P E Monahan

Summary

Affiliation: University of North Carolina
Country: USA

Publications

  1. doi request reprint Gene therapy for hemophilia: advancing beyond the first clinical success
    Paul E Monahan
    Harold R Roberts Comprehensive Hemophilia Diagnostic and Treatment Center, University of North Carolina School of Medicine, Chapel Hill, North Carolina 27599, USA
    Curr Opin Hematol 20:410-6. 2013
  2. doi request reprint Physical functioning in boys with hemophilia in the U.S
    Paul E Monahan
    Department of Pediatrics, Hematology Oncology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7016, USA
    Am J Prev Med 41:S360-8. 2011
  3. pmc Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application
    Paul E Monahan
    Gene Therapy Center, University of North Carolina at Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 18:1907-16. 2010
  4. doi request reprint Recombinant factor IX for clinical and research use
    Paul E Monahan
    Department of Pediatrics, Gene Therapy Center, Harold R Roberts Comprehensive Hemophilia Treatment Center, University or North Carolina at Chapel Hill, Chapel Hill, NC 27599 7236, USA
    Semin Thromb Hemost 36:498-509. 2010
  5. doi request reprint Hemophilic synovitis: factor VII and the potential role of extravascular factor VIIa
    Paul E Monahan
    Pediatric Hematology Oncology, University of North Carolina at Chapel Hill, NC 27599, USA
    Thromb Res 125:S63-6. 2010
  6. ncbi request reprint Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B
    P E Monahan
    Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599 7352, USA
    Haemophilia 16:460-8. 2010
  7. ncbi request reprint Factor IX: Insights from knock-out and genetically engineered mice
    Paul E Monahan
    Department of Pediatrics, Hematology Oncology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC 27599 7352, USA
    Thromb Haemost 100:563-75. 2008
  8. ncbi request reprint Experimental animal use in the study of haemophilic bleeding
    P E Monahan
    Department of Pediatrics and Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    Haemophilia 14:112-6. 2008
  9. ncbi request reprint Neonatal immune tolerance for hemophilia: can we "tolerate" new paradigms for gene therapy trials?
    P E Monahan
    Department of Pediatrics and Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7220, USA
    J Thromb Haemost 5:1801-4. 2007
  10. ncbi request reprint Factors affecting choice of hemostatic agent for the hemophilia patient with an inhibitor antibody
    Paul E Monahan
    Department of Pediatrics, University of North Carolina at Chapel Hill, North Carolina, USA
    Am J Hematol 77:346-50. 2004

Collaborators

Detail Information

Publications28

  1. doi request reprint Gene therapy for hemophilia: advancing beyond the first clinical success
    Paul E Monahan
    Harold R Roberts Comprehensive Hemophilia Diagnostic and Treatment Center, University of North Carolina School of Medicine, Chapel Hill, North Carolina 27599, USA
    Curr Opin Hematol 20:410-6. 2013
    ..This review examines the successes and limitations of this clinical trial for hemophilia B and approaches to advance beyond this milestone...
  2. doi request reprint Physical functioning in boys with hemophilia in the U.S
    Paul E Monahan
    Department of Pediatrics, Hematology Oncology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7016, USA
    Am J Prev Med 41:S360-8. 2011
    ..Although the most frequent complication of repeated hemorrhages is a crippling joint disease that begins in childhood, the extent of resultant joint functional impairment varies widely within the hemophilia population...
  3. pmc Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application
    Paul E Monahan
    Gene Therapy Center, University of North Carolina at Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 18:1907-16. 2010
    ....
  4. doi request reprint Recombinant factor IX for clinical and research use
    Paul E Monahan
    Department of Pediatrics, Gene Therapy Center, Harold R Roberts Comprehensive Hemophilia Treatment Center, University or North Carolina at Chapel Hill, Chapel Hill, NC 27599 7236, USA
    Semin Thromb Hemost 36:498-509. 2010
    ..The experience with the existing recombinant FIX product is reviewed with a focus on the novel products and the potential to improve the quality of life for individuals with hemophilia B...
  5. doi request reprint Hemophilic synovitis: factor VII and the potential role of extravascular factor VIIa
    Paul E Monahan
    Pediatric Hematology Oncology, University of North Carolina at Chapel Hill, NC 27599, USA
    Thromb Res 125:S63-6. 2010
    ..The potential role of extravascular factor VIIa is considered...
  6. ncbi request reprint Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B
    P E Monahan
    Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599 7352, USA
    Haemophilia 16:460-8. 2010
    ..Safety was established by the low incidence of treatment-related adverse events...
  7. ncbi request reprint Factor IX: Insights from knock-out and genetically engineered mice
    Paul E Monahan
    Department of Pediatrics, Hematology Oncology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC 27599 7352, USA
    Thromb Haemost 100:563-75. 2008
    ....
  8. ncbi request reprint Experimental animal use in the study of haemophilic bleeding
    P E Monahan
    Department of Pediatrics and Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    Haemophilia 14:112-6. 2008
  9. ncbi request reprint Neonatal immune tolerance for hemophilia: can we "tolerate" new paradigms for gene therapy trials?
    P E Monahan
    Department of Pediatrics and Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7220, USA
    J Thromb Haemost 5:1801-4. 2007
  10. ncbi request reprint Factors affecting choice of hemostatic agent for the hemophilia patient with an inhibitor antibody
    Paul E Monahan
    Department of Pediatrics, University of North Carolina at Chapel Hill, North Carolina, USA
    Am J Hematol 77:346-50. 2004
    ..Specific knowledge deficits in need of prospective research were identified with respect to the rational treatment of inhibitor patients...
  11. ncbi request reprint Adeno-associated virus vectors for gene therapy: more pros than cons?
    P E Monahan
    Division of Hematology Oncology and Gene Therapy Center, University of North Carolina at Chapel Hill School of Medicine, CB 7352, Thurston Bowles Bldg, Chapel Hill, NC 27599, USA
    Mol Med Today 6:433-40. 2000
    ..This review examines a number of long-standing concerns regarding the utility of AAV for gene transfer in light of many new insights into the biology, immunology and production of AAV...
  12. ncbi request reprint Hemophilia gene therapy: update
    Paul E Monahan
    Department of Pediatrics, University of North Carolina at Chapel Hill, 418 MacNider Building, CB 7220 UNC CH, School of Medicine, Chapel Hill, North Carolina 27599 7220, USA
    Curr Opin Hematol 9:430-6. 2002
    ..The results of these trials indicate that gene transfer in hemophilia with the vectors and doses used is safe and well tolerated. Efforts continue to understand the basic biology and improve the efficiency of gene transfer...
  13. ncbi request reprint Safety of adeno-associated virus gene therapy vectors: a current evaluation
    Paul E Monahan
    Gene Therapy Center, CB 7220, University of North Carolina Chapel Hill School of Medicine, 418 MacNider Building, Chapel Hill, NC 27599, USA
    Expert Opin Drug Saf 1:79-91. 2002
    ....
  14. pmc Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX
    Junjiang Sun
    Gene Therapy Center, School of Medicine, University of North Carolina at Chapel Hill, NC 27599 7220, USA
    Blood 112:4532-41. 2008
    ..Extravascular factor activity and joint-directed gene transfer may ameliorate hemophilic joint destruction, even in the absence of circulating FIX...
  15. ncbi request reprint Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
    D M McCarty
    UNC Gene Therapy Center, University of North Carolina at Chapel Hill, NC, USA
    Gene Ther 8:1248-54. 2001
    ..These novel scAAV vectors represent a biochemical intermediate in rAAV transduction and should provide new insights into the biology of vector transduction...
  16. ncbi request reprint Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors
    H Chao
    Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599, USA
    Mol Ther 4:217-22. 2001
    ..Finally, we report that correction of the hemophilia phenotype using AAV1-F9 was complete and persistent (over 8 months), a result that underscores the value of continued exploration of alternative AAV serotype vectors...
  17. ncbi request reprint Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice
    T P Zhang
    The Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7220, USA
    Gene Ther 14:429-40. 2007
    ..This genotype/phenotype is strongly protective against antibody formation in response to FIX therapy...
  18. doi request reprint Abnormal hemostasis in a knock-in mouse carrying a variant of factor IX with impaired binding to collagen type IV
    T Gui
    Department of Biology, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    J Thromb Haemost 7:1843-51. 2009
    ..Factor IX binds to collagen type IV, but this binding has no known consequence. Objectives: To determine the effect of reduced binding of FIX to collagen IV...
  19. ncbi request reprint Creation of a mouse expressing defective human factor IX
    Da Yun Jin
    Gene Therapy Center, Department of Biology, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    Blood 104:1733-9. 2004
    ..In contrast, given the same treatment, FIXKO mice consistently develop antibodies. Our R333Q-hFIX mice strain will complement the FIXKO mice for studying factor IX circulating kinetics and gene therapy...
  20. pmc IL-6 receptor antagonist as adjunctive therapy with clotting factor replacement to protect against bleeding-induced arthropathy in hemophilia
    N Narkbunnam
    Pediatric Hematology, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    J Thromb Haemost 11:881-93. 2013
    ..Specific therapies to oppose inflammatory cytokines, including Interleukin 6 (IL-6) receptor antagonists, have become important in the management of inflammatory arthritides...
  21. doi request reprint Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
    C Li
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    Gene Ther 19:288-94. 2012
    ..The results may guide rational design of clinical trials using alternative AAV serotypes and suggest that younger patients who are given AAV gene therapy will benefit from the lower prevalence of NAbs...
  22. ncbi request reprint Off-label use of recombinant factor VIIa in patients following bone marrow transplantation
    J Blatt
    Division of Pediatric Hematology-Oncology, University of North Carolina School of Medicine, Chapel Hill, NC 27599, USA
    Bone Marrow Transplant 28:405-7. 2001
    ....
  23. ncbi request reprint Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose
    Zhijian Wu
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:280-9. 2008
    ....
  24. ncbi request reprint In vivo response to vascular injury in the absence of factor IX: examination in factor IX knockout mice
    Tong Gui
    Department of Biology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    Thromb Res 121:225-34. 2007
    ..In vivo modeling is possible because of the generation of factor IX(-/-) mice...
  25. ncbi request reprint Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    D M McCarty
    Division of Drug Delivery and Disposition, School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7352, USA
    Gene Ther 10:2112-8. 2003
    ..The scAAV is unaffected by this barrier, and provides an extremely efficient vector for gene transfer into many types of cells in vivo...
  26. pmc Familial deficiency of vitamin K-dependent clotting factors
    B W Weston
    The Harold R Roberts Comprehensive Hemophilia Treatment Center, Department of Pediatrics, Division of Hematology Oncology, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7220, USA
    Haemophilia 14:1209-13. 2008
    ..The results may also provide potential targets for molecular therapeutics and pharmacogenetics...
  27. ncbi request reprint Thrombosis in children: current perspective and distinct challenges
    Matthew W Richardson
    Department of Pediatrics, University of North Carolina at Chapel Hill 27599, USA
    Thromb Haemost 88:900-11. 2002
    ..This approach, however, results in diagnostic and therapeutic pitfalls. An understanding of issues unique to pediatric thrombosis is required; recent insights and the ongoing challenges are reviewed...
  28. pmc Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection
    Boris Kantor
    Gene Therapy Center, University of North Carolina School of Medicine, Chapel Hill, NC 27514, USA
    Proc Natl Acad Sci U S A 106:18786-91. 2009
    ..Finally, we propose a mechanism describing the role of episomal HIV-1 forms in the viral life cycle in a SCFA-rich gut environment...