Donald Kohn

Summary

Affiliation: University of California
Country: USA

Publications

  1. pmc A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors
    Satiro N De Oliveira
    Department of Pediatrics, University of California, Los Angeles UCLA, 10833 Le Conte Avenue, A2 410 MDCC, MC 175217, Los Angeles, CA 90095 1752, USA
    J Transl Med 11:23. 2013
  2. pmc Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells
    Francesca Giannoni
    Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California, USA
    Mol Ther 21:1044-54. 2013
  3. pmc Update on gene therapy for immunodeficiencies
    Donald B Kohn
    Departments of Microbiology, Immunology and Immunology and Pediatrics, University of California, Los Angeles, 290D Biomedical Sciences Research Building, 615 Charles E Young Drive South, Los Angeles, CA 90095, USA
    Clin Immunol 135:247-54. 2010
  4. pmc In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors
    Gerhard Bauer
    Stem Cell Program, Department of Internal Medicine, University of California, Davis, Sacramento, California 95817, USA
    Mol Ther 16:1308-15. 2008
  5. ncbi request reprint Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB
    Yi Zheng
    Department of Biological Chemistry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1737, USA
    Mol Genet Metab 82:286-95. 2004
  6. ncbi request reprint Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow
    Yi Zheng
    Department of Biological Chemistry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1737, USA
    Mol Genet Metab 79:233-44. 2003
  7. ncbi request reprint Perspectives on gene therapy for immune deficiencies
    Greg M Podsakoff
    Division of Research Immunology, Bone Marrow Transplantation, Children s Hospital Los Angeles, Los Angeles, California 90027, USA
    Biol Blood Marrow Transplant 11:972-6. 2005
  8. ncbi request reprint Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector
    Hiroshi Kobayashi
    Department of Pediatrics, Division of Research Immunology Bone Marrow Transplantation, The Saban Research Institute at Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 11:776-89. 2005
  9. pmc Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system
    Teiko Sumiyoshi
    Division of Research Immunology Bone Marrow Transplantation, Saban Research Institute of Childrens Hospital Los Angeles, Los Angeles, CA 90027, USA
    Hum Gene Ther 20:1607-26. 2009
  10. 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

Research Grants

  1. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2005
  2. MND-ADA Transduced CD34 Cells for ADA-SCID
    Donald Kohn; Fiscal Year: 2009
  3. MND-ADA Transduced CD34 Cells for ADA-SCID
    Donald Kohn; Fiscal Year: 2005
  4. In Vivo ADA Gene Delivery for the Treatment of SCID
    Donald Kohn; Fiscal Year: 2009
  5. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2004
  6. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2003
  7. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2002
  8. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 2001
  9. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 2000
  10. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 1999

Collaborators

  • Ami J Shah
  • Barbara C Engel
  • R Parkman
  • Alice Tarantal
  • David Baltimore
  • Antoni Ribas
  • E M Smogorzewska
  • Christof von Kalle
  • Joseph Glorioso
  • G J Nabel
  • Tal Kafri
  • Mary Kearns-Jonker
  • M Sadelain
  • Rebecca Chan
  • Gay M Crooks
  • Aaron C Logan
  • Ingrid Bahner
  • Shundi Ge
  • Karen Pepper
  • Greg M Podsakoff
  • Denise Petersen
  • Roger P Hollis
  • Xiao Jin Yu
  • Teiko Sumiyoshi
  • Dennis L Haas
  • Yi Zheng
  • Gerhard Bauer
  • Satiro N De Oliveira
  • Sarah J Nightingale
  • Francesca Giannoni
  • Xiuli Wang
  • Cinnamon L Hardee
  • Karen A Pepper
  • Shantha Senadheera
  • Carolyn Lutzko
  • Nathalia Holt
  • Eszter Pais
  • Paula M Cannon
  • Tanja A Gruber
  • Malinda Lin
  • Kit Shaw
  • Dianne Skelton
  • Jung Eun Jang
  • Noboru Mitsuhashi
  • C Chang I Lee
  • Denise Peterson
  • Scott S Case
  • Hiroshi Kobayashi
  • Chang I Lee
  • Gerald J Cho
  • Sergey Ryazantsev
  • Elizabeth F Neufeld
  • Denise A Carbonaro
  • Nora Rozengurt
  • Mary A Price
  • Jiexin Wang
  • David Gjertson
  • Christine Ryan
  • Vivian Gersuk
  • Eric Gschweng
  • Sherie L Morrison
  • Xiaoyan Wang
  • Jennifer Wherley
  • James S Economou
  • Michael L Kaufman
  • Owen N Witte
  • Tamas Alexy
  • Vahagn Nikolian
  • Vanessa Taupin
  • Xingchao Wang
  • Philip D Gregory
  • Michael C Holmes
  • Roger Hollis
  • Jean Park
  • Kenneth Kim
  • Jianbin Wang
  • Geoffrey Friedman
  • Colleen Azen
  • Neena Kapoor
  • Sean McKnight
  • Hisham Abdel-Azim
  • Nathalia G Holt
  • Drew White
  • Sudhir Gupta
  • Michelle Hernandez
  • Karen Epport
  • Susie Csik
  • Ping Zhou
  • Louisa Wirthlin
  • Mo A Dao

Detail Information

Publications35

  1. pmc A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors
    Satiro N De Oliveira
    Department of Pediatrics, University of California, Los Angeles UCLA, 10833 Le Conte Avenue, A2 410 MDCC, MC 175217, Los Angeles, CA 90095 1752, USA
    J Transl Med 11:23. 2013
    ....
  2. pmc Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells
    Francesca Giannoni
    Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California, USA
    Mol Ther 21:1044-54. 2013
    ..In summary, we demonstrated the feasibility of engineering human HSPC to express a tumor-specific TCR to serve as a long-term source of tumor-targeted mature T cells for immunotherapy of melanoma...
  3. pmc Update on gene therapy for immunodeficiencies
    Donald B Kohn
    Departments of Microbiology, Immunology and Immunology and Pediatrics, University of California, Los Angeles, 290D Biomedical Sciences Research Building, 615 Charles E Young Drive South, Los Angeles, CA 90095, USA
    Clin Immunol 135:247-54. 2010
    ..New approaches using safer integrating vectors or direct correction of the defective gene underlying the PID are being developed and may lead to safer and effective gene therapy for PID...
  4. pmc In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors
    Gerhard Bauer
    Stem Cell Program, Department of Internal Medicine, University of California, Davis, Sacramento, California 95817, USA
    Mol Ther 16:1308-15. 2008
    ..Our in vivo system, therefore, helps to provide an assessment of the risks involved when retroviral or lentiviral vectors are considered for use in clinical gene therapy applications...
  5. ncbi request reprint Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB
    Yi Zheng
    Department of Biological Chemistry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1737, USA
    Mol Genet Metab 82:286-95. 2004
    ..These results show that genetically modified cells of hematopoietic origin can reduce the pathologic manifestations of MPS IIIB in the Naglu -/- mouse brain...
  6. ncbi request reprint Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow
    Yi Zheng
    Department of Biological Chemistry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095 1737, USA
    Mol Genet Metab 79:233-44. 2003
    ..The partial correction of brain pathology is attributed to migration of donor hematopoietic cells, demonstrated by the presence of the Y chromosome and of normal microglia in the brain of mice receiving IDUA cDNA...
  7. ncbi request reprint Perspectives on gene therapy for immune deficiencies
    Greg M Podsakoff
    Division of Research Immunology, Bone Marrow Transplantation, Children s Hospital Los Angeles, Los Angeles, California 90027, USA
    Biol Blood Marrow Transplant 11:972-6. 2005
  8. ncbi request reprint Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector
    Hiroshi Kobayashi
    Department of Pediatrics, Division of Research Immunology Bone Marrow Transplantation, The Saban Research Institute at Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 11:776-89. 2005
    ..The sustained expression of enzymatically active IDUA in multiple organs had a significant beneficial effect on the phenotypic abnormalities of MPS I, which may be translated to clinical gene therapy of patients with Hurler disease...
  9. pmc Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system
    Teiko Sumiyoshi
    Division of Research Immunology Bone Marrow Transplantation, Saban Research Institute of Childrens Hospital Los Angeles, Los Angeles, CA 90027, USA
    Hum Gene Ther 20:1607-26. 2009
    ....
  10. 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?..
  11. ncbi request reprint Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells
    Mary A Price
    Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, Los Angeles, California 90027, USA
    Mol Ther 6:645-52. 2002
    ..We conclude that FIV vectors can transfer genes into human hematopoietic cells as effectively as HIV-1 vectors, but that unknown elements in the current FIV backbone inhibit expression from FIV vectors in human hematopoietic cells...
  12. ncbi request reprint Transient gene expression by nonintegrating lentiviral vectors
    Sarah J Nightingale
    Division of Research Immunology Bone Marrow Transplant, The Saban Research Institute of Children s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 13:1121-32. 2006
    ..The development of NIL vectors provides a novel tool for efficient transient gene expression in primary stem cells and hematopoietic and lymphoid cells...
  13. ncbi request reprint Update on gene therapy of inherited immune deficiencies
    Barbara C Engel
    Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital Los Angeles, 4650 Sunset Blvd, Los Angeles, CA 90027, USA
    Curr Opin Mol Ther 5:503-7. 2003
    ..Depending upon the results of long-term follow-up, the successes together with the side effects highlight the relative merits of this therapeutic approach...
  14. ncbi request reprint [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]
    Elzbieta Monika Smogorzewska
    Research Immunology, Children Hospital LA, Los Angeles, CA 90027, USA
    Med Wieku Rozwoj 7:27-34. 2003
    ..This case is being investigated to determine whether the genetic manipulations of the patient's HSC could be the reason for mutagenesis and how other factors could have contributed to this unfortunate event...
  15. ncbi request reprint Advances in lentiviral vector design for gene-modification of hematopoietic stem cells
    Aaron C Logan
    Division of Research Immunology Bone Marrow Transplantation, Children s Hospital Los Angeles, 4650 Sunset Blvd, MS 62, Los Angeles, CA 90027, USA
    Curr Opin Biotechnol 13:429-36. 2002
    ....
  16. ncbi request reprint The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants
    Ami J Shah
    Department of Pediatrics, Keck School of Medicine, Los Angeles, California, USA
    Biol Blood Marrow Transplant 13:584-93. 2007
    ..Further studies are warranted to assess if a lack of difference in infection rates are still demonstrated in larger cohorts...
  17. pmc Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
    Nathalia Holt
    Keck School of Medicine of the University of Southern California, Los Angeles, California, USA
    Nat Biotechnol 28:839-47. 2010
    ..The demonstration that a minority of CCR5(-/-) HSPCs can populate an infected animal with HIV-1-resistant, CCR5(-/-) progeny supports the use of ZFN-modified autologous hematopoietic stem cells as a clinical approach to treating HIV-1...
  18. doi request reprint Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies
    Ami J Shah
    Department of Pediatrics, Division of Research Immunology Bone Marrow Transplantation, Childrens Hospital, Los Angeles, CA 90027, USA
    J Pediatr Hematol Oncol 30:411-8. 2008
    ..Although some patients have acute deficits that appear and improve over time, other patients have progressive declines in neurocognitive function that are chronic...
  19. ncbi request reprint Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT)
    Malinda Lin
    Department of Pediatrics, Keck School of Medicine, Los Angeles, CA, USA
    J Clin Immunol 29:231-7. 2009
    ..The purpose of this study was to evaluate long-term neurodevelopment of patients with SCID following myeloablative chemotherapy and HSCT...
  20. doi request reprint Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency
    Tanja A Gruber
    Divisions of Hematology and Oncology, Children s Hospital Los Angeles, 4650 Sunset Blvd, Los Angeles, CA 90027, USA
    Pediatr Transplant 13:244-50. 2009
    ..These cases highlight the clinical and genetic heterogeneity of OS...
  21. pmc Regulated expansion of human pancreatic beta-cells
    Eszter Pais
    Division of Research Immunology Bone Marrow Transplantation, Department of Pediatrics, Childrens Hospital Los Angeles, Los Angeles, California, USA
    Mol Ther 18:1389-96. 2010
    ..In addition, specific proliferation of human pancreatic beta-cells was induced utilizing this approach. Selective, regulated beta-cell expansion may help to provide greater availability of cells for transplantation in patients with DM...
  22. doi request reprint Neurocognitive function of patients with severe combined immunodeficiency
    Ami J Shah
    Division of Research Immunology Bone Marrow Transplantation, Department of Pediatrics, The Saban Research Institute, Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, 4650 Sunset Boulevard, MS 62, Los Angeles, CA 90027, USA
    Immunol Allergy Clin North Am 30:143-51. 2010
    ....
  23. ncbi request reprint Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication
    Ingrid Bahner
    Division of Research Immunology Bone Marrow Transplantation, The Saban Research Institute of Childrens Hospital Los Angeles, Los Angeles, California, USA
    Mol Ther 15:76-85. 2007
    ..These studies indicate that lentiviral vectors can be made effective for use in gene therapy for HIV-1...
  24. ncbi request reprint Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors
    Jung Eun Jang
    Department of Molecular Microbiology and Immunology, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Stem Cells Dev 15:109-17. 2006
    ..These pseudotyped lentiviral vectors may be valuable tools for efficient, specific and stable gene modification of hESCs...
  25. pmc The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells
    Dennis L Haas
    Division of Research Immunology BMT, Children s Hospital Los Angeles, Los Angeles, California 90027, USA
    J Virol 77:9439-50. 2003
    ....
  26. ncbi request reprint Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging
    Xiuli Wang
    Division of Research Immunology BMT, Department of Radiology, and Congresman Dixon Cellular Imaging Core, Childrens Hospital Los Angeles, 4650 Sunset Blvd, Los Angeles, CA 90027, USA
    Blood 102:3478-82. 2003
    ..In vivo bioluminescence imaging provides a novel method to track the dynamics of engraftment of human HSC and progenitors in vivo...
  27. ncbi request reprint Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells
    Chang I Lee
    California National Primate Research Center, University of California, Davis, California 95616 8542, USA
    Mol Ther 9:112-23. 2004
    ..that the SIN HIV-1-derived lentiviral vectors used in these studies can efficiently transduce rhMSC in vitro (CMV > EF1alpha > PGK) without inhibiting differentiation potential, although the cell cycle was transiently altered at high MOI..
  28. ncbi request reprint Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts
    Mary Kearns-Jonker
    Department of Cardiothoracic Surgery, Children s Hospital of Los Angeles and the University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Transplantation 77:1748-54. 2004
    ..Limitations to the success of this methodology include short-term expression of the introduced gene and rejection of gal hearts transplanted into these animals within a month...
  29. pmc Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration
    Aaron C Logan
    Department of Molecular Microbiology and Immunology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
    J Virol 78:8421-36. 2004
    ..Knowledge of the potential for mobilization of HIV-1-derived SIN lentivectors will be important for the design of future gene therapy trials with such vectors...
  30. ncbi request reprint Factors influencing the titer and infectivity of lentiviral vectors
    Aaron C Logan
    Department of Molecular Microbiology and Immunology, Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA
    Hum Gene Ther 15:976-88. 2004
    ..For the transduction of CD34+ HPCs, we found that the simplest method of increasing vector infectivity is to pseudotype vector particles with the RD114 envelope instead of vesicular stomatitis virus G glycoprotein (VSV-G)...
  31. ncbi request reprint Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta)
    C Chang I Lee
    California National Primate Research Center, University of California, Davis, CA 95616 8542, USA
    Hum Gene Ther 16:417-25. 2005
    ....
  32. ncbi request reprint Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells
    Greg M Podsakoff
    Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 12:77-86. 2005
    ..These findings indicate that there was a selective survival advantage for PBMC containing the huM10 gene during the time of increased HIV-1 load...
  33. ncbi request reprint Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery
    Alice F Tarantal
    California National Primate Research Center, University of California, Davis, CA 95616, USA
    Mol Ther 12:87-98. 2005
    ....
  34. ncbi request reprint Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen
    Noboru Mitsuhashi
    Childrens Hospital of Los Angeles, Department of Cardiothoracic Surgery, Los Angeles, CA 90027, USA
    Blood 107:2286-93. 2006
    ..Mice reconstituted with GalT accepted alphaGal+ heart grafts over 100 days. Transduction with lentiviral vectors results in chimerism at levels sufficient to induce long-term tolerance under nonmyeloablative conditions...
  35. ncbi request reprint Adenosine deaminase gene therapy protocol revisited
    Donald B Kohn
    Division of Research Immunology B M T, Childrens Hospital Los Angeles, Keck U S C School of Medicine, Los Angeles, California 90027, USA
    Mol Ther 5:96-7. 2002

Research Grants12

  1. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2005
    ..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
  2. MND-ADA Transduced CD34 Cells for ADA-SCID
    Donald Kohn; Fiscal Year: 2009
    ..If sufficient gene-modified cells result, and PEG-ADA enzyme replacement therapy can be permanently discontinued, the advantage of this therapeutic approach may change the standard of care for these patients. ..
  3. MND-ADA Transduced CD34 Cells for ADA-SCID
    Donald Kohn; Fiscal Year: 2005
    ..If sufficient gene-modified cells result, and PEG-ADA enzyme replacement therapy can be permanently discontinued, the advantage of this therapeutic approach may change the standard of care for these patients. ..
  4. In Vivo ADA Gene Delivery for the Treatment of SCID
    Donald Kohn; Fiscal Year: 2009
    ..Therefore, it is imperative to continue to develop new and improved methods to treat primary immune deficiency diseases and that is the primary goal of this project have significant efficacy, but remain sub-optimal. ..
  5. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2004
    ..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
  6. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2003
    ..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
  7. LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    Donald Kohn; Fiscal Year: 2002
    ..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
  8. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 2001
    ..Successful tolerance induction in murine and canine models of MPS I will be an Important step toward application of gene therapy in human patients. ..
  9. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 2000
    ..Successful tolerance induction in murine and canine models of MPS I will be an Important step toward application of gene therapy in human patients. ..
  10. GENE THERAPY FOR HURLERS DISEASE
    Donald Kohn; Fiscal Year: 1999
    ..Successful tolerance induction in murine and canine models of MPS I will be an Important step toward application of gene therapy in human patients. ..
  11. In Vivo ADA Gene Delivery for the Treatment of SCID
    Donald B Kohn; Fiscal Year: 2010
    ..Therefore, it is imperative to continue to develop new and improved methods to treat primary immune deficiency diseases and that is the primary goal of this project have significant efficacy, but remain sub-optimal. ..