Catherine Manno

Summary

Affiliation: The Children's Hospital of Philadelphia
Country: USA

Publications

  1. ncbi request reprint Gene therapy for bleeding disorders
    Catherine S Manno
    University of Pennsylvania School of Medicine, Philadelphia, USA
    Curr Opin Hematol 9:511-5. 2002
  2. ncbi request reprint AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    Catherine S Manno
    Department of Pediatrics, University of Pennsylvania and The Children s Hospital of Philadelphia, PA, 19104, USA
    Blood 101:2963-72. 2003
  3. ncbi request reprint The promise of third-generation recombinant therapy and gene therapy
    Catherine S Manno
    Department of Pediatrics, University of Pennsylvania School of Medicine, and the Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Semin Hematol 40:23-8. 2003
  4. pmc Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome
    David T Teachey
    Pediatric Hematology and Oncology, Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA
    Br J Haematol 145:101-6. 2009
  5. ncbi request reprint May-Thurner syndrome (iliac vein compression) and thrombosis in adolescents
    Leslie Raffini
    Department of Pediatrics, Division of Hematology, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104 4399, USA
    Pediatr Blood Cancer 47:834-8. 2006
  6. doi request reprint Treatment with sirolimus ameliorates tacrolimus-induced autoimmune cytopenias after solid organ transplant
    David T Teachey
    Division of Hematology, Children s Hospital of Philadelphia, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
    Pediatr Blood Cancer 53:1114-6. 2009
  7. doi request reprint The clinical management of hemophilia and head trauma: a survey of current clinical practice among pediatric hematology/oncology physicians
    Char M Witmer
    Division of Hematology, Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA
    Pediatr Blood Cancer 53:406-10. 2009
  8. doi request reprint Incidence of bleeding complications in pediatric patients with type 1 von Willebrand disease undergoing adenotonsillar procedures
    Char M Witmer
    Department of Pediatrics, Division of Hematology, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA
    J Pediatr 155:68-72. 2009
  9. pmc Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS)
    David T Teachey
    Divisions of Oncology and Hematology, Children s Hospital of Philadelphia, ARC 902, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA
    Blood 108:1965-71. 2006
  10. ncbi request reprint Cystic fibrosis as a risk factor for recurrent venous thrombosis at a pediatric tertiary care hospital
    Leslie J Raffini
    Division of Hematology, The Children s Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia 19104 4399, USA
    J Pediatr 148:659-64. 2006

Detail Information

Publications21

  1. ncbi request reprint Gene therapy for bleeding disorders
    Catherine S Manno
    University of Pennsylvania School of Medicine, Philadelphia, USA
    Curr Opin Hematol 9:511-5. 2002
    ..Multiple approaches to gene transfer in the hemophilias are currently under investigation...
  2. ncbi request reprint AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    Catherine S Manno
    Department of Pediatrics, University of Pennsylvania and The Children s Hospital of Philadelphia, PA, 19104, USA
    Blood 101:2963-72. 2003
    ....
  3. ncbi request reprint The promise of third-generation recombinant therapy and gene therapy
    Catherine S Manno
    Department of Pediatrics, University of Pennsylvania School of Medicine, and the Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Semin Hematol 40:23-8. 2003
    ..This article reviews the results of recent clinical studies investigating third-generation recombinant products and gene-based approaches to hemophilia treatment...
  4. pmc Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome
    David T Teachey
    Pediatric Hematology and Oncology, Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA
    Br J Haematol 145:101-6. 2009
    ..Based on these significant responses, we recommend that sirolimus be considered as second-line therapy for patients with steroid-refractory disease...
  5. ncbi request reprint May-Thurner syndrome (iliac vein compression) and thrombosis in adolescents
    Leslie Raffini
    Department of Pediatrics, Division of Hematology, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104 4399, USA
    Pediatr Blood Cancer 47:834-8. 2006
    ..Though uncommon, pediatric hematologists should consider May-Thurner syndrome in adolescents who present with a left lower extremity thrombosis. Aggressive therapy may be warranted due to the risk of post-thrombotic syndrome...
  6. doi request reprint Treatment with sirolimus ameliorates tacrolimus-induced autoimmune cytopenias after solid organ transplant
    David T Teachey
    Division of Hematology, Children s Hospital of Philadelphia, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA
    Pediatr Blood Cancer 53:1114-6. 2009
    ..Altering the immunosuppressive regimen to modify T-cell dysregulation may be beneficial for patients who develop post-transplant autoimmune disease and allow continued preservation of allograft...
  7. doi request reprint The clinical management of hemophilia and head trauma: a survey of current clinical practice among pediatric hematology/oncology physicians
    Char M Witmer
    Division of Hematology, Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA
    Pediatr Blood Cancer 53:406-10. 2009
    ..Determining the appropriate evaluation for a pediatric patient with hemophilia and head trauma is a diagnostic challenge with no neuroimaging guidelines and limited clinical evidence to direct care...
  8. doi request reprint Incidence of bleeding complications in pediatric patients with type 1 von Willebrand disease undergoing adenotonsillar procedures
    Char M Witmer
    Department of Pediatrics, Division of Hematology, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA
    J Pediatr 155:68-72. 2009
    ..To review the incidence of postoperative bleeding in children with type 1 von Willebrand disease (VWD) who were treated with a single institution protocol...
  9. pmc Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS)
    David T Teachey
    Divisions of Oncology and Hematology, Children s Hospital of Philadelphia, ARC 902, 3615 Civic Center Boulevard, Philadelphia, PA 19104, USA
    Blood 108:1965-71. 2006
    ..We conclude that rapamycin is an effective treatment for murine ALPS and should be explored as treatment for affected humans...
  10. ncbi request reprint Cystic fibrosis as a risk factor for recurrent venous thrombosis at a pediatric tertiary care hospital
    Leslie J Raffini
    Division of Hematology, The Children s Hospital of Philadelphia, Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia 19104 4399, USA
    J Pediatr 148:659-64. 2006
    ..To evaluate risk factors for recurrent thrombosis in pediatric patients...
  11. ncbi request reprint Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    Catherine S Manno
    The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania, 19104, USA
    Nat Med 12:342-7. 2006
    ..We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression...
  12. ncbi request reprint HIV infection presenting as severe autoimmune hemolytic anemia with disseminated intravascular coagulation in an infant
    Susan R Rheingold
    Divisions of Oncology, Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA
    J Pediatr Hematol Oncol 26:9-12. 2004
    ..The authors describe a 5-month-old infant who presented with severe autoimmune hemolytic anemia and disseminated intravascular coagulation as his initial manifestation of HIV infection...
  13. doi request reprint Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States
    Char Witmer
    Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, USA
    Br J Haematol 152:211-6. 2011
    ..This is the first study to demonstrate that prescribed prophylaxis conferred a protective effect against ICH in patients with uncomplicated severe disease...
  14. ncbi request reprint Modern management of haemophilic arthropathy
    Leslie Raffini
    Division of Hematology, Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, PA 19104 4399, USA
    Br J Haematol 136:777-87. 2007
    ..Patients with high titre inhibitors are at great risk for the development of joint disease and present the greatest therapeutic challenges when joint surgery is needed...
  15. doi request reprint A prospective observational study of IVC filters in pediatric patients
    Leslie Raffini
    Division of Hematology, Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA
    Pediatr Blood Cancer 51:517-20. 2008
    ..Both permanent and retrievable filters have been used in the pediatric population, though reports describing such patients and their outcomes are limited...
  16. ncbi request reprint Unmasking Evans syndrome: T-cell phenotype and apoptotic response reveal autoimmune lymphoproliferative syndrome (ALPS)
    David T Teachey
    Division of Oncology, Department of Pediatrics, Children s Hospital of Philadelphia, University of Pennsylvania School of Medicine, ARC 902, 3615 Civic Center Blvd, Philadelphia, PA 19104, USA
    Blood 105:2443-8. 2005
    ..Our data further suggest that a number of patients with ES may have ALPS, a novel finding with important therapeutic implications...
  17. ncbi request reprint CD8(+) T-cell responses to adeno-associated virus capsid in humans
    Federico Mingozzi
    The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA
    Nat Med 13:419-22. 2007
    ..In addition, we show that AAV-2 induced human T cells proliferate upon exposure to alternate AAV serotypes, indicating that other serotypes are unlikely to evade capsid-specific immune responses...
  18. ncbi request reprint Therapeutic choices in the current millennium: hemophilia workshop highlights
    Prasad Mathew
    Department of Pediatrics, University of New Mexico, Albuquerque, NM 87131, USA
    Pediatr Blood Cancer 45:611-5. 2005
    ..This paper provides a summary of the discussions at these workshops. Despite the progress made in the management of patients with hemophilia, the workshop highlighted the fact that there were still many unanswered questions...
  19. ncbi request reprint Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B
    Haiyan Jiang
    Avigen, Inc, Alameda, CA 94502, USA
    Mol Ther 14:452-5. 2006
    ..These results demonstrate, for the first time, multiyear FIX expression by AAV2 vector in humans and suggest that improved muscle delivery provides effective treatment for protein deficiencies or muscle-specific diseases...
  20. doi request reprint Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia
    Paul Harmatz
    Children s Hospital and Research Center Oakland, 747 52 Street, Oakland, CA 94609, USA
    Haematologica 93:1247-51. 2008
    ..The molar efficacy of deferoxamine improved with reduction in liver inflammation on biopsy (p=0.001). In conclusion, antiviral treatment is safe if transfusion requirement, iron toxicities and neutropenia are monitored...
  21. ncbi request reprint Guidelines for assessing appropriateness of pediatric transfusion
    Susan D Roseff
    Department of Pathology, Virginia Commonwealth University School of Medicine, Richmond, Virginia 23298 0662, USA
    Transfusion 42:1398-413. 2002