Research Topics
| P E MonahanSummaryAffiliation: University of North Carolina Country: USA Publications
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Detail Information
Publications
Physical functioning in boys with hemophilia in the U.SPaul 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...
Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical applicationPaul E Monahan
Gene Therapy Center, University of North Carolina at Chapel Hill, North Carolina 27599 7352, USA
Mol Ther 18:1907-16. 2010....- Recombinant factor IX for clinical and research usePaul 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... - Hemophilic synovitis: factor VII and the potential role of extravascular factor VIIaPaul 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... - Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia BP 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... 
Factor IX: Insights from knock-out and genetically engineered micePaul 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....- Experimental animal use in the study of haemophilic bleedingP 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 - 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... - 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 - Factors affecting choice of hemostatic agent for the hemophilia patient with an inhibitor antibodyPaul 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... - Hemophilia gene therapy: updatePaul 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... - Safety of adeno-associated virus gene therapy vectors: a current evaluationPaul 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.... - Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IXJunjiang 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... - Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesisD 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... - Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectorsH 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... - Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant miceT 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... - Abnormal hemostasis in a knock-in mouse carrying a variant of factor IX with impaired binding to collagen type IVT 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... - Creation of a mouse expressing defective human factor IXDa 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... - Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophiliaC 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... - Off-label use of recombinant factor VIIa in patients following bone marrow transplantationJ 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.... - In vivo response to vascular injury in the absence of factor IX: examination in factor IX knockout miceTong 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... - Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector doseZhijian Wu
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
Mol Ther 16:280-9. 2008.... - Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivoD 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... - Familial deficiency of vitamin K-dependent clotting factorsB 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... - Thrombosis in children: current perspective and distinct challengesMatthew 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... - Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infectionBoris 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...