Christopher E Walsh
Affiliation: Mount Sinai School of Medicine
- Hemophilia clinical gene therapy: brief reviewChristopher 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...
- Factor VIII prophylaxis for adult patients with severe haemophilia A: results of a US survey of attitudes and practicesC 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...
- Adeno-associated virus serotype 8 ApoA-I gene transfer reduces progression of atherosclerosis in ApoE-KO mice: comparison of intramuscular and intravenous administrationGiovanni Cimmino
Atherothrombosis Research Unit, Mount Sinai School of Medicine, New York, NY 10029, USA
J Cardiovasc Pharmacol 57:325-33. 2011....
- RNA repair for haemophilia AHengjun 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...
- AAV vectors for hemophilia B gene therapyHengjun 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...
- Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicingHengjun 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...
- Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in ratsBenjamin 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...
- 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 transferGiovanni 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...
- Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vectorKaoru 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...
- Intrathecal gene transfer by adeno-associated virus for painAndreas 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...
- Spliceosome-mediated RNA trans-splicingYanping 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...
- Gene therapy progress and prospects: gene therapy for the hemophiliasChristopher 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...
- Targeted integration of a rAAV vector into the AAVS1 regionPeter 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...
- Chimeric AAV Cap sequences alter gene transductionPeter 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...
- Current and future prospects for hemophilia gene therapyPeter Ward
a Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, One Gustave Levy Place, New York City, NY, USA
Expert Rev Hematol 9:649-59. 2016..As with any new technology, there are drawbacks, and the potential for immune/inflammatory and oncogenic risks have emerged and will be discussed. ..
- New paradigms for gene transfer: RNA trans-splicing and small interfering RNA as therapeutic strategiesChristopher 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...
- Hemophilia gene therapy: novel rAAV vectors and RNA repair strategyHengjun 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...
- RNA repair using spliceosome-mediated RNA trans-splicingS Gary Mansfield
INTRONN Inc, Gaithersburg, MD 20878, USA
Trends Mol Med 10:263-8. 2004
- Lentivirus vector purification using anion exchange HPLC leads to improved gene transferKaoru 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...
- Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotidesThipparat Suwanmanee
Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, North Carolina 27599 7295, USA
Mol Pharmacol 62:545-53. 2002....
- Gene therapy for the hemophiliasChristopher 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...
- Gene Transfer of Hematopoietic Stem CellsChristopher 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. ..
- Gene Therapy for the HemophiliasChristopher 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. ..