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Robert R. Montgomery, MD

Robert Montgomery, MD

Senior Investigator

Professor of Pediatric Hematology
Department of Pediatrics
Medical College of Wisconsin

Contact Us

Robert Montgomery, MD

Senior Investigator
Email 414-937-3802 Fax: 414-937-6284

  • Research Interests

    Over the past year we have 1) demonstrated that FVIII is made in endothelial cells; 2) completed enrollment of our historical type 1 Von Willebrand disease cohort of 500 families done across the USA; 3) initiated a prospective study of newly diagnosed VWD patients across the USA; 4) did BM TxP with gene therapy of the stem cells to direct FVIII production in platelets; and 5) initialed studies to study acquired abnormalities of von Willebrand factor in newborns having open heart surgery for complex congenital heart disease.

    While scientists have recognized for years that liver transplant can cure hemophilia and concluded that FVIII must be made in the liver hepatocyte. Using various mouse models that we developed, studies were possible to block the FVIII production in a single type of cell. For example we can remove the FVIII functional gene from all hepatocytes. Interestingly, the FVIII level in the mouse plasma was normal and not affected – thereby proving that hepatocytes didn’t make FVIII. We then did further “tissue-specific” knockout or elimination of the functional FVIII gene from other cells and thereby proved that if you stopped FVIII production in endothelial cells, there was no FVIII in plasma and thereby proved that FVIII was produced in endothelial cells where it was stored and released physiologically with its companion molecule von Willebrand factor.

    Plasma FVIII Activity Chart

    This figure demonstrates that crossing our mouse with a “floxed” FVIII gene with mice with tissue-specific “Cre-recombinase” demonstrate no reduction of FVIII in the albumin-Cre (hepatocyte-specific) mouse, but total elimination of plasma FVIII in the Tek-Cre (endothelial cell specific) mouse. 

    We have finished the 7th year of a multinational program project that is focused on von Willebrand Disease (VWD). VWD is probably the most common hereditary bleeding disorder and may affect 1 in 50 to 1 in 1000 of our general population. In studying our cohort of 500 families with the common form of VWD, type 1 VWD, we found that not all of those subjects continue to have abnormal levels of VWF. We do find that most have a gene mutation in their VWF gene that can be associated with low VWF in other family members. We have recently found a severe form of VWD in which their blood VWF doesn’t bind to exposed collagen after blood vessels are damaged. Current testing as done widely in the United States misses this form of VWD, yet these patients would benefit from VWF replacement therapy. Also we have shown that certain VWF laboratory test (ristocetin-cofactor) is not accurate in as many as 67% of African Americans – they might be assumed to have VWD when they really are normal.

    Since our historic diagnosis of VWD might have diagnostic problems, might be develop algorithms that correctly diagnose VWD patients more correctly and provide individualized advice on need for treatment when bleeding is encountered.

    For hemophilia we have devised a strategy of gene therapy where we would take a hemophilia patients bone marrow and do gene therapy on his hematopoietic stem cells (blood stem cells) so that the FVIII (or FIX) gene is turned on only in platelets where it is stored in storage granules. If blood vessels are injured the platelet sticks to the site because of VWF and the platelets activate and release the FVIII that was put there by gene therapy and the FVIII accelerates clotting and stops the clinical bleeding. To see if there is any thrombosis risk, we have done similar bone marrow transplants with platelet FVIII into mice with a thrombotic propensity. Adding the platelet FVIII does NOT seem to increase the likelihood of clinical thrombosis. This suggests this approach may be safe.

    Our lab has shown that heart valve problems result in the loss of the most active “large” forms of VWF that are most likely to stop clinical bleeding. When infants in their first month of life have major heart surgery for complex congenital heart disease, they sometimes encounter excess abnormal bleeding. Our studies are directed at defining if this bleeding is caused by abnormal VWF and if so, could VWF replacement be optimized to control hemorrhage.

    These 5 studies are directed at increasing our understanding of both bleeding and clotting disorders and their appropriate clinical management.

  • Grant Support
    • P01 HL81588, NIH, Zimmerman Program for the Molecular and Clinical Biology of VWD. 2012-2017.
      • RR Montgomery, Program Director.
    • R01 HL112614, NIH, Comparative Effectiveness in the Diagnosis of VWD.  2013-2018.
      • RR Montgomery and TC Abshire, Principle Investigators.
    • P01 HL44612, NIH, Molecular and Cellular Mechanisms in Transfusion Medicine. 2011-2016.
      • Project 5: Critical Molecular Interactions of VWF and FVIII.
        RR Montgomery, Project Leader.

    Research Projects:

    • Gene Therapy of hemophilia through platelet targeted FVIII of FIX
    • Tissue of origin for FVIII production
    • Murine models of VWD
    • Molecular and clinical biology of VWD
    • Clinical assays of various VWF functions
    • Comparative effectiveness of methods to diagnose VWD
    • Abnormal VWF functions during open heart surgery in neonates
  • Lab

    Veronica Flood
    MCW Asst. Professor

    Scot Fahs
    Senior Research Technologist 

    Patricia Morateck
    Senior Research Technologist

    Pam Christopherson
    Senior Research Coordinator

    Crystal Perry
    Research Technologist

     Tricia Slobodianuk
    Research Technologist

    Jeremy Mattson
    Research Technologist

    Aminah Hamdan
    Assistant Research Coordinator

    Tia Baas
    Assistant Research Technologist

    Ursula Sicking
    Assistant Research Technologist

    Christopher Nasby
    Assistant Research Technologist

  • Publications

    Selected Publications

    • Montgomery RR, Zimmerman TS: von Willebrand's disease antigen II ‑ a new plasma and platelet antigen deficient in severe von Willebrand's disease. J Clin Invest 61:1498‑1507, 1978. 
    • Rosenberg JB, Foster PA, Kaufman RJ, Vokac EA, Kroner PA, Montgomery RR: Intracellular trafficking of Factor VIII to von Willebrand factor storage granules. J Clin Invest 101: 613-624, 1998.  (PMCID: PMC508605)
    • Montgomery RR and Cox Gill J:  Interactions Between von Willebrand factor and factor VIII: Where did they first meet? J Pediatr Hematol Oncol 22(3):269-275, 2000.   
    • Haberichter SL, Fahs SA, Montgomery RR: Von Willebrand factor (vWF) storage and multimerization: Two independent intracellular processes. Blood 96(5):1808-1815, 2000.   
    • Rosenberg JB, Greengard JS, and Montgomery RR: Genetic induction of a releasable pool of factor VIII in human endothelial cells. Arterioscler Thromb Vasc Biol. 20:2689-2695, 2000.   
    • Haberichter SL, Jozwiak MA, Rosenberg JB, Christopherson PA, Montgomery RR: The von Willebrand Factor Propeptide VWFpp Traffics An Unrelated Protein to Storage. Arterioscler Thromb Vasc Biol 22:921-926, 2002.   
    • Haberichter SL, Jacobi P, and Montgomery RR: Critical Independent Regions in the VWF Propeptide and Mature VWF That Enable Normal VWF Storage. Blood 101(4):1384-1391, 2003.   
    • Shi Q, Wilcox DA, Fahs SA, Kroner PA, Montgomery RR: Expression of Human Factor VIII under Control of the αIIb Platelet-Specific Promoter in Megakaryocytic Cell Line as well as Storage Together with VWF. Molecular Genetics and Metabolism 79:25-33, 2003.      
    • Wilcox DA, Shi Q, Nurden P, Haberichter SL, Rosenberg JB, Johnson BD, Nurden AT, White II GC, Montgomery RR: Induction of Megakaryocytes to Synthesize and Store a Releasable Pool of Human Factor VIII. J Thromb Haemost 1:2477-2489, 2003.   
    • Shi Q, Wilcox DA, Morateck PA, Fahs SA, Kenny D, Montgomery RR: Targeting Platelet GPIba Transgene Expression to Human Megakaryocytes and Forming a Complete Complex with Endogenous GPIbb and GPIX. J Thromb Haemost 2004; 2(11):1989-1997, 2004.   
    • Haberichter SL, Merricks EP, Fahs SA et al. Re-establishment of VWF-dependent Weibel-Palade bodies in VWD endothelial cells. Blood 105:145-152, 2005.     
    • Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR: Factor VIII Ectopically Targeted Top Platelets is Therapeutic in Hemophilia A With High-Titer Inhibitory Antibodies. J Clin Invest 116:1974-82, 2006.
    • Haberichter SL, Balistreri M, Christopherson P, Morateck P, Gavazova S, Manco-Johnson MJ, Gill JC, Montgomery RR: Assay of the von Willebrand Factor (VWF) Propeptide to Identify Type 1 von Willebrand Disease Patients with Decreased VWF Survival. Blood. 108: 3344-3351, 2006.  
    • Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, Desai D, Morateck PA, Montgomery RR. Lentivirus-mediated Platelet-derived factor VIII (FVIII) Gene Therapy in Murine Hemophilia A. J Thromb Haemost, 5:352-361 2007.   
    • Nichols WL, Hultin MB, James AH, Manco-Johnson MJ, Montgomery RR, Ortel TL, Rick ME, Sadler JE, Weinstein M, and Yawn BP: Von Willebrand Disease (VWD): Evidence-Based Diagnosis and Management Guidelines from the National Heart, Lung, and Blood Institute (NHLBI) Expert Panel (USA). Haemophilia, 14(2):171-232, 2008.   
    • Haberichter SL, Allmann AM, Jozwiak MA, Montgomery RR, Gill JC: Genetic Alteration of the D2 Domain Abolishes von Willebrand Factor Multimerization and Trafficking into Storage. J Thromb Haemost, 7:641-50, 2009.   
    • Shi Q, Fahs SA, Wilcox DA, Kuether EL, Morateck PA, Mareno N, Weiler H, Montgomery RR: 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 Immunity. Blood 112:7, 2713-2721 2008.
    • Flood VH, Friedman KD, Gill JC, Morateck PA, Wren JS, Scott JP, Montgomery RR. Limitations of the ristocetin cofactor assay in measurement of von Willebrand factor function. J.Thromb.Haemost. 2009, 7:1832-1839.
    • Flood VH, Lederman CA, Wren JS, Christopherson PA, Friedman KD, Hoffmann RG, Montgomery RR. Absent collagen binding in a VWF A3 domain mutant: utility of the VWF:CB in diagnosis of VWD. J.Thromb.Haemost. 2010, 8:1431-1433.
    • Zhang G, Shi Q, Fahs SA, Kuether EL, Walsh CE, Montgomery RR. Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice. Blood 2010, 116:1235-1243.
    • Flood VH, Gill JC, Morateck PA, Christopherson PA, Friedman KD, Haberichter SL, Branchford BR, Hoffmann RG, Abshire TC, DiPaola JA, Hoots WK, Leissinger C, Lusher JM, Ragni MV, Shapiro AD, Montgomery RR for the Zimmerman Program for the Molecular and Clinical Biology of VWD: VWF Exon 28 Polymorphisms in African Americans Affecting the VWF:RCo/VWF:Ag Ratio. Blood 2010, 116(2):280-286. 
    • Castaman G, Motgomery RR, Meschengieser SS, Haberichter SL, Woods AI, and Lazzari, MA. 2010 von Willwbrand's disease diagnosis and laboratory issues. Hemophilia. 16 Suppl 5:67-73.
    • Montgomery RR, Monahan PE, Ozelo MC: Unique Strategies for Therapeutic Gene Transfer in Haemophilia A and Haemophilia B.   Haemophilia 2010; 16-S5 529-534.
    • Shi Q, Fahs SA, Kuether EL, Cooley BC, Weiler H, Montgomery RR. Targeting FVIII Expression to Endothelial Cells Regenerates a Releasable Pool of FVIII and Restores Hemostasis in a Mouse Model of Hemophilia A. Blood 2010 116:16 3049-3057.
    • Shi,Q., and Montgomery,R.R. 2010. Platelets as delivery systems for disease treatments. Adv. Drug Deliv. Rev. 62:1196-1203.
    • Flood VH, Gill JC, Morateck PA, Christopherson PA, Friedman KD, Haberichter SL, Hoffmann RG, Montgomery RR. Gain-of-function GPIb ELISA assay for VWF activity in the Zimmerman Program for the Molecular and Clinical Biology of VWD. Blood 2011;117(6):e67-e74. (PMCID: PMC3056647).
    • Flood VH, Gill JC, Bellissimo DB, Haberichter SL, Friedman KD, Montgomery RR. VWD in the United States: A Perspective from Milwaukee, Wisconsin. Semin. Thromb. Hemost., 2011 37:528-534.
    • Shi Q, Kuether EL, Schroeder JA, Zhang G, Fahs SA, Montgomery RR. 2012.  Intraperitoneal Injection and Recovery of VWF and FVIII and Differences from Intravenous and Subcutaneous Injection in Mice. Haemophilia. 18:639-646. 
    • Bellissimo DB, Christopherson PA, Flood VH, Gill JC, Friedman KD, Haberichter SL, Shapiro AD, Abshire TC, Leissinger C, Hoots WK, Lusher JM, Ragni MV, Montgomery RR. 2012. Von Willebrand Factor Mutations and New Sequence Variations Identified in Healthy Controls Enrolled in the Zimmerman Program for the Molecular and Clinical Biology of VWD (ZPMCB-VWD) are More Frequent in the African American Population. Blood119:2135-2140.
    • Flood VH, Gill JC, Christopherson PA, Wren JS, Friedman KD, Haberichter SL, Lentz SR, Hoffmann RG, Montgomery RR. VWF Mutations Can Selectively Alter Binding to Type VI or Types I and III Collagen. In Press, JTH, 2012.
    • Kuether EL, Fahs SA, Cooley BC, Schroeder JA, Montgomery RR, Wilcox DA, Shi Q. 2012.  Lentivirus-mediated platelet gene therapy of murine hemophiia A with pre-existinganti-factor VIII immunity. J. Thromb. Haemost. 10:1570-1580.
    • Kanaji S, Kuether EL, Fahs SA, Schroeder JA, Ware J, Montgomery RR, Shi Q. Correction of Murine Bernard Soulier Syndrome by Lentavirus-mediated Gene Therapy. Molecular Therapy, 2012. 20:625-632.
    • Montgomery RR, Shi Q. Platelet and Endothelial Expression of Clotting Factors for the Treatment of Hemophilia. Thrombosis Research, 2012.  129 Suppl 2:S46-S48.
    • Hubbard,A.R., Hamill,M., Eikenboom,H.C., Montgomery,R.R., Mertens,K., Haberichter,S., and SSC sub-committee on von Willebrand factor of ISTH 2012. Standardization of von Willebrand factor propeptide: value assignment to the WHO 6th IS Factor VIII/von Willebrand factor, plasma (07/316). J. Thromb. Haemost. 10:959-960.
    • Kanaji,S., Fahs,S.A., Shi,Q., Haberichter,S.L., and Montgomery,R.R. 2012. Contribution of platelet vs. endothelial VWF to platelet adhesion and hemostasis. J. Thromb. Haemost. 10:1646-1652.
    • Ghosh,A., Vo,A., Twiss,B.K., Kretz,C.A., Jozwiak,M.A., Montgomery,R.R., and Shavit,J.A. 2012. Characterization of zebrafish von Willebrand factor reveals conservation of domain structure, multimerization, and intracellular storage. Adv. Hematol. 2012:214209.
    • Shi, Q., Kuether, E.L., Schroeder, J.A., Perry, C.L. Fahs, S.A., Cox, G.J., and Montgomery, R.R. 2012.  Factor VIII inhibitors: von Willebrand factor makes a difference in vitro and in vivo.  J. Thromb Haemost, 10(11):2328-37.
    • Berntorp, E., Peake, I., Budde, U., Laffan, M., Montgomery, R., Windyga, J., Goodeve, A., Petrini, P., von, D.M., Miesbach, W., et all 2012.  von WIllebrand's disease: a report from a meeting in the Aland islands.  Haemophilia, 18 Suppl 6:1-13.
    • Flood, V.H., Gill, J.C., Friedman, K.D., Christopherson, P.A., Jacobi, P.M., Hoffman, R.G., Montgomery, R.R., Haberichter, S.L., and Zimmerman Program. 2013.  Collagen binding provides a sensitive screen for variant von Willebrand disease.  Clin Chem, 59:684-691.
    • Johansson, M.W., Kruger, S.J., Schiebler, M.L., Evans, M.D., Sorkness, R.L., Denlinger, L.C., Busse, W.W., Jarjour, N.N., Montgomery, R.R., Mosher, D.F., et al. 2013.  Markers of vascular perturbation correlate with airway structural change in asthma.  Am. J. Respir. Crit Care Med. 188:167-178.
    • Berntorp, E., Fuchs, B., Makris, M., Montgomery, R., Flood, V., O'Donnell, J.S., Federici, A.B., Lillicrap, D., James, P., Budde, U., et al. 2013. Third Aland islands conference on von Willibrand disease, 26-28 September 2012: meeting report.  Haemophilia. 19 Suppl 3:1-18.
    • Rydz, N., Swystun, L.L., Notley, C., Paterson, A.D., Riches, J.J., Sponagle, K., Boonyawat, B., Montgomery, R.R., James, P.D., and Lillicrap, D., 2013.  The C-type lectin receptor CLEC4M binds, internalizes and clears von Willwbrand factor levels.  Blood, 121:5228-5237.
    • Flood, V.H., Friedman, K.D., Gill, J.C., Haberichter, S.L., Christopherson, P.A., Branchford, B.R., Hoffman, R.G., Abshire, T.C., Dunn, A.L., DiPaola, J.A., Hoots, W.R., Brown, D.L., Leissinger, C., Lusher, J.M., Ragni, M.V., Shapiro, A.D., Montgomery, R.R. 2013.  No Increase in bleeding identified in type 1 VWD subjects with D1472H sequence variation.  Blood, 18:3742-3744.
    • Kanaji, T., Kanaji, S., Montgomery, R.R., Patel, S.B., Newman, P.J. 2013. Platelet hyperreactivity explains the bleeding abnormality and macrothrombocytopenia in a murine model of sitosterolemia.  Blood, Oct 10; 122(15):2732-2742.
    • Shi, Q., Kuether, E.L., Chen, Y., Schroeder, J.A., Fahs, S.A., Montgomery, R.R. 2013. Platelet gene therapy corrects the hemophilic phenotype in immunocompromized hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.  Blood, in press.
    • Brott, D.A., Katein, A., Thomas, H., Lawton, M., Montgomery, R.R., Richardson, R.J., Louden, C.S. Evaluation of Von Willebrand Factor and Von Willebrand Factor Propeptide in Models of Vascular Endothelial Cell Activation, Perturbation, and/or Injury.  Toxicologic Pathology In Press. 2013.
    • Elbatarny, M., Mollah, S., Grabell, J., Bae, S., Deforest, M., Tuttle, A., Clark, A.D., Mauer, A.C., Bowman, M., Riddel, J., Christopherson, P.A., Montgomery, R.R., Rand, M.L., Coller, B., James, P.A.  Normal Range of Bleeding Scores for the ISTH-BAT: Adult and Pediatric Data from the Merging Project.  In Press. 

    Selected Book Chapters 2010-present (from 44 publications)

    • Montgomery RR, Flood VH: Biological Diagnosis of VWD. Textbook of Hemophilia, 2nd Edition. Eds. Lee C, Berntorp E, Hoots K. Wiley-Blackwell Publishing.   In Press January 2010.
    • Haberichter SL, Montgomery RR: Chapter 2. Biosynthesis and Organization of VWF. von Willebrand Disease: Basic and Clinical Aspects, 1st Edition. Eds. Federici AB, Lee, C, Berntorp E, Lillicrap D, Montgomery RR. Wiley-Blackwell Publishing, 2011.
    • Montgomery RR, Haberichter SL: Chapter 3. VWF Structure and Function. Textbook of Hemophilia, 2nd Edition. Eds. Federici AB, Lee C, Berntorp E, Lillicrap D, Montgomery RR. Wiley-Blackwell Publishing, 2011.
    • Montgomery RR, Shi Q: New Strategies for Gene Therapy of Hemophilia. ASH State of the Art Book, December 2010.
    • Haberichter SL, Montgomery RR: Structure and Function of von Willebrand Factor. Hemostasis and Thrombosis – Basic Principles and Clinical Practice, 6th Edition. Eds. Coleman, Marder, Clowes, George, and Goldhaber. Lippincott, Williams, Wilkins, Philadelphia PA, 2012.

    Selected Abstracts

    • Shi Q, Kuether EL, Cooley BC, Montgomery RR, Wilcox DA, Fahs SA, Schroeder J. Gene Therapy of Hemophilia A Mice with Pre-existing Immunity Using Lentivirus-Mediated Platelet-Specific Gene Transfer. Submitted for presentation at the 14th Annual Meeting of American Society of Gene and Cell Therapy, Seattle, May 2011.
    • Johnasson MW, Han S-T, Gunderson KA, Montgomery RR, Busse WW, Jarjour NN, Mosher DF. Platelet Activation, P-selectin Mmobilization, and Eosinophil β1 Integrin Activation Occur in Asthma and are Associated with Clinical Phenotypes. Presented at the American Thoracic Society Meeting, Denver, Colorado, May 2011 .
    • Bellissimo DB, Chin E, Hegde MR, Christopherson PA, Haberichter SL, Montgomery RR, and Zimmerman Program Investigators. Detection of VWF Copy Number Variations by Microarray Analysis in Von Willebrand Disease Ty[e 3 Index Cases. ASH 2011.
    • Chen Y, Kuether EL, Schroeder JA, Montgomery RR, Scott DW, Shi Q. Targeting FVIII Expression to Platelets Induces Immune Tolerance in Hemophilia A Mice with or without Pre-Existing Anti-FVIII Immunity.   ASH 2011.
    • Flood VH, Gill JC, Friedman KD, Christopherson PA, Jacobi PM, Montgomery RR, Haberichter SL. Von Willebrand Factor Collagen Binding Provides a Sensitive Screen for Identification of Type 2A and 2B Von Willebrand Disease. ASH 2011.
    • Shi Q, Kuether EL, Fahs SA, Schroeder JA, Montgomery RR. Targeting FVIII Expression to Human Platelets Corrects the Hemophilic Phenotype in an Immunocompromised Hemophilia A Mouse Model Transplanted with Genetically Manipulated Human Cord Blood Stem Cells. ASH 2011.
    • Montgomery RR, Christopherson PA, Haberichter SL, Flood VH, Gill JC, and The Zimmerman Investigators. Diagnostic Fidelity of Historically Diagnosed Patients with VWD Enrolled from 27 Centers in the ZPMCB-VWD. ASH 2011.
    • Haberichter SL, Jacobi PM, Endres JL, Covill S, Flood VH, Montgomery RR, Gill JC, Friedman KD.  Discrimination between VWD Subtypes by Quantitative Analysis of VWF Multimer Structure. Submitted to Thrombosis and Hemostasis North America Summit, 2011. Shi Q, Kuether EL, Schroeder JA, Perry CL, Fahs SA, Gill JC, Montgomery RR. VWF Exerts A Protective Effect on FVIII from Inhibitor Inactivation Both In Vitro and In Vivo. Submitted to ISTH SSC 2012.
    • Shi Q, Kuether EL, Fahs SA, Schroeder JA, Montgomery RR. Platelet Gene Therapy Corrects the Hemophilic Phenotype in Immunocompromised Hemophilia A Mice Transplanted with Genetically Manipulated Human Cord Blood Stem Cells. Submitted to ISTH SSC 2012.
    • Gupta S, Heiman M, Duncan N, Yoder P, Montgomery R, Hinckley J, DiPaola J, Shapiro AD. Comparison of Quality of Life in an Amish and Non-Amish Cohort Affected with Von Willebrand Disease. Submitted to ISTH SSC 2012.
    • Mollah S, Grabell MS, Rand MK, Clarke D, Christopherson P, Mauer A, Montgomery RR, Zimmerman Program Investigators, Coller B, James PD.  The Merging Project: A Machine Learning Approach to Merge and Analyze Data from Four Different Bleeding Questionnaires, submitted to ISTH Amsterdam, 2013. 
    • Fahs S, Hille M, Jozwiak M, Shi Q, Weiler H, Montgomery R., Factor VIII: Where is it synthesized?  ISTH, 2013.
    • Kanaji S, Fahs S, Ware J, Montgomery R, Shi Q.  Non-myeloablative conditioning with busulfan prior to hematopoetic stem cell transplantation leads to phenotypic correction of murine Bernard Soulier Syndrome.  ISTH, 2013. 
    • Chen Y, Schroeder J, Kuether E, Montgomery R, Shi Q. Platelet-specific expression of FIX induced by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces immune tolerance in hemophilia B mice.  ISTH, 2013.
    • Baumgartner CK, Shi Q, and Montgomery RR.  Native Whole Blood Thrombin Generation Assay Evaluates Therapeutic Efficacy of Plasma and Platelet-Derived FVIII.  American Society of Hematology, 2013.
    • Hinckley DJ, Burns TL, Wang K, Ozel AB, Heiman M, Shapiro A, Montgomery RR, Desch K, and Paola JD.  SNP Analysis of the VWF Gene Identifies Multiple Common Varients that Affect VWF Levels and Occur at Different Frequencies in Patients with Type 1 VWD, American Society of Hematology, 2013. 
    • Fahs S, Hille M, and Montgomery RR.  Transcriptional Analysis of a Tissue-Specific Factor VIII Knockout Model Demonstrates the Importance of Endothelial Factor VIII Synthesis, American Society of Hematology, 2013. 
    • Flood VH, Gill JC, Haberichter SL, Bellissimo DB, Christopherson PA, Friedman KD, Montgomery RR, and The Zimmerman Program Investigators.  Binding of VWF to Type IV Collagen: An Additional Collagen Binding Mechanism Beyond Types I, III, and VI Collagen? American Society of Hematology, 2013.
    • Flood VH, Schlauderaff AC, Jacobi PM, Slobodianuk TL, Montgomery RR, Haberichter SL, and The Zimmerman Program Investigators. VWF Interaction With Type IV Collagen Is Mediated Through Critical VWF A1 Domain Residues, American Society of Hematology, 2013.
    • Haberichter SL, Christopherson PA, Flood VH, Gill JC, Bellissimo DB, Friedman KD, Montgomery RR, and The Zimmerman Program Investigators.  Critical Importance of VWF Propeptide VWFpp) in the Diagnosis of Type 1 Von Willebrand Disease (VWD), American Society of Hematology, 2013. 
    • Montgomery RR, Haberichter SL, Hille M, Morateck P, Scott JP, and Flood VH.  Alanine Scanning Mutagenesis of the VWF-A1 Flanking Regions of VWF and the Impact of Ristocetin-induced VWF Interation with Platelets, American Society of Hematology, 2013.
    • Montgomery RR, Christopherson PA, Bellissimo DB, Gill JC, Haberichter SL, Flood VH, Lillicrap D, Goodeve ACX, Friedman KD, Abshire TC, Shapiro AD, Ragni MV, Lentz SR, Leissinger CA, Dunn AL, Lusher J, and Brown D. The Complete Type I VWD Cohort Of The Zimmerman Program For The Molecular and Clinical Biology of VWD - Phenotypic Assignment, Mutation Frequency, and Bleeding Assessment, American Society of Hematology, 2013.
    • Roberts JC, Morateck PA, Christopherson PA, Yan K, Hoffman RG, Gill JC, Montgomery RR, and The Zimmerman Program Investigators.  A Novel Rapid Screening Assay For The Diagnosis Of The Phenotypic Variants Of Von Willebrand Disease (VWD), S American Society of Hematology, 2013.
    • Shi Q, Schroeder JA, Wilcox DA, Montgomery RR, and Chen Y.  In Vivo Selection Of Genetically Manipulated Hematopoietic Stem Cells For Platelet Gene Therapy of Hemophilia A, American Society of Hematology, 2013. 
    • Elbatarny M, Mollah S, Grabell J, Bae S, Deforest M, Tuttle A, Clark AD, Mauer AC, Bowman M, Riddel J, Christopherson PA, Montgomery RR, Rand ML, Coller B, James PA.  Normal Range of Bleeding Scores for the ISTH-BAT: Adult and Pediatric Data from the Merging Project.  American Society of Hematology, 2013.
  • Other


    • "Method for maintaining intact, non-degraded factor VIII/von Willebrand factor during blood processing." filed 1984, issued 1987. TJ Kunicki, PhD, and RR Montgomery, MD.
    • "Method for maintaining intact, non-degraded factor VIII/von Willebrand factor during blood processing." filed 1987, issued 1992. TJ Kunicki, PhD, and RR Montgomery, MD.
    • "Synthetic and Recombinant Substrates for the Detection of the von Willebrand Factor-Cleaving Protease.” filed 2001, RR Montgomery, MD.
    • "Methods and kits for measuring von Willebrand factor." filed 2008, issued 2012. RR Montgomery, MD.
    • "Methods and kits for measuring von Willebrand factor." filed 2011, issued 2012.  RR Montgomery, MD.

    Invited Lectures 2010-present

    • “Laboratory Testing and diagnosis of vWD” Symposium Co-Chair, Contemporary Challenges in the Diagnosis and Management of von Willebrand Disease, Montreal, Quebec, Canada April, 2010.
    • “Pathophysiology of Type 1 VWD” Canadian Von Willebrand Disease Update, Toronto, Canada, April, 2010. 
    • “Alternative Assays for GP1-Binding of VWD” , 56th Annual Meeting Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis, Cairo, Egypt, May 2010.
    • “Accelerated Clearance of VWF”, XXIX International Congress of the World Federation of Hemophilia – Hemophilia 2010, Buenos Aires, Argentina, July 2010.
    • “Therapeutic Gene Transfer”, XXIX International Congress of the World Federation of Hemophilia – Hemophilia 2010, Buenos Aires, Argentina, July 2010.
    • “Platelet-Based Gene Therapy”, XXIX International Congress of the World Federation of Hemophilia – Hemophilia 2010, Buenos Aires, Argentina, July 2010.
    • “von Willebrand Disease – the Diagnostic Relevance of the Phenotype” UK NEQAS for Blood Coagulation Annual Scientific Meeting, Sheffield, UK, September, 2010.
    • “Gene Therapy Through Platelet Drug Delivery in Hemophilia A & B”, Seminar Series, University of North Carolina at Chapel Hill, September, 2010.
    • “Classification of VWD” The Aland Islands Meeting, Mariehamm, the Aland Islands, Finland, September, 2010.
    • “Fidelity of VWD Diagnosis – from ristocetin to collagen”, 7th Bari International Conference, Pugnochiuso, Vieste, Italy, May 2011
    • “The Trojan Horse Approach to Gene Therapy of Hemophilia A and B”, 2011 Annual BIIB Hemophilia Research Seminar Series, Biogen Idec Inc., Waltham, Massachusetts, June 7, 2011.
    • “The Fidelity of VWD Diagnosis”, 2011 Annual BIIB Hemophilia Research Seminar Series, Biogen Idec, Inc., Waltham, Massachusetts, June 7, 2011.
    • “Zimmerman Project (ZPMCB-VWD) Update on Activities”, von Willebrand Factor SSC Meeting, XXIII Congress and 57th Annual SSC Meeting, International Society of Thrombosis and Hemostasis (ISTH), Kyoto, Japan, July 24, 2011. 
    • “Gene Therapy of Hemophilia through Platelet-targeted Gene delivery”, Bayer Hemophilia Care Innovation Summit, Budapest, Hungary, October 13, 2011.
    • “Hemophilias and von Willebrand Disease”, Continuing Education in Thrombosis & Hemostasis ISTH-ESH Joint Advanced Training Course, Cascais, Portugal, November 7, 2011.
    • “Genes and Platelets”, XVth Annual London Symposium, Do Genes Trump Current Therapy? London, United Kingdom, January 8-10, 2012.
    • "Pathophysiology of von Willebrand Disease," Advances in Thrombosis and Hemostasis, Chilian Society of Hematology, Santiago, Chile, April 12, 2012.
    • "Diagnosis and Treatment of von Willebrand Disease", Advances in Thrombosis and Hemostasis, CHilian Society of Hematology, Santiago, Chile, April 12, 2012.
    • "VWF Function: A Laboratory Perspective", Hemophilia Research Study Update 2012, Miami, Florida, March 22, 2012. 
    • "Non-Hepatic Expression of Clotting Factors", 6th Symposium on Hemostasis with Special Focus on Tissue Factor, Factor VIIa, and Tissue Factor Pathway Inhibitor: Hemostasis and Beyond.  Chapel Hill, North Carolina, May 5, 2012.
    • "The Fidelity of the Diagnosis of von Willebrand Disease", Spaet Visiting Professor for the Theodore H. Spaet Symposium on the Basic Science & Laboratory Diagnosis of Platelet Dysfunction.  Montefiore Medical Center, The Albert Einstein College of Medicine, Bronx, New York, May 17, 2012.
    • "Fidelity of VWD Testing", Lab Medicine 2012 The 47th Annual Laboratory Medicine Meeting of the Academy of Clinical Laboratory Physicians & Scientists, Milwaukee, Wisconsin, May 31, 2012.
    • "Von Willebrand Disease", The Mike Nesheim "Meet the Expert" Session at the 58th Meeting of the Scientific & Standardization Committee of the International Society on Thrombosis and Haemostasis, Liverpool, United Kingdom, June 27, 2012.
    • "Zimmerman Project (ZPMCB-VWD)", Scientific & Standardization Committee of the International Society on Thrombosis and Haemostasis, Liverpool, United Kingdom, June 30, 2012. 
    • "Measuring VWF - Gp1b Binding: Potential for Better Understanding of VWF Functionality compared to RiCoF?". Aland Island Meeting, Mariehamn, Aland Islands, Finland, September 26, 2012.
    • "Laboratory and Translational Hematology - Trainee Career Development Lunch", 54th ASH Annual Meeting and Exposition, Atlanta, GA, December 2012.
    • "Platelet Targeted Gene Therapy for Hemophilia", Visitning Professor, Centre Blood Research in Vancouver, British Columbia, Canada, March 5, 2013.
    • "The Fidelity of VWD Diagnosis", Visiting Professor, Center for Blood Research, Diamond Health Care Centre, Vancouver, British Columbia, Canada, March 5, 2013.
    • "Diagnosis of Von Willebrand Disease", LIV Annual Congress AMEH 2013, Hematology Mexican Society, Mazatlan, Mexico, April 29, 2013.

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