doi.org/10.1111/jth.14379
Luo, X., Chen, J., Schroeder, J. A., Baumgartner, K. C., Subramaniam, M., Hu, J., Williams, C. B., & Shi, Q. (2018). Platelet gene therapy provokes targeted peripheral tolerance by clonal deletion and induction of antigen-specific regulatory T cells. Frontiers in Immunology, 9, 1950. https://doi.org/10.3389/fimmu.2018.01950
Shi, Q. (2018). Platelet-targeted gene therapy for hemophilia. Molecular Therapy: Methods & Clinical Development, 9(6), 100–108. https://doi.org/10.1016/j.omtm.2018.02.007
Chen, Y., Luo, X., Chen, J., Schroeder, J. A., Baumgartner, K. C., Hu, J., & Shi, Q. (2017). Immune tolerance developed in platelet-targeted FVIII gene therapy is CD4 T cell-mediated. Journal of Thrombosis and Haemostasis, 15(10), 1994–2004. https://doi.org/10.1111/jth.13780
Chen, J., Schroeder, J. A., Luo, X., & Shi, Q. (2017). The impact of von Willebrand factor on factor VIII memory immune responses. Blood Advances, 1(19), 1565–1574. https://doi.org/10.1182/bloodadvances.2017007096
Baumgartner, K. C., Mattson, J. G., Weiler, H., Shi, Q., & Montgomery, R. R. (2017). Targeting FVIII expression to platelets for hemophilia A gene therapy does not induce an apparent thrombotic risk in mice. Journal of Thrombosis and Haemostasis, 15(1), 98–109. https://doi.org/10.1111/jth.13436
Haribhai, D., Luo, X., Chen, J., Jia, S., Shi, L., Schroeder, J. A., Hessner, M. J., Aster, D., Hu, J., Williams, C. B., & Shi, Q. (2016). TGFβ1 along with other platelet contents augments Treg cells to suppress anti-FVIII immune responses in hemophilia A mice. Blood Advances, 1(2), 139–151. https://doi.org/10.1182/bloodadvances.2016000422
Chen, Y., Schroeder, J. A., Chen, J., Luo, X., Baumgartner, K. C., Montgomery, R. R., Hu, J., & Shi, Q. (2016). The immunogenicity of platelets containing FVIII in murine hemophilia A with or without pre-existing anti-FVIII immunity. Blood, 127(10), 1346–1354. https://doi.org/10.1182/blood-2015-07-659656
Baumgartner, K. C., Kuether, E. L., Zhang, G., Weiler, H., Shi, Q., & Montgomery, R. R. (2015). Native whole blood thrombin generation assay evaluates therapeutic efficacy of plasma and platelet-derived FVIII. Journal of Thrombosis and Haemostasis, 13(12), 2210–2219. https://doi.org/10.1111/jth.13143
Shi, Q., Schroeder, J. A., Kuether, E. L., & Montgomery, R. R. (2015). The important role of von Willebrand factor in platelet-derived factor VIII gene therapy of murine hemophilia A in the presence of inhibitory antibodies. Journal of Thrombosis and Haemostasis, 13(7), 1301–1309. https://doi.org/10.1111/jth.12992
Kanaji, S., Fahs, S. A., Ware, J., Montgomery, R. R., & Shi, Q. (2014). Non-myeloablative conditioning with busulfan prior to hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard Soulier syndrome. Journal of Thrombosis and Haemostasis, 12(10), 1726–1732. https://doi.org/10.1111/jth.12694
Mannucci, P. M., Shi, Q., Bonanad, S., & Klamroth, R. (2014). Novel investigations on the protective role of the FVIII/VWF complex in inhibitor development. Haemophilia, 20(Suppl. 6), 2–16. https://doi.org/10.1111/hae.12505
Schroeder, J. A., Chen, Y., Fang, J., Wilcox, D. A., & Shi, Q. (2014). In vivo enrichment of manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection. Journal of Thrombosis and Haemostasis, 12(8), 1283–1293. https://doi.org/10.1111/jth.12632
Fahs, S. A., Hille, M. T., Shi, Q., Weiler, H., & Montgomery, R. R. (2014). Conditional knockout mouse model reveals endothelial cells as the predominant and possibly exclusive source of plasma factor VIII. Blood, 123(24), 3706–3713. https://doi.org/10.1182/blood-2013-12-547810
Shi, Q., Kuether, E. L., Chen, Y., Schroeder, J. A., Fahs, S. A., & Montgomery, R. R. (2014). Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells. Blood, 123(3), 395–403. https://doi.org/10.1182/blood-2013-01-478024
Chen, Y., Schroeder, J. A., Kuether, E. L., Zhang, G., & Shi, Q. (2014). Lentivirus-mediated platelet gene therapy corrects bleeding diathesis and induces humoral immune tolerance in hemophilia B mice. Molecular Therapy, 22(1), 169–177. https://doi.org/10.1038/mt.2013.201
Du, L. M., Nurden, P., Nurden, A. T., Nichols, T. C., Bellinger, D. A., Jensen, E. S., Haberichter, S. L., Merricks, E., Raymer, R. A., Fang, J., Koukouritaki, S. B., Jacobi, P. M., Hawkins, T. B., Cornetta, K., Shi, Q., & Wilcox, D. A. (2013). Platelet α-granules containing human factor VIII induce hemostasis for canine hemophilia A. Nature Communications, 4, 2773. https://doi.org/10.1038/ncomms3773
Shi, Q., Kuether, E. L., Schroeder, J. A., Perry, C. L., Fahs, S. A., Gil, J. C., & Montgomery, R. R. (2012). FVIII inhibitors: VWF makes a difference in vitro and in vivo. Journal of Thrombosis and Haemostasis, 10(11), 2328–2337. https://doi.org/10.1111/j.1538-7836.2012.04929.x
Kuether, E. L., Fahs, S. A., Cooley, B. C., Schroeder, J. A., Chen, Y., Montgomery, R. R., Wilcox, D. A., & Shi, Q. (2012). Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-FVIII immunity. Journal of Thrombosis and Haemostasis, 10(8), 1570–1580. https://doi.org/10.1111/j.1538-7836.2012.04791.x
Kanaji, S., Fahs, S. A., Shi, Q., Haberichter, S. L., & Montgomery, R. R. (2012). Contribution of platelet versus endothelial VWF to platelet adhesion and hemostasis. Journal of Thrombosis and Haemostasis, 10(8), 1646–1652. https://doi.org/10.1111/j.1538-7836.2012.04797.x
Montgomery, R. R., & Shi, Q. (2012). Platelet and endothelial expression of clotting factors for the treatment of hemophilia. Thrombosis Research, 129(Suppl. 2), S46–S48. https://doi.org/10.1016/j.thromres.2012.02.041
Shi, Q., Kuether, E. L., Schroeder, J. A., Fahs, S. A., & Montgomery, R. R. (2012). Intravascular recovery of VWF and FVIII following intraperitoneal injection and differences from intravenous and subcutaneous injection in mice. Haemophilia, 18(4), 639–646. https://doi.org/10.1111/j.1365-2516.2011.02738.x
Kanaji, S., Kuether, E. L., Schroeder, J. A., Fahs, S. A., Ware, J., Montgomery, R. R., & Shi, Q. (2012). Lentivirus-mediated gene therapy of Bernard-Soulier syndrome in a GPIbα deficient mouse model. Molecular Therapy, 20(3), 625–632. https://doi.org/10.1038/mt.2011.265
Montgomery, R. R., & Shi, Q. (2010). Alternative strategies for gene therapy of hemophilia. Hematology. American Society of Hematology. Education Program, 2010, 197–202. https://doi.org/10.1182/asheducation-2010.1.197
Shi, Q., & Montgomery, R. R. (2010). Platelets as delivery systems for disease treatments. Advanced Drug Delivery Reviews, 62(12), 1196–1203. https://doi.org/10.1016/j.addr.2010.06.007
Shi, Q., Fahs, S. A., Kuether, E. L., Cooley, B. C., Weiler, H., & Montgomery, R. R. (2010). Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A. Blood, 116(16), 3049–3057. https://doi.org/10.1182/blood-2010-03-272187
Zhang, G., Shi, Q., Fahs, S. A., Kuether, E. L., Walsh, C. E., & Montgomery, R. R. (2010). Factor IX ectopically expressed in platelets can be stored in α-granules and corrects the phenotype of hemophilia B mice. Blood, 116(8), 1235–1243. https://doi.org/10.1182/blood-2009-11-255729
Shi, Q., Fahs, S. A., Wilcox, D. A., Kuether, E. L., Morateck, P. A., Mareno, N., Weiler, H., & Montgomery, R. R. (2008). Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with pre-existing FVIII immunity. Blood, 112(7), 2713–2721. https://doi.org/10.1182/blood-2008-04-149823
Shi, Q., Wilcox, D. A., Fahs, S. A., Fang, J., Johnson, B. D., Du, L., Desai, D., & Montgomery, R. R. (2007). Lentivirus-mediated platelet-derived factor VIII gene therapy of murine hemophilia A. Journal of Thrombosis and Haemostasis, 5(2), 352–361. https://doi.org/10.1111/j.1538-7836.2006.02376.x
Shi, Q., Wilcox, D. A., Fahs, S. A., Weiler, H., Well, C. C., Cooley, B. C., Desai, D., Morateck, P. A., Gorski, J., & Montgomery, R. R. (2006). Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. Journal of Clinical Investigation, 116(7), 1974–1982. https://doi.org/10.1172/JCI27219
Haberichter, S. L., Shi, Q., & Montgomery, R. R. (2006). The regulated release of VWF and FVIII and the biologic implications. Pediatric Blood & Cancer, 46(5), 547–553. https://doi.org/10.1002/pbc.20631
Haberichter, S. L., Shi, Q., & Montgomery, R. R. (2005). The biology of von Willebrand factor and factor VIII-regulated release. Hematologica Reports, 1(6), 9–14. https://doi.org/10.3324/haematol.2005.s1.9
Shi, Q., Wilcox, D. A., Morateck, P. A., Fahs, S. A., Kenny, D., & Montgomery, R. R. (2004). Targeting GPIbα transgene expression to human megakaryocytes and forming a complete GPIb/IX complex with endogenous GPIbβ and GPIX. Journal of Thrombosis and Haemostasis, 2(11), 1989–1997. https://doi.org/10.1111/j.1538-7836.2004.00975.x
Yarovoi, H., Kufrin, D., Eslin, D. E., Thornton, M. A., Haberichter, S. L., Shi, Q., Zhu, H., Camire, R., Farkharzadeh, S. S., Kowalska, M. A., Wilcox, D. A., Montgomery, R. R., & Poncz, M. (2003). Factor VIII ectopically expressed in platelets: Efficacy in hemophilia A treatment. Blood, 102(12), 4006–4013. https://doi.org/10.1182/blood-2003-05-1519
Shi, Q., Wilcox, D. A., Fahs, S. A., Kroner, P. A., & Montgomery, R. R. (2003). Expression of human factor VIII under control of the IIb promoter in megakaryocytic cell line as well as storage together with VWF. Molecular Genetics and Metabolism, 79(1), 25–33. https://doi.org/10.1016/S1096-7192(03)00052-4
Wilcox, D. A., Shi, Q., Nurden, P., Haberichter, S. L., Rosenberg, J. B., Johnson, B. D., Nurden, A. T., White, G. C., II, & Montgomery, R. R. (2003). Induction of megakaryocytes to synthesize and store a releasable pool of human FVIII. Journal of Thrombosis and Haemostasis, 1(12), 2477–2489. https://doi.org/10.1046/j.1538-7836.2003.00477.x

Qizhen

Shi, MD, PhD

Senior Investigator and Program Co-Leader

Thrombosis and Hemostasis