M Sadelain

Summary

Affiliation: Memorial Sloan-Kettering Cancer Center
Country: USA

Publications

  1. pmc CD19 CAR-targeted T cells induce long-term remission and B Cell Aplasia in an immunocompetent mouse model of B cell acute lymphoblastic leukemia
    MARCO L DAVILA
    Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, United States of America
    PLoS ONE 8:e61338. 2013
  2. pmc The basic principles of chimeric antigen receptor design
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, and Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Cancer Discov 3:388-98. 2013
  3. doi request reprint Safe harbours for the integration of new DNA in the human genome
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Nat Rev Cancer 12:51-8. 2012
  4. ncbi request reprint Globin gene transfer for treatment of the beta-thalassemias and sickle cell disease
    Michel Sadelain
    Laboratory of Gene Transfer and Gene Expression, Gene Transfer and Somatic Cell Engineering Facility, Box 182 Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Best Pract Res Clin Haematol 17:517-34. 2004
  5. pmc Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Mol Ther 17:1994-9. 2009
  6. doi request reprint The promise and potential pitfalls of chimeric antigen receptors
    Michel Sadelain
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Curr Opin Immunol 21:215-23. 2009
  7. ncbi request reprint Targeting tumours with genetically enhanced T lymphocytes
    Michel Sadelain
    Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Nat Rev Cancer 3:35-45. 2003
  8. ncbi request reprint Stem cell engineering for the treatment of severe hemoglobinopathies
    Michel Sadelain
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Curr Mol Med 8:690-7. 2008
  9. ncbi request reprint Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy
    Michel Sadelain
    Gene Transfer and Gene Expression Laboratory, Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Hum Gene Ther 18:1-9. 2007
  10. ncbi request reprint Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia
    Michel Sadelain
    Memorial Sloan Kettering Cancer Center, New York 10021, USA
    Curr Opin Hematol 13:142-8. 2006

Research Grants

Detail Information

Publications94

  1. pmc CD19 CAR-targeted T cells induce long-term remission and B Cell Aplasia in an immunocompetent mouse model of B cell acute lymphoblastic leukemia
    MARCO L DAVILA
    Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, United States of America
    PLoS ONE 8:e61338. 2013
    ..The mouse model we report here should prove valuable for investigating CAR-based and other therapies for adult B-ALL...
  2. pmc The basic principles of chimeric antigen receptor design
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, and Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Cancer Discov 3:388-98. 2013
    ..CARs represent a new class of drugs with exciting potential for cancer immunotherapy...
  3. doi request reprint Safe harbours for the integration of new DNA in the human genome
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Nat Rev Cancer 12:51-8. 2012
    ..In this Opinion article, we discuss 'genomic safe harbours' - chromosomal locations where therapeutic transgenes can integrate and function in a predictable manner without perturbing endogenous gene activity and promoting cancer...
  4. ncbi request reprint Globin gene transfer for treatment of the beta-thalassemias and sickle cell disease
    Michel Sadelain
    Laboratory of Gene Transfer and Gene Expression, Gene Transfer and Somatic Cell Engineering Facility, Box 182 Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Best Pract Res Clin Haematol 17:517-34. 2004
    ..Here we review the principles underlying the genesis of regulated vectors for stem cell therapy...
  5. pmc Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells
    Michel Sadelain
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Mol Ther 17:1994-9. 2009
    ..These early but promising studies raise the prospect of further developing these strategies for clinical investigation...
  6. doi request reprint The promise and potential pitfalls of chimeric antigen receptors
    Michel Sadelain
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Curr Opin Immunol 21:215-23. 2009
    ..If the immunogenicity of CARs can be averted, the versatility of their design and HLA-independent antigen recognition will make CARs tools of choice for T cell engineering for the development of targeted cancer immunotherapies...
  7. ncbi request reprint Targeting tumours with genetically enhanced T lymphocytes
    Michel Sadelain
    Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Nat Rev Cancer 3:35-45. 2003
    ..Genetically modified T cells are therefore proving to be of great value for basic immunology and experimental immunotherapy...
  8. ncbi request reprint Stem cell engineering for the treatment of severe hemoglobinopathies
    Michel Sadelain
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Curr Mol Med 8:690-7. 2008
    ..Altogether, these recent advances bode well for the advent of curative stem cell-based therapies...
  9. ncbi request reprint Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy
    Michel Sadelain
    Gene Transfer and Gene Expression Laboratory, Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Hum Gene Ther 18:1-9. 2007
  10. ncbi request reprint Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia
    Michel Sadelain
    Memorial Sloan Kettering Cancer Center, New York 10021, USA
    Curr Opin Hematol 13:142-8. 2006
    ....
  11. ncbi request reprint Progress toward the genetic treatment of the beta-thalassemias
    Michel Sadelain
    Gene Transfer and Gene Expression Laboratory, Memorial Sloan Kettering Cancer Center, Box 182, 1275 York Ave, New York, NY 10021, USA
    Ann N Y Acad Sci 1054:78-91. 2005
    ..As such, they provide a general paradigm for improving vector safety in stem cell-based gene therapy...
  12. ncbi request reprint Globin gene transfer as a potential treatment for the beta-thalassaemias and sickle cell disease
    M Sadelain
    Laboratory of Gene Transfer and Gene Expression, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Vox Sang 87:235-42. 2004
  13. ncbi request reprint Sturm und drang over suicidal lymphocytes
    Michel Sadelain
    The Gene Transfer and Somatic Cell Engineering Laboratory, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York 10021, USA
    Mol Ther 5:655-7. 2002
  14. ncbi request reprint Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells
    M Sadelain
    Department of Human Genetics, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Curr Opin Hematol 7:364-77. 2000
    ..Investigation of the control of transgene expression and prevention of vector silencing will become increasingly important...
  15. doi request reprint T-cell engineering for cancer immunotherapy
    Michel Sadelain
    Molecular Pharmacology and Chemistry Program, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Cancer J 15:451-5. 2009
    ....
  16. doi request reprint Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in beta-thalassemia
    Michel Sadelain
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, New York, USA
    Ann N Y Acad Sci 1202:52-8. 2010
    ..8 vector copy per cell in bulk CD34(+) cells) and to supply clinical grade vector to collaborating centers in the U.S.A. and in Europe. We anticipate that the first U.S. trial of globin gene transfer will start in 2010...
  17. ncbi request reprint A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer
    Stefano Rivella
    Department of Human Genetics Medicine, The Gene Transfer and Somatic Cell Engineering Laboratory, and the Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Blood 101:2932-9. 2003
    ..Our findings demonstrate the remarkable efficacy of lentivirus-mediated globin gene transfer in treating a fulminant blood disorder and strongly support the efficacy of gene therapy in the severe hemoglobinopathies...
  18. ncbi request reprint Mass cultured human fibroblasts overexpressing hTERT encounter a growth crisis following an extended period of proliferation
    K L MacKenzie
    James Ewing Laboratory of Developmental Hematopoiesis, Sloan Kettering Cancer Institute, New York, New York, 10021, USA
    Exp Cell Res 259:336-50. 2000
    ..However, these investigations also indicate that hTERT-expressing cells may undergo crisis following an extended life span and that immortality is not the universal outcome of hTERT expression in normal diploid fibroblasts...
  19. pmc Imaging transcriptional regulation of p53-dependent genes with positron emission tomography in vivo
    M Doubrovin
    Department of Neurology, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Proc Natl Acad Sci U S A 98:9300-5. 2001
    ..g., p21) by using conventional molecular-biological assays. PET imaging of p53 transcriptional activity in tumor xenografts by using the Cis-p53TKGFP reporter system may be useful in assessing novel therapeutic approaches...
  20. ncbi request reprint Targeted elimination of prostate cancer by genetically directed human T lymphocytes
    Terence P F Gade
    Department of Medical Physics, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Cancer Res 65:9080-8. 2005
    ..These results thus provide a strong rationale for undertaking phase I clinical studies to assess PSMA-targeted T cells in patients with metastatic prostate cancer...
  21. ncbi request reprint Globin gene transfer for the treatment of severe hemoglobinopathies: a paradigm for stem cell-based gene therapy
    Michel Sadelain
    Department of Human Genetics, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Gene Med 4:113-21. 2002
    ..This article addresses basic issues in stem cell-based gene therapy from the perspective of regulating transgene expression, taking globin gene transfer for the treatment of severe hemoglobinopathies as a paradigm...
  22. ncbi request reprint Prostate-specific membrane antigen (PSMA)-specific monoclonal antibodies in the treatment of prostate and other cancers
    M C Gong
    Department of Urology, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Cancer Metastasis Rev 18:483-90. 1999
    ..Currently, clinical usage of PSMA specific monoclonal antibodies has been limited to diagnostic immunohistochemistry and imaging of patients with prostate cancer. Novel applications for these antibodies will be discussed...
  23. doi request reprint Quantitative analysis of clinically relevant mutations occurring in lymphoid cells harboring gamma-retrovirus-encoded hsvtk suicide genes
    X Wang
    Gene Transfer and Somatic Cell Engineering Facility, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Gene Ther 15:1454-9. 2008
    ....
  24. ncbi request reprint Langerhans cells derived from genetically modified human CD34+ hemopoietic progenitors are more potent than peptide-pulsed Langerhans cells for inducing antigen-specific CD8+ cytolytic T lymphocyte responses
    Jianda Yuan
    Laboratory of Cellular Immunobiology, Division of Hematologic Oncology, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunol 174:758-66. 2005
    ..LCs genetically modified to express fluMP are also more potent stimulators of Ag-specific CD8(+) T cell responses than are peptide-pulsed LCs...
  25. pmc Monitoring the efficacy of adoptively transferred prostate cancer-targeted human T lymphocytes with PET and bioluminescence imaging
    Konstantin Dobrenkov
    Department of Radiology, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    J Nucl Med 49:1162-70. 2008
    ....
  26. ncbi request reprint Recombinant retroviruses pseudotyped with the vesicular stomatitis virus G glycoprotein mediate both stable gene transfer and pseudotransduction in human peripheral blood lymphocytes
    H F Gallardo
    Department of Human Genetics, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Blood 90:952-7. 1997
    ..Nonetheless, the physical stability of VSV-G-coated particles enables the concentration of viral stocks to 10(9) infectious particles per milliliter or more...
  27. ncbi request reprint Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRzeta /CD28 receptor
    John Maher
    Department of Human Genetics Medicine, Gene Transfer and Somatic Cell Engineering Laboratory, and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Nat Biotechnol 20:70-5. 2002
    ..These findings have important implications for adoptive immunotherapy of cancer, especially in the context of tumor cells that fail to express major histocompatibility complex antigens and co-stimulatory molecules...
  28. ncbi request reprint Eradication of systemic B-cell tumors by genetically targeted human T lymphocytes co-stimulated by CD80 and interleukin-15
    Renier J Brentjens
    Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, USA
    Nat Med 9:279-86. 2003
    ..In addition, transduced T cells from patients with chronic lymphocytic leukemia (CLL) effectively lyse autologous tumor cells. These findings strongly support the clinical feasibility of this therapeutic strategy...
  29. pmc Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras
    Eirini P Papapetrou
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center MSKCC, New York, NY 10065, USA
    J Clin Invest 119:157-68. 2009
    ..These results demonstrate the potential of miRNA-regulated transgene expression in stem cell-based therapies, including cancer immunotherapy...
  30. ncbi request reprint Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene
    Chad May
    Department of Human Genetics Medicine, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Blood 99:1902-8. 2002
    ..These results demonstrate for the first time that viral-mediated globin gene transfer in hematopoietic stem cells effectively treats a severe hemoglobin disorder...
  31. ncbi request reprint Generation of dual resistance to 4-hydroperoxycyclophosphamide and methotrexate by retroviral transfer of the human aldehyde dehydrogenase class 1 gene and a mutated dihydrofolate reductase gene
    N Takebe
    Department of Medicine, Program of Molecular Pharmacology and Experimental Therapeutics, Sloan-Kettering Institute, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue No. 78, New York, New York 10021, USA
    Mol Ther 3:88-96. 2001
    ..This construct may be useful for protecting patients from high-dose CTX- and MTX-induced myelosuppression...
  32. doi request reprint Current status of globin gene therapy for the treatment of beta-thalassaemia
    Leszek Lisowski
    Memorial Sloan Kettering Cancer Center, and Weill Graduate School of Mediccal Sciences, Cornell University, New York, NY 10021, USA
    Br J Haematol 141:335-45. 2008
    ..Altogether, recent advances in globin vector design bode well for upcoming clinical trials to assess the therapeutic value of globin gene transfer...
  33. ncbi request reprint Serial in vivo imaging of the targeted migration of human HSV-TK-transduced antigen-specific lymphocytes
    Guenther Koehne
    Allogeneic Bone Marrow Transplantation Service, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Nat Biotechnol 21:405-13. 2003
    ..This technique for imaging the migration of ex vivo-transduced antigen-specific T cells in vivo is informative, nontoxic, and potentially applicable to humans...
  34. ncbi request reprint Multiple stages of malignant transformation of human endothelial cells modelled by co-expression of telomerase reverse transcriptase, SV40 T antigen and oncogenic N-ras
    Karen L MacKenzie
    James Ewing Laboratory of Developmental Haematopoiesis and Department of Human Genetics, Sloan Kettering Cancer Institute, New York, NY, USA
    Oncogene 21:4200-11. 2002
    ..This model will be useful for understanding mechanisms underlying vascular and angiogenic neoplasias, as well as for testing drugs designed to curtail aberrant EC growth...
  35. doi request reprint Retroviral transduction of murine primary T lymphocytes
    James Lee
    Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA
    Methods Mol Biol 506:83-96. 2009
    ....
  36. pmc Chimeric antigen receptors combining 4-1BB and CD28 signaling domains augment PI3kinase/AKT/Bcl-XL activation and CD8+ T cell-mediated tumor eradication
    Xiao Song Zhong
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Mol Ther 18:413-20. 2010
    ..These findings further support the concept of integrating optimized costimulatory properties into recombinant antigen receptors to augment the survival and function of genetically targeted T cells within the tumor microenvironment...
  37. ncbi request reprint Stem cell-derived erythroid cells mediate long-term systemic protein delivery
    Alex H Chang
    Laboratory of Gene Transfer and Gene Expression, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Nat Biotechnol 24:1017-21. 2006
    ....
  38. ncbi request reprint Scalable expansion of potent genetically modified human langerhans cells in a closed system for clinical applications
    Jianda Yuan
    Laboratory of Cellular Immunobiology, Memorial Sloan Kettering Cancer Center, New York, NY, USA
    J Immunother 30:634-43. 2007
    ..We have thus developed a scalable closed process to expand genetically modified, biologically functional CD34+ hematopoietic progenitor cell-derived LCs for phase I clinical trials...
  39. ncbi request reprint In vivo imaging and quantitation of adoptively transferred human antigen-specific T cells transduced to express a human norepinephrine transporter gene
    Mikhail M Doubrovin
    Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Cancer Res 67:11959-69. 2007
    ..The hNET-based system described may thus have significant potential as a nonimmunogenic reporter for extended repeated quantitative in vivo imaging of transduced cells in man...
  40. ncbi request reprint T cell-encoded CD80 and 4-1BBL induce auto- and transcostimulation, resulting in potent tumor rejection
    Matthias T Stephan
    Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan Kettering Cancer Center MSKCC, New York, New York 10021, USA
    Nat Med 13:1440-9. 2007
    ..This new strategy of endowing T cells with constitutively expressed costimulatory ligands could be extended to other ligand-receptor pairs and used to enhance any targeted adoptive transfer therapy...
  41. pmc Concurrent visualization of trafficking, expansion, and activation of T lymphocytes and T-cell precursors in vivo
    Il Kang Na
    Department of Immunology, Memorial Sloan Kettering Cancer Center, New York, NY
    Blood 116:e18-25. 2010
    ..Because NFAT plays an important role in a wide range of cell types, our system could provide new insights into a variety of biologic processes...
  42. pmc Locus control region elements HS1 and HS4 enhance the therapeutic efficacy of globin gene transfer in beta-thalassemic mice
    Leszek Lisowski
    Center for Cell Engineering, Gene Transfer and Somatic Cell Engineering Laboratory, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Blood 110:4175-8. 2007
    ..Thus, while vectors encoding HS2-3-4 provide curative levels of hemoglobin at 1 to 2 copies per cell, adding HS1 is a promising alternative strategy if upcoming clinical trials prove higher levels of expression to be necessary...
  43. ncbi request reprint Production scale-up and validation of packaging cell clearance of clinical-grade retroviral vector stocks produced in cell factories
    M Przybylowski
    Gene Transfer and Somatic Cell Engineering Facility, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Gene Ther 13:95-100. 2006
    ..This methodology is not limited to the generation of cell-free clinical oncoretroviral VS, and can be applied to other types of vectors produced in packaging cell lines, such as lentiviral vectors...
  44. ncbi request reprint Insertional oncogenesis in gene therapy: how much of a risk?
    M Sadelain
    1Memorial Sloan Kettering Cancer Center, New York, NY, USA
    Gene Ther 11:569-73. 2004
  45. pmc Imaging TCR-dependent NFAT-mediated T-cell activation with positron emission tomography in vivo
    V Ponomarev
    Departments of Neurology and Radiology, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Neoplasia 3:480-8. 2001
    ..PET imaging of TCR-induced NFAT-dependent transcriptional activity may be useful in the assessment of T cell responses, T-cell-based adoptive therapies, vaccination strategies and immunosuppressive drugs...
  46. ncbi request reprint A promising genetic approach to the treatment of beta-thalassemia
    C May
    Laboratory of Gene Transfer and Gene Expression, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Trends Cardiovasc Med 11:276-80. 2001
    ..Our results demonstrate that with thoughtful vector design one can successfully express long-term, therapeutic levels of virally encoded human beta-globin in the erythroid progeny of hematopoietic stem cells...
  47. ncbi request reprint Genetically targeted T cells eradicate systemic acute lymphoblastic leukemia xenografts
    Renier J Brentjens
    Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Clin Cancer Res 13:5426-35. 2007
    ..Here, we investigate the etiologies of treatment failure in this model and design approaches to enhance the efficacy of this adoptive strategy...
  48. ncbi request reprint Multifactorial optimization of gammaretroviral gene transfer into human T lymphocytes for clinical application
    Alfonso Quintas-Cardama
    Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Hum Gene Ther 18:1253-60. 2007
    ....
  49. doi request reprint A genetic strategy for single and combinatorial analysis of miRNA function in mammalian hematopoietic stem cells
    Eirini P Papapetrou
    Center for Cell Engineering Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
    Stem Cells 28:287-96. 2010
    ..The strategy described herein will prove useful in functional miRNA studies in mammalian hematopoietic stem cells...
  50. pmc A panel of artificial APCs expressing prevalent HLA alleles permits generation of cytotoxic T cells specific for both dominant and subdominant viral epitopes for adoptive therapy
    Aisha N Hasan
    Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunol 183:2837-50. 2009
    ..It may be of particular value for recipients of transplants from HLA-disparate donors...
  51. doi request reprint Gene therapy for homozygous beta-thalassemia. Is it a reality?
    Farid Boulad
    Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
    Hemoglobin 33:S188-96. 2009
    ..The protocol will be offered to patients with transfusion-dependent ss-thalassemia who are 15 years or older and lack a matched, related donor...
  52. pmc Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice
    Alex H Chang
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, New York, USA
    Mol Ther 16:1745-52. 2008
    ....
  53. pmc Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs
    Gabsang Lee
    Developmental Biology Program, Sloan Kettering Institute, 1275 York Ave, USA
    Nature 461:402-6. 2009
    ..Our study illustrates the promise of iPSC technology for gaining new insights into human disease pathogenesis and treatment...
  54. pmc Sensitive in vivo imaging of T cells using a membrane-bound Gaussia princeps luciferase
    Elmer B Santos
    Department of Radiology, Memorial Sloan Kettering Cancer Center MSKCC, 1275 York Avenue, New York, New York 10021, USA
    Nat Med 15:338-44. 2009
    ..This sensitive imaging technology has application to many in vivo cell-based studies in a wide array of mouse models...
  55. pmc Manufacturing validation of biologically functional T cells targeted to CD19 antigen for autologous adoptive cell therapy
    Daniel Hollyman
    Gene Transfer and Somatic Cell Engineering Facility, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunother 32:169-80. 2009
    ..It can also be adapted for other clinical trials involving the expansion and transduction of patient or donor T cells using any CAR or T-cell receptor...
  56. pmc Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling
    Stuart M Chambers
    Developmental Biology Program, Sloan Kettering Institute, 1275 York Ave, New York, New York 10065, USA
    Nat Biotechnol 27:275-80. 2009
    ..Noggin/SB431542-based neural induction should facilitate the use of hES and hiPS cells in regenerative medicine and disease modeling and obviate the need for protocols based on stromal feeders or embryoid bodies...
  57. ncbi request reprint Langerhans-type dendritic cells genetically modified to express full-length antigen optimally stimulate CTLs in a CD4-dependent manner
    Jianda Yuan
    Laboratory of Cellular Immunobiology, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunol 176:2357-65. 2006
    ..LCs, transduced with a retroviral vector encoding full-length Ag, stimulate potent CTLs directed against multiple epitopes in a CD4(+) Th cell-dependent manner...
  58. ncbi request reprint The ABCs of artificial antigen presentation
    Jiyun V Kim
    Laboratory of Gene Transfer and Gene Expression, Gene Transfer and Somatic Cell Engineering Facility, Department of Medicine and Immunology Program, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York 10021, USA
    Nat Biotechnol 22:403-10. 2004
    ....
  59. ncbi request reprint A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
    Selda Samakoglu
    Laboratory of Gene Transfer and Gene Expression, Sloan Kettering Institute, New York, New York 10021, USA
    Nat Biotechnol 24:89-94. 2006
    ....
  60. ncbi request reprint CTLA-4 blockade in combination with xenogeneic DNA vaccines enhances T-cell responses, tumor immunity and autoimmunity to self antigens in animal and cellular model systems
    Polly D Gregor
    Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Vaccine 22:1700-8. 2004
    ..Based on these pre-clinical studies, we suggest that anti-CTLA-4 should be tested with xenogeneic DNA vaccines against cancer and that special attention should be given to sequence and schedule of administration...
  61. ncbi request reprint [131I]FIAU labeling of genetically transduced, tumor-reactive lymphocytes: cell-level dosimetry and dose-dependent toxicity
    Pat Zanzonico
    Department of Medical Physics, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY, USA
    Eur J Nucl Med Mol Imaging 33:988-97. 2006
    ....
  62. ncbi request reprint Novel strategies for cancer therapy: the potential of genetically modified T lymphocytes
    Isabelle Riviere
    Department of Medicine, Gene Transfer and Somatic Cell Engineering Laboratory, Immunology Program, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Curr Hematol Rep 3:290-7. 2004
    ..T cells can also be engineered in an attempt to control their homing, to increase the safety of adoptive T-cell therapies, and express markers that can be tracked by noninvasive imaging technologies...
  63. ncbi request reprint Artificial antigen-presenting cells transduced with telomerase efficiently expand epitope-specific, human leukocyte antigen-restricted cytotoxic T cells
    Jakob Dupont
    Department of Medicine, Memorial Sloan Kettering Cancer Center and the Joan and Sanford Weill Medical College of Cornell University and Immunology Program, Sloan Kettering Institute, New York, New York 10021, USA
    Cancer Res 65:5417-27. 2005
    ....
  64. pmc Induction of autoantibodies to syngeneic prostate-specific membrane antigen by xenogeneic vaccination
    Polly D Gregor
    Genitourinary Oncology Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Int J Cancer 116:415-21. 2005
    ..These results establish the basis for clinical trials to test PSMA DNA vaccines in patients with solid tumors that either express PSMA directly or that depend on normal endothelial cells expressing PSMA for their continued growth...
  65. ncbi request reprint Negative-feedback regulation of CD28 costimulation by a novel mitogen-activated protein kinase phosphatase, MKP6
    F Marti
    T Cell Signal Transduction Laboratory, Hospital for Special Surgery, Weill Medical College of Cornell University, New York, NY 10021, USA
    J Immunol 166:197-206. 2001
    ..Taken together, these results suggest that MKP6 functions as a novel negative-feedback regulator of CD28 costimulatory signaling that controls the activation of MAP kinases...
  66. ncbi request reprint Glucose 6-phosphate dehydrogenase expression is less prone to variegation when driven by its own promoter
    M De Angioletti
    Department of Human Genetics, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, New York, NY 10021, USA
    Gene 267:221-31. 2001
    ..This decreased variability is important in order to help ensuring a consistent level of delivery of the needed gene product in future therapeutic protocols...
  67. pmc Tumor immunotherapy across MHC barriers using allogeneic T-cell precursors
    Johannes L Zakrzewski
    Department Immunology, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, New York 10021, USA
    Nat Biotechnol 26:453-61. 2008
    ..We conclude that ex vivo generated MHC-disparate T-cell precursors from any donor can be used universally for 'off-the-shelf' immunotherapy, and can be further enhanced by genetic engineering for targeted immunotherapy...
  68. pmc IL-7 and IL-21 are superior to IL-2 and IL-15 in promoting human T cell-mediated rejection of systemic lymphoma in immunodeficient mice
    John C Markley
    Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, New York, NY, USA
    Blood 115:3508-19. 2010
    ..These results support the use of gamma(c)-cytokines in cancer immunotherapy, and establish that there exists more than 1 human T-cell memory phenotype associated with long-term tumor immunity...
  69. ncbi request reprint The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors
    Alex H Chang
    Laboratory of Gene Transfer and Gene Expression, Memorial Sloan Kettering Cancer Center, New York, New York, USA
    Mol Ther 15:445-56. 2007
    ....
  70. ncbi request reprint Long-term survival of dogs with advanced malignant melanoma after DNA vaccination with xenogeneic human tyrosinase: a phase I trial
    Philip J Bergman
    Donaldson Atwood Cancer Clinic and Flaherty Comparative Oncology Laboratory, The E and M Bobst Hospital of the Animal Medical Center, New York, New York 10021, USA
    Clin Cancer Res 9:1284-90. 2003
    ..These studies provided the rationale for a trial of xenogeneic DNA vaccination in CMM using the human tyrosinase gene...
  71. ncbi request reprint Rapid expansion of cytomegalovirus-specific cytotoxic T lymphocytes by artificial antigen-presenting cells expressing a single HLA allele
    Genovefa A Papanicolaou
    Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA
    Blood 102:2498-505. 2003
    ..Furthermore, the CTL expansion obtained with AAPCs encoding full-length pp65 indicates that AAPCs may be used to present known as well as unknown CTL epitopes in the context of the AAPC's HLA...
  72. ncbi request reprint Ligand binding to inhibitory killer cell Ig-like receptors induce colocalization with Src homology domain 2-containing protein tyrosine phosphatase 1 and interruption of ongoing activation signals
    Yatin M Vyas
    Department of Pediatrics, Sloan Kettering Institute for Cancer Research, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunol 173:1571-8. 2004
    ..These results indicate that the outcome of NK cell-target cell interactions is dictated by early quantitative differences in cumulative activating and inhibitory signals...
  73. ncbi request reprint A unique folate hydrolase, prostate-specific membrane antigen (PSMA): a target for immunotherapy?
    J Tasch
    Department of Urology, Memorial Hospital, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Crit Rev Immunol 21:249-61. 2001
    ..This review discusses the historical background, biochemical characteristics, gene regulation, potential for targeting, tissue localization, and a novel T-body strategy...
  74. ncbi request reprint Fulminant experimental autoimmune encephalo-myelitis induced by retrovirally mediated TCR gene transfer
    Amy L Stolzer
    The Laboratory of T cell Immunobiology, Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Eur J Immunol 35:1822-30. 2005
    ..Therefore, retroviral TCR delivery into the bone marrow supports the development of T cells into fully functional effector cells...
  75. ncbi request reprint Limited proliferation and telomere dysfunction following telomerase inhibition in immortal murine fibroblasts
    Jessica Boklan
    James Ewing Laboratory of Developmental Hematopoiesis, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Cancer Res 62:2104-14. 2002
    ..Moreover, the murine model used here should prove useful in further evaluating telomerase inhibition as an anticancer therapy...
  76. ncbi request reprint Somatic cell engineering and the immunotherapy of leukemias and lymphomas
    Renier J Brentjens
    Department of Medicine and Clinical Laboratories, Leukemia Service Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    Adv Pharmacol 51:347-70. 2004
  77. doi request reprint A new pyrimidine-specific reporter gene: a mutated human deoxycytidine kinase suitable for PET during treatment with acycloguanosine-based cytotoxic drugs
    Yury Likar
    Department of Radiology, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Nucl Med 51:1395-403. 2010
    ..In this article, we describe a series of new human-derived reporter genes based on human deoxycytidine kinase (dCK) suitable for clinical PET...
  78. ncbi request reprint Development of a new reporter gene system--dsRed/xanthine phosphoribosyltransferase-xanthine for molecular imaging of processes behind the intact blood-brain barrier
    Mikhail Doubrovin
    Memorial Sloan Kettering Cancer Center, 1275 York Avenue, Box 513, New York, NY 10021, USA
    Mol Imaging 2:93-112. 2003
    ..quot; Therefore, the novel dsRed/XPRT fusion gene can be used as a multimodality reporter system in the biological applications requiring two independent reporter genes, including the cells located behind the BBB...
  79. ncbi request reprint Development of a xenogeneic DNA vaccine program for canine malignant melanoma at the Animal Medical Center
    P J Bergman
    Donaldson Atwood Cancer Clinic and Flaherty Comparative Oncology Laboratory, The Animal Medical Center, 510 East 62nd Street, New York, NY 10021, USA
    Vaccine 24:4582-5. 2006
    ..These studies provided the impetus for development of a xenogeneic DNA vaccine program in CMM...
  80. pmc Frequency of missense mutations in the coding region of a eukaryotic gene transferred by retroviral vectors
    Maria De Angioletti
    Department of Human Genetics, Memorial Sloan Kettering Cancer Center, New York, New York 10021, USA
    J Virol 76:1991-4. 2002
    ..4 x 10(-5) per base pair per replication cycle. Mutations in the transferred gene can thus contribute to the impairment of the effectiveness of retrovirus-mediated gene transfer...
  81. pmc Conserved vertebrate mir-451 provides a platform for Dicer-independent, Ago2-mediated microRNA biogenesis
    Jr Shiuan Yang
    Department of Developmental Biology, Sloan Kettering Institute, New York, NY 10065, USA
    Proc Natl Acad Sci U S A 107:15163-8. 2010
    ..Beyond the demonstration of an alternative strategy to direct gene silencing, these observations open the way for transgenic rescue of Dicer conditional knockouts...
  82. pmc Intercellular transfer of P-glycoprotein mediates acquired multidrug resistance in tumor cells
    Andre Levchenko
    Department of Biomedical Engineering, Johns Hopkins University, Baltimore, MD 21218, USA
    Proc Natl Acad Sci U S A 102:1933-8. 2005
    ..These findings have important implications for proteomic analyses in tumor samples and resistance to cancer therapy...
  83. ncbi request reprint Therapeutic globin gene delivery using lentiviral vectors
    Stefano Rivella
    Curr Opin Mol Ther 4:505-14. 2002
    ..Here, we review the advantages afforded by lentivirus-mediated globin gene transfer and recent studies based on this strategy...
  84. pmc Fetal gene therapy of alpha-thalassemia in a mouse model
    Xiao Dong Han
    Cardiovascular Research Institute, Institute of Human Genetics and Department of Medicine, University of California, San Francisco, CA 94143, USA
    Proc Natl Acad Sci U S A 104:9007-11. 2007
    ....
  85. doi request reprint Towards the genetic treatment of beta-thalassemia: new disease models, new vectors, new cells
    Paolo Moi
    Haematologica 93:325-30. 2008
  86. ncbi request reprint Functional assessment of the engraftment potential of gammaretrovirus-modified CD34+ cells, using a short serum-free transduction protocol
    Tulin Budak-Alpdogan
    Department of Medicine, Cancer Institute of New Jersey, Robert Wood Johnson Medical School, University of Medicine and Dentistry of New Jersey, New Brunswick, 08903, USA
    Hum Gene Ther 17:780-94. 2006
    ..We have thus developed a short and efficient human MBP CD34(+) transduction protocol under serum-free conditions that is suitable and broadly applicable for phase I clinical trials...
  87. ncbi request reprint American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells
    Donald B Kohn
    Mol Ther 8:180-7. 2003
  88. ncbi request reprint Occurrence of leukaemia following gene therapy of X-linked SCID
    Donald B Kohn
    Division of Research Immunology BMT, Childrens Hospital Los Angeles, USC Keck School of Medicine, 4650 Sunset Boulevard, Los Angeles, California 90027, USA
    Nat Rev Cancer 3:477-88. 2003
    ..What were the causes of this cancer, and how can the therapeutic benefits of gene therapy be achieved while minimizing risk to the patient?..
  89. ncbi request reprint A "vector drain" in US gene therapy development?
    Michel Sadelain
    Mol Ther 16:801-2. 2008
  90. ncbi request reprint Cutting Edge: CD28 controls dominant regulatory T cell activity during active immunization
    Clay Lyddane
    Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    J Immunol 176:3306-10. 2006
    ..Immunization with adjuvant abrogates regulatory dominance, reducing overall Foxp3 expression in a CD28-dependent manner. We conclude that CD28 licenses active immunization by regulating Ag-induced immunoregulation...
  91. ncbi request reprint Production of clinical-grade plasmid DNA for human Phase I clinical trials and large animal clinical studies
    Mark Przybylowski
    Gene Transfer and Somatic Cell Engineering Facility, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Vaccine 25:5013-24. 2007
    ..We have therefore developed a reproducible and cost effective process to manufacture clinical-grade plasmid DNA. This process can be adapted by other academic centers for human or large animal clinical trials...
  92. pmc Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model
    Elizabeth M Kang
    Laboratory of Host Defense, National Institute of Allergy and Infectious Disease, National Institutes of Health, Bethesda, MD, USA
    Exp Hematol 34:132-9. 2006
    ..To decrease the toxicity profile, we sought to develop nonmyeloablative conditioning regimens and in this work, explored the use of intravenous busulfan in a large animal model...

Research Grants8

  1. GLOBIN GENE TRANSFER FOR THERAPY OF SICKLE CELL ANEMIA
    Michel Sadelain; Fiscal Year: 2004
    ....