Christopher E Walsh

Summary

Affiliation: Mount Sinai School of Medicine
Country: USA

Publications

  1. doi request reprint Hemophilia clinical gene therapy: brief review
    Christopher E Walsh
    Mount Sinai School of Medicine, New York City, NY 10029, USA
    Transl Res 161:307-12. 2013
  2. doi request reprint Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices
    C E Walsh
    Department of Medicine, Tisch Cancer Center, Mount Sinai School of Medicine, New York, NY 10029, USA
    Haemophilia 15:1014-21. 2009
  3. doi request reprint Adeno-associated virus serotype 8 ApoA-I gene transfer reduces progression of atherosclerosis in ApoE-KO mice: comparison of intramuscular and intravenous administration
    Giovanni Cimmino
    Atherothrombosis Research Unit, Mount Sinai School of Medicine, New York, NY 10029, USA
    J Cardiovasc Pharmacol 57:325-33. 2011
  4. ncbi request reprint RNA repair for haemophilia A
    Hengjun Chao
    Department of Medicine, Division of Hematology and Medical Oncology, Mt Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA
    Expert Rev Mol Med 8:1-8. 2006
  5. ncbi request reprint Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing
    Hengjun Chao
    Department of Medicine, Mt Sinai School of Medicine, New York, New York 10029, USA
    Nat Med 9:1015-9. 2003
  6. ncbi request reprint AAV vectors for hemophilia B gene therapy
    Hengjun Chao
    Department of Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA
    Mt Sinai J Med 71:305-13. 2004
  7. pmc Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats
    Benjamin Storek
    Department of Medicine Hematology Oncology, Mount Sinai School of Medicine, New York, NY, USA
    Mol Pain 2:4. 2006
  8. doi request reprint Safe and sustained overexpression of functional apolipoprotein A-I/high-density lipoprotein in apolipoprotein A-I-null mice by muscular adeno-associated viral serotype 8 vector gene transfer
    Giovanni Cimmino
    Mount Sinai School of Medicine, New York, NY 10029, USA
    J Cardiovasc Pharmacol 54:405-11. 2009
  9. ncbi request reprint Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector
    Kaoru Yamada
    Department of Medicine, Mount Sinai School of Medicine, New York, New York 10029, USA
    Mol Ther 8:600-10. 2003
  10. ncbi request reprint Intrathecal gene transfer by adeno-associated virus for pain
    Andreas S Beutler
    Mount Sinai School of Medicine, Department of Medicine, Division of Hematology Oncology, New York, NY 10029, USA
    Curr Opin Mol Ther 7:431-9. 2005

Research Grants

  1. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2001
  2. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2005
  3. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2004
  4. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2004
  5. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2002
  6. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2002
  7. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2002
  8. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2003
  9. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2003
  10. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2002

Collaborators

Detail Information

Publications20

  1. doi request reprint Hemophilia clinical gene therapy: brief review
    Christopher E Walsh
    Mount Sinai School of Medicine, New York City, NY 10029, USA
    Transl Res 161:307-12. 2013
    ..This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias...
  2. doi request reprint Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practices
    C E Walsh
    Department of Medicine, Tisch Cancer Center, Mount Sinai School of Medicine, New York, NY 10029, USA
    Haemophilia 15:1014-21. 2009
    ..These findings suggest that prophylaxis prevents bleeding in adults with severe haemophilia A and that discontinuation of the prophylactic regimen is associated with increased bleeding events...
  3. doi request reprint Adeno-associated virus serotype 8 ApoA-I gene transfer reduces progression of atherosclerosis in ApoE-KO mice: comparison of intramuscular and intravenous administration
    Giovanni Cimmino
    Atherothrombosis Research Unit, Mount Sinai School of Medicine, New York, NY 10029, USA
    J Cardiovasc Pharmacol 57:325-33. 2011
    ....
  4. ncbi request reprint RNA repair for haemophilia A
    Hengjun Chao
    Department of Medicine, Division of Hematology and Medical Oncology, Mt Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA
    Expert Rev Mol Med 8:1-8. 2006
    ..The use of trans-splicing to effect phenotypic change in the hereditary bleeding disorder haemophilia A will be discussed...
  5. ncbi request reprint Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing
    Hengjun Chao
    Department of Medicine, Mt Sinai School of Medicine, New York, New York 10029, USA
    Nat Med 9:1015-9. 2003
    ..This is the first description of phenotypic correction of a genetic defect by RNA repair in a knockout animal model. Our results indicate the feasibility of using SMaRT to repair RNA for the treatment of genetic diseases...
  6. ncbi request reprint AAV vectors for hemophilia B gene therapy
    Hengjun Chao
    Department of Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA
    Mt Sinai J Med 71:305-13. 2004
    ..The latest progress in gene delivery of coagulant factor IX (for hemophilia B) using AAV serotype vectors is described in detail...
  7. pmc Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats
    Benjamin Storek
    Department of Medicine Hematology Oncology, Mount Sinai School of Medicine, New York, NY, USA
    Mol Pain 2:4. 2006
    ..Our studies focused on recombinant adeno-associated virus (rAAV), one of the most promising vector types for clinical use...
  8. doi request reprint Safe and sustained overexpression of functional apolipoprotein A-I/high-density lipoprotein in apolipoprotein A-I-null mice by muscular adeno-associated viral serotype 8 vector gene transfer
    Giovanni Cimmino
    Mount Sinai School of Medicine, New York, NY 10029, USA
    J Cardiovasc Pharmacol 54:405-11. 2009
    ..Our data suggest that intramuscular AAV8-mediated gene transfer of human ApoA-I results in a significant and maintained increase in ApoA-I and functional HDL...
  9. ncbi request reprint Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector
    Kaoru Yamada
    Department of Medicine, Mount Sinai School of Medicine, New York, New York 10029, USA
    Mol Ther 8:600-10. 2003
    ..Our results suggest that the lentiviral vector transduces stem cells capable of self-renewal and long-term hematopoiesis in vivo and is potentially useful for clinical gene therapy of FA hematopoietic cells...
  10. ncbi request reprint Intrathecal gene transfer by adeno-associated virus for pain
    Andreas S Beutler
    Mount Sinai School of Medicine, Department of Medicine, Division of Hematology Oncology, New York, NY 10029, USA
    Curr Opin Mol Ther 7:431-9. 2005
    ..AAV vectors may therefore become an important tool for translational studies to validate newly identified therapeutic targets in clinical pain states...
  11. ncbi request reprint Spliceosome-mediated RNA trans-splicing
    Yanping Yang
    Department of Medicine, Mt Sinai School of Medicine, One Gustave Levy Place, New York, NY 10029, USA
    Mol Ther 12:1006-12. 2005
    ..We also provide an update on the progress of this emerging technology toward the development of molecular therapy and diagnosis for human diseases and discuss the outstanding issues and challenges confronting RNA therapeutics...
  12. ncbi request reprint Gene therapy progress and prospects: gene therapy for the hemophilias
    Christopher E Walsh
    Mt Sinai School of Medicine, One Gustave Levy Place, Rm 24 42C Annenberg Building, New York City, NY 10029, USA
    Gene Ther 10:999-1003. 2003
    ..Based on a scientific understanding of the molecular and cellular defects, leading to the bleeding phenotype, impressive strides have been made in the last 2 years...
  13. doi request reprint Targeted integration of a rAAV vector into the AAVS1 region
    Peter Ward
    Mount Sinai School of Medicine, Tisch Cancer Center, New York, NY 10029, United States
    Virology 433:356-66. 2012
    ..Based on the pattern of integrants we propose, for potential use in ex vivo targeted gene therapy, a simplified PCR method to identify clones that have rAAV genomes integrated into AAVS1...
  14. doi request reprint Chimeric AAV Cap sequences alter gene transduction
    Peter Ward
    Mount Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA
    Virology 386:237-48. 2009
    ..The selected virus turned out to be surprisingly limited by its target cell and method of selection...
  15. ncbi request reprint New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategies
    Christopher E Walsh
    Division of Hematology Oncology, Mount Sinai School of Medicine, New York, NY 10029, USA
    Semin Hematol 41:297-302. 2004
    ..In this article we will describe the use of RNA species to either suppress unwanted gene activity or to repair defective genes. Examples of RNA inhibition and repair will be discussed...
  16. ncbi request reprint Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy
    Hengjun Chao
    UNC Gene Therapy Center, Department of Medicine, University of North Carolina, Chapel Hill 27599, USA
    Curr Opin Mol Ther 4:499-504. 2002
    ..This review describes recent novel molecular strategies for the treatment of the hemophilias...
  17. ncbi request reprint RNA repair using spliceosome-mediated RNA trans-splicing
    S Gary Mansfield
    INTRONN Inc, Gaithersburg, MD 20878, USA
    Trends Mol Med 10:263-8. 2004
  18. ncbi request reprint Lentivirus vector purification using anion exchange HPLC leads to improved gene transfer
    Kaoru Yamada
    University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
    Biotechniques 34:1074-8, 1080. 2003
    ..We conclude that the purification method using the HPLC system provides the highly purified virus vector that reduces cell toxicity and significantly improves gene transfer in primary cells...
  19. ncbi request reprint Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides
    Thipparat Suwanmanee
    Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina 27599 7295, USA
    Mol Pharmacol 62:545-53. 2002
    ....
  20. ncbi request reprint Gene therapy for the hemophilias
    Christopher E Walsh
    Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599, USA
    Curr Opin Pediatr 14:12-6. 2002
    ..The author here provides a critical assessment of the state of hemophilia gene transfer and its relevance to the field as a whole...

Research Grants12

  1. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2001
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  2. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2005
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..
  3. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2004
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..
  4. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2004
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  5. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2002
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..
  6. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2002
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  7. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2002
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  8. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2003
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  9. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2003
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..
  10. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2002
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..
  11. Gene Transfer of Hematopoietic Stem Cells
    Christopher Walsh; Fiscal Year: 2002
    ..Information obtained from the planned studies will provide a better understanding of abnormal hematopoiesis in FA and better define therapeutic strategies important for designing future human clinical trials. ..
  12. Gene Therapy for the Hemophilias
    Christopher Walsh; Fiscal Year: 2006
    ..Each method will be optimized and in AAV vectors for FVIII production and tested in vivo using immunodeficient and FVIII knockout mice and hemophilic A canines. ..