D Lillicrap

Summary

Affiliation: Queen's University
Country: Canada

Publications

  1. ncbi Efficacy and safety of the factor VIII/von Willebrand factor concentrate, haemate-P/humate-P: ristocetin cofactor unit dosing in patients with von Willebrand disease
    D Lillicrap
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    Thromb Haemost 87:224-30. 2002
  2. ncbi Cellular and genetic therapies for haemophilia
    D Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Ontario, Canada
    Haemophilia 12:36-41. 2006
  3. ncbi Extending half-life in coagulation factors: where do we stand?
    David Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Canada
    Thromb Res 122:S2-8. 2008
  4. ncbi Genotype/phenotype association in von Willebrand disease: is the glass half full or empty?
    D Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Canada K7L 3N6
    J Thromb Haemost 7:65-70. 2009
  5. ncbi [Application studies on the gene diagnosis and carrier detection of hemophilia A by using polymerase chain reaction-conformation sensitive gel electrophoresis]
    David Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory of Queen s University, Kingston, Ontario, Canada
    Zhonghua Yi Xue Yi Chuan Xue Za Zhi 26:393-9. 2009
  6. ncbi Gene expression: overview and clinical implications
    David Lillicrap
    Dept. of Pathology, Queen's University, Kingston, ON, Canada
    Vox Sang 83:77-9. 2002
  7. ncbi The improved factor concentrate
    D Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Canada K7L 3N6
    Hamostaseologie 29:71-3. 2009
  8. ncbi The genetics of venous and arterial thromboembolism
    D Lillicrap
    Department of Pathology, Queen s University, 99 University Avenue, Kingston, Ontario, K7L 3N6, Canada
    Curr Atheroscler Rep 3:209-15. 2001
  9. ncbi Hemophilia treatment. Gene therapy, factor VIII antibodies and immune tolerance: hopes and concerns
    D Lillicrap
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    Haematologica 85:108-11; discussion 111-2. 2000
  10. ncbi Von Willebrand disease - phenotype versus genotype: deficiency versus disease
    David Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Canada
    Thromb Res 120:S11-6. 2007

Collaborators

Detail Information

Publications78

  1. ncbi Efficacy and safety of the factor VIII/von Willebrand factor concentrate, haemate-P/humate-P: ristocetin cofactor unit dosing in patients with von Willebrand disease
    D Lillicrap
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    Thromb Haemost 87:224-30. 2002
    ..The findings in this study confirm the safety and efficacy of Haemate-P/Humate-P using VWF:RCo dosing in pediatric and adult patients with various types of VWD...
  2. ncbi Cellular and genetic therapies for haemophilia
    D Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Ontario, Canada
    Haemophilia 12:36-41. 2006
    ..Two new clinical trials, both using AAV vectors, will likely start within the next year, and additional large animal pre-clinical studies using other viral vector-mediated approaches for gene transfer are expected in the near future...
  3. ncbi Extending half-life in coagulation factors: where do we stand?
    David Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Canada
    Thromb Res 122:S2-8. 2008
    ..One of the more promising approaches involves prolonging the half-life of FVIII. This article summarizes the methods that are being used to extend FVIII half-life...
  4. ncbi Genotype/phenotype association in von Willebrand disease: is the glass half full or empty?
    D Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Canada K7L 3N6
    J Thromb Haemost 7:65-70. 2009
    ..Most recently, preliminary results relating to the mutational landscape of type 1 disease have been published that highlight the complex pathogenic background of this mild/moderate quantitative trait...
  5. ncbi [Application studies on the gene diagnosis and carrier detection of hemophilia A by using polymerase chain reaction-conformation sensitive gel electrophoresis]
    David Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory of Queen s University, Kingston, Ontario, Canada
    Zhonghua Yi Xue Yi Chuan Xue Za Zhi 26:393-9. 2009
    ..To establish a simple, rapid and easy method for screening the gene mutation in hemophilia A, which was further applied to a direct diagnosis and carrier detection at gene level...
  6. ncbi Gene expression: overview and clinical implications
    David Lillicrap
    Dept. of Pathology, Queen's University, Kingston, ON, Canada
    Vox Sang 83:77-9. 2002
  7. ncbi The improved factor concentrate
    D Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Canada K7L 3N6
    Hamostaseologie 29:71-3. 2009
    ..Coincident with these approaches, it is hoped that there will be more widespread availability of these concentrates and that their cost will be contained...
  8. ncbi The genetics of venous and arterial thromboembolism
    D Lillicrap
    Department of Pathology, Queen s University, 99 University Avenue, Kingston, Ontario, K7L 3N6, Canada
    Curr Atheroscler Rep 3:209-15. 2001
    ..Despite the documentation of associations between several genetic polymorphisms with plasma procoagulant levels, consistent associations with arterial thrombotic disease have not been found...
  9. ncbi Hemophilia treatment. Gene therapy, factor VIII antibodies and immune tolerance: hopes and concerns
    D Lillicrap
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    Haematologica 85:108-11; discussion 111-2. 2000
    ....
  10. ncbi Von Willebrand disease - phenotype versus genotype: deficiency versus disease
    David Lillicrap
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Canada
    Thromb Res 120:S11-6. 2007
    ..These results suggest that the molecular correlates for type 1 VWD are complex and, in addition to a wide array of changes at the VWF locus, are likely to involve mutations in other genes...
  11. ncbi Improvements in factor concentrates
    David Lillicrap
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Canada
    Curr Opin Hematol 17:393-7. 2010
    ..During the past 5 years, significant progress has been made with a variety of protein-engineering initiatives, some of which are already in early-phase clinical trials...
  12. ncbi The molecular basis of haemophilia B
    D Lillicrap
    Department of Pathology, Richardson Laboratory, Queen s University, Kingston, Ontario, Canada
    Haemophilia 4:350-7. 1998
    ..umds.ac.uk/molgen/haemBdatabase for a complete current listing of the mutations that cause this phenotype. In addition, other recent reviews have discussed complementary issues relating to this topic...
  13. ncbi Challenges in defining type 2M von Willebrand disease: results from a Canadian cohort study
    P D James
    Department of Medicine, Queen s University, Kingston, Ontario, Canada
    J Thromb Haemost 5:1914-22. 2007
    ..05 and 0.50 IU mL(-1) on at least two occasions and RCo/Ag ratio < 0.6 and no loss of high molecular weight multimers). Available family members (16 affected, 23 unaffected and six unknown) were sequenced for identified mutations...
  14. ncbi An assessment of the pathogenic significance of the R924Q von Willebrand factor substitution
    E Berber
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, K7L 3N6 Canada
    J Thromb Haemost 7:1672-9. 2009
    ..In this study, R924Q was detected in a compound heterozygote possessing both type 2N and 924Q substitutions whose VWF:FVIIIB and FVIII levels were disproportionately low for the heterozygous type 2N state...
  15. ncbi Variability of thromboelastographic responses following the administration of rFVIIa to haemophilia A dogs supports the individualization of therapy with a global test of haemostasis
    M Othman
    Department of Pathology and Molecular Medicine, Queen s University, and Clinical Research Centre, Kingston General Hospital, Kingston, Ontario, Canada
    Haemophilia 16:919-25. 2010
    ..Together, these data support the value of TEG not only as an effective monitoring haemostatic test, but also as a tool for individualization of therapy to achieve the best haemostatic and cost effectiveness of rFVIIa therapy...
  16. ncbi Reduction of the immune response to factor VIII mediated through tolerogenic factor VIII presentation by immature dendritic cells
    M Qadura
    Richardson Laboratory, Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada
    J Thromb Haemost 6:2095-104. 2008
    ..The development of neutralizing antibodies to factor FVIII (FVIII) represents the most serious complication in the treatment of hemophilia A...
  17. ncbi Immunoglobulin isotypes and functional anti-FVIII antibodies in response to FVIII treatment in Balb/c and C57BL/6 haemophilia A mice
    M Qadura
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, ON, Canada
    Haemophilia 17:288-95. 2011
    ..Therefore, genetic strain-dependent differences must be considered when evaluating immunological outcomes in mouse models of haemophilia A...
  18. ncbi Genetic linkage and association analysis in type 1 von Willebrand disease: results from the Canadian type 1 VWD study
    P D James
    Department of Medicine, Queen's University, Kingston, Canada
    J Thromb Haemost 4:783-92. 2006
    ..These studies provide further evidence to support the role for genetic loci other than VWF and ABO in the pathogenesis of type 1 VWD...
  19. ncbi The effect of exercise on von Willebrand factor and ADAMTS-13 in individuals with type 1 and type 2B von Willebrand disease
    J Stakiw
    Department of Medicine, Queen s University, Kingston, ON, Canada
    J Thromb Haemost 6:90-6. 2008
    ..The effect of exercise on von Willebrand factor (VWF) and ADAMTS-13 levels in individuals with von Willebrand disease (VWD) has never been reported...
  20. ncbi Undetected factor VIII in a patient with type 3 von Willebrands disease mistaken as severe haemophilia A
    A M Mullah-Ali
    Department of Pediatric Hematology Oncology, McMaster University, Hamilton, ON, Canada
    Haemophilia 15:1258-61. 2009
    ..A total absence of FVIII:C has never been reported in type 3 VWD. This case illustrates the effect of severe von Willebrand factor (VWF) deficiency on the factor VIII level...
  21. ncbi A novel type 2A (Group II) von Willebrand disease mutation (L1503Q) associated with loss of the highest molecular weight von Willebrand factor multimers
    L A O'Brien
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Ontario, Canada
    J Thromb Haemost 2:1135-42. 2004
    ..The mutation L1503Q does not significantly disrupt the conformation of the protein; thus the subtle loss of multimers in this patient may be due to altered interactions with the ADAMTS13 protease...
  22. ncbi Induction of partial immune tolerance to factor VIII through prior mucosal exposure to the factor VIII C2 domain
    F E Rawle
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, ON, Canada
    J Thromb Haemost 4:2172-9. 2006
    ..Based on these results, the potential use of FVIII-specific mucosal tolerance induction as an immunotherapy treatment for anti-FVIII inhibitor development warrants further investigation...
  23. ncbi Factors influencing therapeutic efficacy and the host immune response to helper-dependent adenoviral gene therapy in hemophilia A mice
    B D Brown
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada
    J Thromb Haemost 2:111-8. 2004
    ....
  24. ncbi Effect of the factor V Leiden mutation on the clinical expression of severe hemophilia A
    D H Lee
    Department of Medicine, Queen s University, Kingston, Ontario, Canada
    Thromb Haemost 83:387-91. 2000
    ....
  25. ncbi Influence of a GT repeat element on shear stress responsiveness of the VWF gene promoter
    C Hough
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, ON, Canada
    J Thromb Haemost 6:1183-90. 2008
    ..It is, however, unknown whether shear stress influences the regulation of VWF gene expression...
  26. ncbi Gene therapy for hemophilia: an imperative to succeed
    C Hough
    Department of Pathology and Molecular Medicine, Richardson Laboratories, Queen's University, Kingston, Ontario, Canada
    J Thromb Haemost 3:1195-205. 2005
  27. ncbi The molecular mechanisms of immunomodulation and tolerance induction to factor VIII
    B Waters
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada
    J Thromb Haemost 7:1446-56. 2009
    ..We also discuss methods to manipulate FVIII loading of antigen-presenting cells...
  28. ncbi DNA microarray analysis for the detection of mutations in hemophilia A
    E Berber
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada
    J Thromb Haemost 4:1756-62. 2006
    ..The methodology is, however, expensive and time consuming, and with the reduction in sequencing costs, direct sequencing is now the most cost and time efficient strategy for hemophilia A mutation analysis...
  29. ncbi A prospective evaluation of the prevalence of symptomatic von Willebrand disease (VWD) in a pediatric primary care population
    M Bowman
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Canada
    Pediatr Blood Cancer 55:171-3. 2010
    ..The prevalence of bleeding/bruising in a general pediatric population is 5%; the prevalence of symptomatic VWD at the level of pediatric primary care is at least 1 in 1,000...
  30. ncbi Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study
    B M Feldman
    Division of Rheumatology, Hospital for Sick Children, Toronto, ON, Canada
    J Thromb Haemost 4:1228-36. 2006
    ..We studied an inception cohort of 25 boys using a tailored prophylaxis approach to see if clotting factor use could be reduced with acceptable outcomes...
  31. ncbi The value of genetic testing for type 2B Von Willebrand disease
    K Roland
    Department of Pathology and Laboratory Medicine, Queen's University, Kingston, ON, Canada
    Clin Lab Haematol 28:17-21. 2006
    ..We report a case in which the diagnosis of VWD type 2B was made via genetic testing, illustrating its value as a useful diagnostic tool...
  32. ncbi Laboratory issues in bleeding disorders
    D Lillicrap
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada
    Haemophilia 12:68-75. 2006
    ....
  33. ncbi Generation and validation of the Condensed MCMDM-1VWD Bleeding Questionnaire for von Willebrand disease
    M Bowman
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada
    J Thromb Haemost 6:2062-6. 2008
    ..Given the challenges involved in obtaining accurate bleeding histories, attempts at standardization have occurred and the value of quantifying hemorrhagic symptoms has been recognized...
  34. ncbi Home management of haemophilia
    J M Teitel
    St Michael s Hospital, University of Toronto, Toronto, Ontario, Canada
    Haemophilia 10:118-33. 2004
    ..Gene therapy trials, which are currently ongoing, will ultimately lead to gene-based treatments as a complement to traditional protein-based therapy...
  35. ncbi Thromboelastography reflects global hemostatic variation among severe haemophilia A dogs at rest and following acute exercise
    M Othman
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, ON, Canada
    Haemophilia 15:1126-34. 2009
    ..The study supports the utilization of TEG in assessment of the hemostatic pattern in severe haemophilia A and provides a potential for utilizing TEG evaluation in managing exercise regimens for haemophilia care...
  36. ncbi Germ-line mosaicism for a valine-to-methionine substitution at residue 553 in the glycoprotein Ib-binding domain of von Willebrand factor, causing type IIB von Willebrand disease
    E W Murray
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    Am J Hum Genet 50:199-207. 1992
    ....
  37. ncbi Frequency of platelet type versus type 2B von Willebrand disease. An international registry-based study
    Alexander Hamilton
    Laurentian University, St Lawrence College Collaborative Program, Kingston, Ontario, Canada
    Thromb Haemost 105:501-8. 2011
    ..Cases that are negative for both VWF and GP1BA gene mutations require further evaluation for alternative diagnoses...
  38. ncbi Founder von Willebrand factor haplotype associated with type 1 von Willebrand disease
    Lee A O'Brien
    Department of Pathology, Queen s University, Kingston, ON, Canada
    Blood 102:549-57. 2003
    ..This is the first report of a mutation that segregates in a significant proportion of patients with type 1 VWD...
  39. ncbi The mutational spectrum of type 1 von Willebrand disease: Results from a Canadian cohort study
    Paula D James
    Department of Medicine, Queen s University, Kingston, ON, Canada K7L 3N6
    Blood 109:145-54. 2007
    ..In more severe cases, genetic changes are common within the VWF gene and are highly penetrant. In milder cases, the genetic determinants are more complex and involve factors outside of the VWF gene...
  40. ncbi In vitro and in vivo stability of diluted recombinant factor VIII for continuous infusion use in haemophilia A
    S Revel-Vilk
    Division of Haematology Oncology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ontario, Canada
    Haemophilia 16:72-9. 2010
    ..Indeed, stable FVIII levels were maintained when diluted KG-FS was administered by CI with the double-pump to a paediatric patient postsurgically...
  41. ncbi A novel type 2A von Willebrand factor mutation located at the last nucleotide of exon 26 (3538G>A) causes skipping of 2 nonadjacent exons
    Paula D James
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada K7L 3N6
    Blood 104:2739-45. 2004
    ..This is the first report of a coding region mutation resulting in the skipping of 2 nonadjacent exons...
  42. ncbi Identification and functional characterization of a novel 27-bp deletion in the macroglycopeptide-coding region of the GPIBA gene resulting in platelet-type von Willebrand disease
    Maha Othman
    Department of Pathology and Molecular Medicine, Queens University, Kingston, ON, Canada
    Blood 105:4330-6. 2005
    ..The mutation provides a molecular basis for the PT-VWD phenotype and supports a role for the macroglycopeptide region in receptor function...
  43. ncbi Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A
    Brian D Brown
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, ON, Canada K7L 3N6
    Blood 103:804-10. 2004
    ..This study supports previous demonstrations of improved safety using HD gene transfer and suggests that these vectors can provide transient FVIII expression with minimal, acute toxicity in the absence of inhibitor formation...
  44. ncbi Aberrant splicing and premature termination of transcription of the FVIII gene as a cause of severe canine hemophilia A: similarities with the intron 22 inversion mutation in human hemophilia
    Christine Hough
    The Department of Pathology, Queen's University, Kingston, Ontario, Canada
    Thromb Haemost 87:659-65. 2002
    ..The mutation mechanism may not be uncommon, as identical mutant transcripts were isolated from two hemophilia A littermates that are unrelated to the Queen's colony and from hemophiliac dogs in the colony at Chapel Hill...
  45. ncbi Enhanced binding of HLF/DBP heterodimers represents one mechanism of PAR protein transactivation of the factor VIII and factor IX genes
    M Begbie
    Department of Pathology, Queen s University, Kingston, Ontario, Canada
    DNA Cell Biol 18:165-73. 1999
    ..These observations further our understanding of the role played by members of the PAR family of transcription factors in regulating expression of the Factor VIII and Factor IX genes...
  46. ncbi The canine factor VIII cDNA and 5' flanking sequence
    C Cameron
    Department of Pathology, Queen s University, Richardson Laboratory, Kingston, Ontario, Canada
    Thromb Haemost 79:317-22. 1998
    ....
  47. ncbi Molecular modeling of the von Willebrand factor A2 Domain and the effects of associated type 2A von Willebrand disease mutations
    Jeffrey J Sutherland
    Department of Chemistry, Queen s University, K7L 3N6, Kingston, Ontario, Canada
    J Mol Model 10:259-70. 2004
    ..Figure: see text]. Homology model of the von Willebrand factor A2 domain..
  48. ncbi The role of molecular genetics in diagnosing von Willebrand disease
    Paula James
    Department of Medicine, Queen s University, Kingston, Canada
    Semin Thromb Hemost 34:502-8. 2008
    ..We have also provided guidelines as to how genetic testing can be used to clarify diagnostic uncertainty that might remain after a clinical evaluation and routine coagulation testing has been completed...
  49. ncbi Preclinical animal models for hemophilia gene therapy: predictive value and limitations
    Fiona E M Rawle
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada
    Semin Thromb Hemost 30:205-13. 2004
    ..This review presents a summary of the animal models available for hemophilia gene therapy, and highlights the various strengths and weaknesses of these models...
  50. ncbi Recombinant and plasma-derived factor VIII products induce distinct splenic cytokine microenvironments in hemophilia A mice
    Mohammad Qadura
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University, Kingston, Ontario, Canada
    Blood 114:871-80. 2009
    ..In summary, these studies report new mechanisms that contribute to reduced anti-FVIII antibody development in hemophilia A after pdFVIII infusions...
  51. ncbi The canine factor VIII 3'-untranslated region and a concatemeric hepatocyte nuclear factor 1 regulatory element enhance factor VIII transgene expression in vivo
    Colleen Notley
    Department of Pathology, Richardson Laboratories, Queen's University, Kingston, Ontario, Canada K7L 3N6
    Hum Gene Ther 13:1583-93. 2002
    ..Introduction of intron 17 proximal to the FVIII cDNA did not enhance in vivo expression of canine FVIII from the transgene...
  52. ncbi Aminoglycoside suppression of nonsense mutations in severe hemophilia
    Paula D James
    Department of Medicine, Queen's University, Kingston, Ontario, Canada K7L 3N6
    Blood 106:3043-8. 2005
    ..This study, however, does provide a proof of principle, suggesting that ribosomal interference with a less toxic agent may be a potential therapeutic mechanism for severe hemophilia patients with nonsense mutations...
  53. ncbi Heterogeneity of the immune response to adenovirus-mediated factor VIII gene therapy in different inbred hemophilic mouse strains
    Fiona E M Rawle
    Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada K7L 3N6
    J Gene Med 6:1358-68. 2004
    ....
  54. ncbi Cell type-specific regulation of von Willebrand factor expression by the E4BP4 transcriptional repressor
    Christine Hough
    The Department of Pathology and Molecular Medicine, Richardson Laboratories, Queen's University, Kingston, ON, Canada, K7L 3N6
    Blood 105:1531-9. 2005
    ....
  55. ncbi Functional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease
    Maha Othman
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Ontario, Canada
    Blood 116:3645-52. 2010
    ....
  56. ncbi Genetic testing for von Willebrand disease: the Canadian experience
    Paula James
    Department of Medicine, Queen's University, Kingston, Canada
    Semin Thromb Hemost 32:546-52. 2006
    ..In summary, the aim of this review is to prompt a careful consideration of how genetic testing can find an appropriate role as a complementary diagnostic modality for vWD...
  57. ncbi A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3+ T regulatory cells
    Hideto Matsui
    Department of Pathology and Molecular Medicine, Queen s University, Richardson Laboratory, 88 Stuart St, Kingston, Ontario, Canada
    Blood 114:677-85. 2009
    ..Finally, induction of FVIII tolerance mediated by this protocol is associated with a FVIII-expandable population of CD4(+)CD25(+)Foxp3(+) regulatory T cells...
  58. ncbi Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors
    Hideto Matsui
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Ontario, Canada
    Stem Cells 25:2660-9. 2007
    ..Disclosure of potential conflicts of interest is found at the end of this article...
  59. ncbi ADAMTS13 cleavage efficiency is altered by mutagenic and, to a lesser extent, polymorphic sequence changes in the A1 and A2 domains of von Willebrand factor
    Cynthia M Pruss
    Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada
    Br J Haematol 143:552-8. 2008
    ..Our two complementary assay conditions show that A-domain changes in VWF alter ADAMTS13-mediated proteolysis...
  60. ncbi Anti-CD3 prevents factor VIII inhibitor development in hemophilia A mice by a regulatory CD4+CD25+-dependent mechanism and by shifting cytokine production to favor a Th1 response
    Braden Waters
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, Ontario, Canada
    Blood 113:193-203. 2009
    ....
  61. ncbi Mutation-specific hemostatic variability in mice expressing common type 2B von Willebrand disease substitutions
    Mia Golder
    Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen s University Cancer Research Institute, Queen s University, Kingston, ON, Canada
    Blood 115:4862-9. 2010
    ..These defects were only partially rescued by normal platelet transfusions, thus emphasizing the key role of the abnormal plasma VWF environment in 2B VWD...
  62. ncbi Adenovirus-induced thrombocytopenia: the role of von Willebrand factor and P-selectin in mediating accelerated platelet clearance
    Maha Othman
    Department of Pathology and Molecular Medicine, Queen s University, Kingston, ON, Canada
    Blood 109:2832-9. 2007
    ..We conclude that VWF and P-selectin are critically involved in a complex platelet-leukocyte-endothelial interplay, resulting in platelet activation and accelerated platelet clearance following adenovirus administration...
  63. ncbi Characterization of viability and proliferation of alginate-poly-L-lysine-alginate encapsulated myoblasts using flow cytometry
    Ajit Thakur
    School of Biomedical Engineering, McMaster University, Hamilton L8N3Z5, Ontario, Canada
    J Biomed Mater Res B Appl Biomater 94:296-304. 2010
    ..In conclusion, we show that flow cytometry analysis allows for a more consistent and comprehensive examination of encapsulated cells to aid in the development of cell therapy protocols...
  64. ncbi Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
    Marinee K L Chuah
    Center for Transgene Technology and Gene Therapy, Flanders Interuniversity Institute for Biotechnology, University of Leuven, Belgium
    Blood 101:1734-43. 2003
    ....
  65. ncbi Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector
    Ciaran D Scallan
    Avigen, Inc, 1201 Harbor Bay Parkway, Alameda, CA 94502
    Blood 102:2031-7. 2003
    ..These data support the use of AAV2 vectors in human clinical trials to treat hemophilia A patients...
  66. ncbi Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
    Haiyan Jiang
    Avigen, Almeda, CA, USA
    Blood 108:107-15. 2006
    ..In summary, this is the first report demonstrating multiyear therapeutic efficacy and safety of multiple AAV-cFVIII vectors in hemophilia A dogs and provides the basis for human clinical studies...
  67. ncbi Efficacy and safety of a new-class hemostatic drug candidate, AV513, in dogs with hemophilia A
    Srinivasa Prasad
    Research and Development, Avigen, Alameda, CA 94502, USA
    Blood 111:672-9. 2008
    ..In summary, the combination of safety and efficacy in 2 global tests of hemostasis in the hemophilia A dog model indicate that further evaluation of AV513 as a hemostatic agent in hemophilia A patients is warranted...
  68. ncbi Inhibitor development in hemophiliacs: the roles of genetic versus environmental factors
    Christine A Lee
    Haemophilia Centre and Haemostasis Unit, Royal Free and University College Medical School, UCL, London, United Kingdom
    Semin Thromb Hemost 32:10-4. 2006
    ..There is much interest in identifying such genetic and treatment-related factors to help minimize the risk of inhibitor development and improve treatment outcomes...
  69. ncbi Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study
    Man Chiu Poon
    University of Calgary, Alberta, Canada
    Thromb Haemost 87:431-5. 2002
    ..No other serious adverse events, including thrombotic episodes, were reported. To the best of our knowledge, this is the first formal report of recovery and inhibitor formation on rFIX in a peer-reviewed manuscript form...
  70. ncbi Reliability and reproducibility of classification of children as "bleeders" versus "non-bleeders" using a questionnaire for significant mucocutaneous bleeding
    Iris Hedlund-Treutiger
    Karolinska Institute, Sach's Children's Hospital, , Stockholm, Sweden
    J Pediatr Hematol Oncol 26:488-91. 2004
    ..71). The validity and utility of the HSC questionnaire for primary screening of children with suspected mucocutaneous bleeding disorders merits assessment in further clinical studies...
  71. ncbi Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy
    Brian D Brown
    Department of Pathology, Queen's University, Kingston, Ontario, Canada
    Blood 100:1133-40. 2002
    ..In taking this perspective, we provide an alternative and complementary insight into some of the failures and successes of current gene therapy protocols...
  72. ncbi A 4% solution of bovine serum albumin may be used in place of factor VIII:C deficient plasma in the control sample in the Nijmegen Modification of the Bethesda factor VIII:C inhibitor assay
    Bert Verbruggen
    Thromb Haemost 88:362-4. 2002
  73. ncbi Correction of the bleeding time in von Willebrand factor (VWF)-deficient mice using murine VWF
    Peter J Lenting
    Blood 109:2267-8. 2007
  74. ncbi Von Willebrand: the scientist, the disease, the factor, and the treatment
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  75. ncbi Von Willebrand disease
    Jeremy Robertson
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    Pediatr Clin North Am 55:377-92, viii-ix. 2008
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  76. ncbi Efficient lentiviral transduction and improved engraftment of human bone marrow mesenchymal cells
    An Van Damme
    Center for Transgene Technology and Gene Therapy, Flanders Interuniversity Institute for Biotechnology, University of Leuven, Herestraat 49, Building O and N1, Leuven B-3000, Belgium
    Stem Cells 24:896-907. 2006
    ....
  77. ncbi Distinguishing between non-identical twins: platelet type and type 2B von Willebrand disease
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    Br J Haematol 138:665-6. 2007
  78. ncbi A survey of recommendations by gynecologists in Canada regarding oral contraceptive use in the perioperative period
    Jennifer Oakes
    Division of Reproductive Endocrinology, Department of Obstetrics and Gynaecology, Queen's University, Kingston, Ontario, Canada
    Am J Obstet Gynecol 187:1539-43. 2002
    ....