K High

Summary

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

Publications

  1. doi request reprint AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects
    N C Hasbrouck
    Department of Pediatrics, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Gene Ther 15:870-5. 2008
  2. pmc Gene therapy for haemophilia
    Samuel L Murphy
    Department of Pediatrics, Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Br J Haematol 140:479-87. 2008
  3. pmc Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs
    Virginia Haurigot
    Division of Hematology and Center for Cellular and Molecular Therapeutics, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 18:1318-29. 2010
  4. pmc Cellular localization and characterization of cytosolic binding partners for Gla domain-containing proteins PRRG4 and PRRG2
    Mustafa N Yazicioglu
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    J Biol Chem 288:25908-14. 2013
  5. pmc A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X
    S J Tai
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    J Thromb Haemost 6:339-45. 2008
  6. pmc In vivo genome editing restores haemostasis in a mouse model of haemophilia
    Hojun Li
    Division of Hematology, CTRB 5000, Children s Hospital of Philadelphia, 3501 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA
    Nature 475:217-21. 2011
  7. pmc The gene therapy journey for hemophilia: are we there yet?
    Katherine A High
    Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Blood 120:4482-7. 2012
  8. doi request reprint The gene therapy journey for hemophilia: are we there yet?
    Katherine A High
    Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Hematology Am Soc Hematol Educ Program 2012:375-81. 2012
  9. ncbi request reprint Gene transfer as an approach to treating hemophilia
    Katherine A High
    University of Pennsylvania School of Medicine, Hematology Division, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA
    Semin Thromb Hemost 29:107-20. 2003
  10. pmc The leak stops here: platelets as delivery vehicles for coagulation factors
    Katherine A High
    Howard Hughes Medical Institute, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    J Clin Invest 116:1840-2. 2006

Research Grants

Collaborators

Detail Information

Publications58

  1. doi request reprint AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects
    N C Hasbrouck
    Department of Pediatrics, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Gene Ther 15:870-5. 2008
    ....
  2. pmc Gene therapy for haemophilia
    Samuel L Murphy
    Department of Pediatrics, Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Br J Haematol 140:479-87. 2008
    ..The progress and problems of gene therapies for haemorrhagic disorders will be discussed. This review will concentrate on approaches in or near clinical application...
  3. pmc Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs
    Virginia Haurigot
    Division of Hematology and Center for Cellular and Molecular Therapeutics, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 18:1318-29. 2010
    ..In summary, this study in a large animal model of HB demonstrates that therapeutic levels of gene transfer can be safely achieved using a novel route of intravascular gene transfer to muscle...
  4. pmc Cellular localization and characterization of cytosolic binding partners for Gla domain-containing proteins PRRG4 and PRRG2
    Mustafa N Yazicioglu
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    J Biol Chem 288:25908-14. 2013
    ..Several of the PRRG-interacting proteins we identified are essential for a variety of physiologic processes. Our findings indicate possible novel and previously unidentified functions for PRRG proteins. ..
  5. pmc A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X
    S J Tai
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    J Thromb Haemost 6:339-45. 2008
    ..There are no reports of humans with complete deficiency of FX, and knockout of murine F10 is embryonic or perinatal lethal...
  6. pmc In vivo genome editing restores haemostasis in a mouse model of haemophilia
    Hojun Li
    Division of Hematology, CTRB 5000, Children s Hospital of Philadelphia, 3501 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA
    Nature 475:217-21. 2011
    ..Thus, ZFN-driven gene correction can be achieved in vivo, raising the possibility of genome editing as a viable strategy for the treatment of genetic disease...
  7. pmc The gene therapy journey for hemophilia: are we there yet?
    Katherine A High
    Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Blood 120:4482-7. 2012
    ..Efforts to extend AAV-mediated gene therapy to hemophilia A, and alternate approaches that may be useful for persons with severe liver disease, who may not be candidates for gene transfer to liver, are also discussed...
  8. doi request reprint The gene therapy journey for hemophilia: are we there yet?
    Katherine A High
    Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Hematology Am Soc Hematol Educ Program 2012:375-81. 2012
    ..Efforts to extend AAV-mediated gene therapy to hemophilia A, and alternate approaches that may be useful for persons with severe liver disease, who may not be candidates for gene transfer to liver, are also discussed...
  9. ncbi request reprint Gene transfer as an approach to treating hemophilia
    Katherine A High
    University of Pennsylvania School of Medicine, Hematology Division, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA
    Semin Thromb Hemost 29:107-20. 2003
    ..The goal of the ongoing clinical studies is to determine whether these results can safely be extended to humans...
  10. pmc The leak stops here: platelets as delivery vehicles for coagulation factors
    Katherine A High
    Howard Hughes Medical Institute, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    J Clin Invest 116:1840-2. 2006
    ..Earlier clinical experience with gene transfer into hematopoietic cells highlighted the potential safety risks of this approach, but an F8 transgene may represent a lower risk than transgenes for growth factors or their receptors...
  11. pmc The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis
    Katherine A High
    3615 Civic Center Boulevard, 302D Abramson Pediatric Research Center, Philadelphia, PA 19104, USA
    Trans Am Clin Climatol Assoc 120:331-59. 2009
    ..This lecture will demonstrate the interconnected nature of progress in these two areas, as careful delineation of the obstacles in hemophilia led to the realization that success could be achieved in Leber's...
  12. ncbi request reprint Clinical gene transfer studies for hemophilia B
    Katherine A High
    Howard Hughes Medical Institute, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    Semin Thromb Hemost 30:257-67. 2004
    ..The safety and efficacy data established in the first trial formed the basis for a second trial in which AAV-FIX is administered systemically to target the liver. The liver study is currently ongoing, with six patients enrolled to date...
  13. pmc Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model
    Valder R Arruda
    The Children s Hospital of Philadelphia, 3615 Civic Center Blvd, 302 Abramson Research Center, Philadelphia, PA 19104, USA
    Blood 105:3458-64. 2005
    ..These findings also have immediate relevance for gene transfer in patients with muscular dystrophy...
  14. ncbi request reprint Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?
    K High
    Abramson Pediatric Research Center, Civic Center Blvd, Philadelphia, PA 19104, USA
    J Thromb Haemost 3:1682-91. 2005
    ..Studies in the next few years will determine whether the problems identified in preclinical and early phase clinical testing can be solved to develop a therapeutic gene transfer approach to hemophilia...
  15. doi request reprint rAAV human trial experience
    Katherine A High
    Howard Hughes Medical Institute, Philadelphia, PA, USA
    Methods Mol Biol 807:429-57. 2011
    ....
  16. ncbi request reprint Adeno-associated virus-mediated gene transfer for hemophilia B
    Katherine A High
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    Int J Hematol 76:310-8. 2002
    ..This goal has already been achieved in the hemophilia B dog model; the ongoing study will determine whether a similar result can be achieved in humans with hemophilia B...
  17. ncbi request reprint Gene-based approaches to the treatment of hemophilia
    Katherine High
    The Children s Hospital of Philadelphia, 3516 Civic Center Boulevard, Philadelphia, PA 19104, USA
    Ann N Y Acad Sci 961:63-4. 2002
  18. ncbi request reprint AAV-mediated gene transfer for hemophilia
    K A High
    Department of Pediatrics, University of Pennsylvania School of Medicine, The Children s Hospital of Philadelphia, 19104, USA
    Ann N Y Acad Sci 953:64-74. 2001
    ..The goal of dose escalation is to find a dose that is nontoxic but that results in circulating levels of factor IX >1% in all patients...
  19. ncbi request reprint Gene transfer as an approach to treating hemophilia
    K A High
    University of Pennsylvania School of Medicine and Hematology Division, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Circ Res 88:137-44. 2001
    ..This report reviews the preclinical data underlying these strategies and the design of the ongoing and proposed clinical trials...
  20. ncbi request reprint Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males
    V R Arruda
    The Children's Hospital of Philadelphia, and Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 4:586-92. 2001
    ..We conclude that the risk of inadvertent germline transmission of vector sequences following IM or hepatic artery injection of AAV-2 vectors is extremely low...
  21. ncbi request reprint Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
    P A Fields
    Department of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA 19104, USA
    Mol Ther 4:201-10. 2001
    ..These data have direct relevance for design of clinical trials and strategies aimed at avoiding immune responses against a secreted transgene product...
  22. ncbi request reprint Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
    R W Herzog
    Department of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA 19104, USA
    Mol Ther 4:192-200. 2001
    ..Treatment with this combination of gene transfer and transient immune modulation has resulted in sustained expression (>8 months) of canine F9 at levels sufficient for partial correction of coagulation parameters...
  23. ncbi request reprint Posttranslational modifications of recombinant myotube-synthesized human factor IX
    V R Arruda
    Department of Pediatrics and Pathology, University of Pennsylvania Medical Center, Philadelphia, Pennsylvania, USA
    Blood 97:130-8. 2001
    ..In vivo experiments in mice showed that these differences affect recovery but not half-life of muscle-synthesized F.IX...
  24. ncbi request reprint Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice
    P A Fields
    Departments of Pediatrics and Pathology, University of Pennsylvania Medical Center and The Children's Hospital of Philadelphia, Philadelphia, PA, USA
    Gene Ther 8:354-61. 2001
    ..Given the well-documented immunogenicity of the first-generation adenoviral vector, data from C57BL/6 mice may therefore grossly underestimate immunological consequences in certain gene therapy protocols...
  25. ncbi request reprint Gene therapy: a 2001 perspective
    K A High
    The Children's Hospital of Philadelphia, Philadelphia, PA 19104-4318, USA
    Haemophilia 7:23-7. 2001
    ..In preclinical studies, this strategy has produced therapeutic levels of circulating factor IX in haemophilic mice and dogs...
  26. pmc A mutation in the propeptide of Factor IX leads to warfarin sensitivity by a novel mechanism
    K Chu
    Department of Pediatrics and Pathology, University of Pennslyvania and Children s Hospital of Philadelphia, 19104, USA
    J Clin Invest 98:1619-25. 1996
    ..These studies delineate a novel mechanism for warfarin sensitivity. In addition, the data may also explain the observation that bone Gla protein is more sensitive to warfarin than the coagulation proteins...
  27. ncbi request reprint Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes
    Hua Li
    Wistar Institute, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 15:792-800. 2007
    ....
  28. 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...
  29. ncbi request reprint Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice
    Denise E Sabatino
    Department of Hematology, Children s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 15:1677-85. 2007
    ..IX expression. This supports the concept of a narrow "window of opportunity" for tolerance induction...
  30. pmc Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
    Federico Mingozzi
    Division of Hematology and Center for Cellular and Molecular Therapeutics, The Children s Hospital of Philadelphia, PA 19104, USA
    Blood 110:2334-41. 2007
    ....
  31. pmc A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin
    Chunmei Yang
    Department of Molecular Genetics, City of Hope National Medical Center, Duarte, CA 91010, USA
    Proc Natl Acad Sci U S A 104:15394-9. 2007
    ..Furthermore, geneticin, its metabolites, or better tolerated analogues should be evaluated as a general treatment with multiday response for severe genetic disease caused by nonsense mutation...
  32. pmc Safety and efficacy of gene transfer for Leber's congenital amaurosis
    Albert M Maguire
    Scheie Eye Institute, University of Pennsylvania, USA
    N Engl J Med 358:2240-8. 2008
    ..Although the follow-up was very short and normal vision was not achieved, this study provides the basis for further gene therapy studies in patients with LCA...
  33. ncbi request reprint Immune responses to AAV in clinical trials
    Federico Mingozzi
    Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Curr Gene Ther 7:316-24. 2007
    ....
  34. ncbi request reprint Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle
    Alice Toromanoff
    INSERM UMR 649, CHU Nantes, Faculte de Medecine, Universite de Nantes, Nantes, France
    Mol Ther 16:1291-9. 2008
    ..However, regardless of the mode of delivery, concerns continue to be raised by the presence of vector sequences detected at distant sites...
  35. pmc Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
    Jeannette Bennicelli
    Department of Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania 19104 6069, USA
    Mol Ther 16:458-65. 2008
    ..The data demonstrate that AAV2.RPE65 delivers the RPE65 transgene efficiently and quickly to the appropriate target cells in vivo in animal models. This vector holds great promise for treatment of LCA due to RPE65 mutations...
  36. 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...
  37. pmc Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII
    Paris Margaritis
    Division of Hematology, The Children s Hospital of Philadelphia, Abramson Research Center, Philadelphia, Pennsylvania 19104, USA
    J Clin Invest 113:1025-31. 2004
    ..These data hold promise for a potential treatment for hemophilia and other bleeding disorders...
  38. ncbi request reprint Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1
    Valder R Arruda
    Department of Pediatrics, University of Pennsylvani Medical Center, The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Blood 103:85-92. 2004
    ..ix...
  39. ncbi request reprint Coaxing coagulation with RNA and cell fragments
    Katherine A High
    Nat Med 9:991-2. 2003
  40. pmc Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia
    Katherine A High
    Trans Am Clin Climatol Assoc 114:337-51; discussion 351-2. 2003
    ..Based on these and other safety studies, the clinical trial has now resumed. A goal of this work will be to determine whether the therapeutic levels achieved in a large animal model of hemophilia can be realized in humans...
  41. pmc Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
    Federico Mingozzi
    Department of Pediatrics, The Children s Hospital of Philadelphia, University of Pennsylvania Medical Center, Philadelphia, Pennsylvania 19104, USA
    J Clin Invest 111:1347-56. 2003
    ..With a strain-dependent rate of success, tolerance to murine F.IX was induced in mice with a large F.IX gene deletion, supporting the relevance of these data for treatment of hemophilia B and other genetic diseases...
  42. 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
    ....
  43. pmc Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector
    Federico Mingozzi
    Department of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA 19104, USA
    J Virol 76:10497-502. 2002
    ..The subpopulations of hepatocytes transduced with either vector widely overlap, with the AAV-5 vector transducing additional hepatocytes and showing a wider area of transgene expression throughout the liver parenchyma...
  44. ncbi request reprint Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
    Roland W Herzog
    Department of Pediatrics and Pathology, University of Pennsylvania Medical Center and Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Hum Gene Ther 13:1281-91. 2002
    ..IX antigen, indicating a helper T cell-dependent mechanism. Anti-cF.IX formation is likely influenced by the extent of local antigen presentation and may be avoided by limited vector doses or by transient immune modulation...
  45. ncbi request reprint Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene
    Denise E Sabatino
    Department of Pediatrics, Graduate Program in Gene Therapy, University of Pennsylvania School of Medicine, Philadelphia, USA
    Blood 104:2767-74. 2004
    ..These new mouse models faithfully mimic the mutations causing human disease, and will prove useful for testing novel therapies for hemophilia...
  46. ncbi request reprint Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector
    Yi Lin Liu
    Department of Pediatrics, Children s Hospital of Philadelphia and University of Pennsylvania Medical Center, Philadelphia, PA 19104, USA
    Hum Gene Ther 15:783-92. 2004
    ..IX transgene product in mice with F.IX gene deletion, indicating that the risk of humoral immune responses remains in the context of an immunologically unfavorable mutation...
  47. ncbi request reprint Genetic analysis of the antibody response to AAV2 and factor IX
    Huang Ge Zhang
    Division of Clinical Immunology and Rheumatology, Department of Medicine, University of Alabama at Birmingham, 701 South 19th Street, LHRB 473, Birmingham, AL 35294, USA
    Mol Ther 11:866-74. 2005
    ..66) for anti-cF.IX. These results indicate that multiple genetic loci independently regulate the isotype-specific antibody response to the AAV2 capsid and the cF.IX transgene...
  48. pmc Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor IX
    Ou Cao
    Department of Pediatrics, Division of Cellular and Molecular Therapy, University of Florida, Alachua, 32615, USA
    Blood 108:480-6. 2006
    ..This was achieved through immune deviation to a T-helper-cell response with increased IL-10 and TGF-beta production and activation of regulatory CD4(+)CD25(+) T cells...
  49. 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...
  50. ncbi request reprint Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
    Jane D Mount
    Scott Ritchey Research Center and Department of Clinical Sciences, College of Veterinary Sciences, Auburn University, AL, USA
    Blood 99:2670-6. 2002
    ..This study demonstrates that hepatic AAV gene transfer can result in sustained therapeutic expression in a large animal model characterized by increased risk of a neutralizing anti-FIX response...
  51. ncbi request reprint Advances in gene therapy using factor VIIa in hemophilia
    Paris Margaritis
    The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Semin Hematol 43:S101-4. 2006
    ..These studies are expanding our knowledge of the effects of continuously expressed rFVIIa, and it is hoped that they may eventually provide a new avenue for treatment of hemophilia...
  52. ncbi request reprint Identification of mouse AAV capsid-specific CD8+ T cell epitopes
    Denise E Sabatino
    The Children s Hospital of Philadelphia, Philadelphia, PA 19104, USA
    Mol Ther 12:1023-33. 2005
    ..Identification of these epitopes will facilitate studies of immune response to AAV capsid in mouse models...
  53. pmc Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
    Haiyan Jiang
    Bayer HealthCare Pharmaceuticals, 800 Dwight Way, Berkeley, CA 94710, USA
    Blood 108:3321-8. 2006
    ..These findings enable a clinical study to assess the effects of immunomodulation on long-term FIX expression in patients with hemophilia B...
  54. ncbi request reprint Gene therapy: the moving finger
    Katherine A High
    Nature 435:577-9. 2005
  55. pmc Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality
    Majed N Aljamali
    Division of Hematology, The Children s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA
    J Clin Invest 118:1825-34. 2008
    ....
  56. ncbi request reprint Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography
    Guang Qu
    Avigen Inc, Alameda, CA 94502, USA
    J Virol Methods 140:183-92. 2007
    ..15 x 10(14)vg containing 0.25 x 10(14) empty capsids, corresponding to 74% vector yield and 86-fold reduction in empty capsids in the vector product...
  57. ncbi request reprint Stakeholders' conference sharpens focus on challenges of clinical gene transfer
    Katherine A High
    Mol Ther 12:581-2. 2005
  58. ncbi request reprint The risks of germline gene transfer
    Katherine A High
    Children s Hospital of Philadelphia, USA
    Hastings Cent Rep 33:3. 2003

Research Grants8

  1. INHIBITOR FORMATION IN GENE THERAPY FOR HEMOPHILIA
    Katherine High; Fiscal Year: 1999
    ..These studies will involve a collaboration between the P.I.'s lab and the laboratory of Dr. Hildegund Ertl, an immunologist with experience in characterizing immune responses in the setting of viral vectors. ..
  2. INHIBITOR FORMATION IN GENE THERAPY FOR HEMOPHILIA
    Katherine High; Fiscal Year: 2000
    ..These studies will involve a collaboration between the P.I.'s lab and the laboratory of Dr. Hildegund Ertl, an immunologist with experience in characterizing immune responses in the setting of viral vectors. ..
  3. INHIBITOR FORMATION IN GENE THERAPY FOR HEMOPHILIA
    Katherine High; Fiscal Year: 2001
    ..These studies will involve a collaboration between the P.I.'s lab and the laboratory of Dr. Hildegund Ertl, an immunologist with experience in characterizing immune responses in the setting of viral vectors. ..
  4. PEDIATRIC HEMATOLOGY RESEARCH TRAINING PROGRAM
    Katherine High; Fiscal Year: 2007
    ..End of Abstract) ..
  5. Gene Therapy for hemophilia using muscle-expressed FVIIa
    Katherine A High; Fiscal Year: 2011
    ..The proposed work will allow us to increase the therapeutic applicability of our approach and enhance its safety, prior to a clinical application. ..