Mark J Osborn

Summary

Affiliation: University of Minnesota
Country: USA

Publications

  1. pmc Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I
    Mark J Osborn
    Department of Pediatrics, Division of Bone Marrow Transplant, University of Minnesota Cancer Center, Minneapolis, Minnesota 55455, USA
    Mol Ther 19:450-60. 2011
  2. pmc Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology
    Mark J Osborn
    Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware St SE, MMC 366, Minneapolis, MN, 55455, USA
    J Inherit Metab Dis . 2016
  3. pmc Synthetic zinc finger nuclease design and rapid assembly
    Mark J Osborn
    University of Minnesota Masonic Cancer Center and Division of Hematology, Oncology, and Blood and Marrow Transplantation, Department of Pediatrics, Minneapolis, MN 55455, USA
    Hum Gene Ther 22:1155-65. 2011
  4. pmc Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa
    Jakub Tolar
    1 Department of Pediatrics, Blood and Marrow Transplant, Medical School, University of Minnesota, Minneapolis, Minnesota, USA 2 Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA
    J Invest Dermatol 134:1246-54. 2014
  5. pmc Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product
    Mark J Osborn
    University of Minnesota Cancer Center, Minneapolis, Minnesota, USA
    Mol Ther 16:1459-66. 2008
  6. pmc Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases
    Mark J Osborn
    Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA
    Mol Ther 24:570-81. 2016
  7. pmc TALEN-based gene correction for epidermolysis bullosa
    Mark J Osborn
    Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA
    Mol Ther 21:1151-9. 2013
  8. pmc Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa
    Jakub Tolar
    Division of Hematology Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota 55455, USA
    J Invest Dermatol 131:848-56. 2011
  9. pmc Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
    Jakub Tolar
    Division of Hematology Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of MN, Minneapolis, MN, USA
    Blood 117:839-47. 2011
  10. pmc Fanconi anemia gene editing by the CRISPR/Cas9 system
    Mark J Osborn
    1 Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455
    Hum Gene Ther 26:114-26. 2015

Collaborators

Detail Information

Publications20

  1. pmc Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I
    Mark J Osborn
    Department of Pediatrics, Division of Bone Marrow Transplant, University of Minnesota Cancer Center, Minneapolis, Minnesota 55455, USA
    Mol Ther 19:450-60. 2011
    ..MC gene delivery combined with costimulatory pathway blockade maximizes safety, efficacy, and sustained gene expression and is a new approach in the treatment of lysosomal storage disease...
  2. pmc Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology
    Mark J Osborn
    Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, 420 Delaware St SE, MMC 366, Minneapolis, MN, 55455, USA
    J Inherit Metab Dis . 2016
    ..These data show a key role for the RAS in MPS associated pathology and support the inclusion of losartan as an augmentation to current therapies...
  3. pmc Synthetic zinc finger nuclease design and rapid assembly
    Mark J Osborn
    University of Minnesota Masonic Cancer Center and Division of Hematology, Oncology, and Blood and Marrow Transplantation, Department of Pediatrics, Minneapolis, MN 55455, USA
    Hum Gene Ther 22:1155-65. 2011
    ..This method, termed CoDA-syn (context-dependent assembly-synthetic), should facilitate a more widespread use of ZFNs in the research community...
  4. pmc Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa
    Jakub Tolar
    1 Department of Pediatrics, Blood and Marrow Transplant, Medical School, University of Minnesota, Minneapolis, Minnesota, USA 2 Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA
    J Invest Dermatol 134:1246-54. 2014
    ..We believe this approach should be the starting point for autologous cellular therapies using 'natural' gene therapy in RDEB and other diseases...
  5. pmc Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product
    Mark J Osborn
    University of Minnesota Cancer Center, Minneapolis, Minnesota, USA
    Mol Ther 16:1459-66. 2008
    ..Short-term treatment resulted in a decrease in GAGs in the cerebellum of mucopolysaccharidosis type I (MPS I) mice. This approach, therefore, represents a potential strategy for the delivery of therapeutic enzyme to the CNS...
  6. pmc Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases
    Mark J Osborn
    Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA
    Mol Ther 24:570-81. 2016
    ....
  7. pmc TALEN-based gene correction for epidermolysis bullosa
    Mark J Osborn
    Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA
    Mol Ther 21:1151-9. 2013
    ....
  8. pmc Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa
    Jakub Tolar
    Division of Hematology Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota 55455, USA
    J Invest Dermatol 131:848-56. 2011
    ..These data identify the potential of RDEB iPS cells to generate autologous hematopoietic grafts and skin cells with the inherent capacity to treat skin and mucosal erosions that typify this genodermatosis...
  9. pmc Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
    Jakub Tolar
    Division of Hematology Oncology, Blood and Marrow Transplantation, Department of Pediatrics, University of MN, Minneapolis, MN, USA
    Blood 117:839-47. 2011
    ....
  10. pmc Fanconi anemia gene editing by the CRISPR/Cas9 system
    Mark J Osborn
    1 Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455
    Hum Gene Ther 26:114-26. 2015
    ..Collectively, we provide proof of principle for precision genome editing in Fanconi anemia, a DNA repair-deficient human disorder. ..
  11. ncbi request reprint A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system
    Mark J Osborn
    Pediatrics, Hematology Oncology, Blood and Marrow Transplant Program, Institute of Human Genetics, Department of Genetics, Cell Biology, and Development, University of Minnesota, Minneapolis, MN 55455, USA
    Mol Ther 12:569-74. 2005
    ....
  12. pmc Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8+ T-cell responses
    Wynette M Dietz
    Department of Laboratory Medicine and Pathology, Center for Immunology and Masonic Cancer Center, University of Minnesota School of Medicine, Minneapolis, Minnesota, USA
    Mol Ther 21:1526-35. 2013
    ..Together, our results suggest intradermal delivery of MC DNA may prove more efficacious for prophylaxis than traditional pDNA vaccines. ..
  13. pmc Short-term inhibition of p53 combined with keratinocyte growth factor improves thymic epithelial cell recovery and enhances T-cell reconstitution after murine bone marrow transplantation
    Ryan M Kelly
    Division of Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN 55455, USA
    Blood 115:1088-97. 2010
    ..Thus, transient p53 inhibition combined with KGF represents a novel and potentially translatable approach to promote rapid and durable thymic and peripheral T-cell recovery after BMT...
  14. ncbi request reprint Sarcoma derived from cultured mesenchymal stem cells
    Jakub Tolar
    Department of Pediatrics, Division of Hematology Oncology, Blood and Marrow Transplant and Cancer Center, University of Minnesota Medical School, Minneapolis, Minnesota, USA
    Stem Cells 25:371-9. 2007
    ..More importantly, our study indicates that sarcoma can evolve from MSC cultures...
  15. ncbi request reprint Overexpression of murine TSLP impairs lymphopoiesis and myelopoiesis
    Mark J Osborn
    Department of Laboratory Medicine and Pathology, Division of Medical Technology, University of Minnesota, BSBE 6 118, 312 Church Street SE, Minneapolis, MN 55455, USA
    Blood 103:843-51. 2004
    ..The arrest in lymphopoiesis and the expansion of myeloid progenitor cells in TSLP transgenic mice suggest that TSLP has negative and positive regulatory effects on lymphoid and myeloid development, respectively...
  16. pmc Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa
    Christopher Perdoni
    Stem Cell Institute, University of Minnesota, Minneapolis, Minn Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minn
    Transl Res 168:50-8. 2016
    ..Here, we review gene delivery and editing platforms and their application toward the development of next-generation treatments designed to correct the causative genetic defects of RDEB. ..
  17. doi request reprint Gene editing and its application for hematological diseases
    Mark J Osborn
    University of Minnesota Twin Cities, Minneapolis, MN, USA
    Int J Hematol 104:18-28. 2016
    ....
  18. pmc B7-H3 expression in donor T cells and host cells negatively regulates acute graft-versus-host disease lethality
    Rachelle G Veenstra
    Department of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota Cancer Center, Minneapolis, MN
    Blood 125:3335-46. 2015
    ....
  19. pmc Targeting G with TAL effectors: a comparison of activities of TALENs constructed with NN and NK repeat variable di-residues
    Michelle L Christian
    Department of Genetics, Cell Biology and Development and Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, United States of America
    PLoS ONE 7:e45383. 2012
    ..TALENs with only 18 aa after the repeat array, however, showed a clear optimum for spacers of 13 to 16 bp. The data presented here provide useful guidelines for increasing the specificity and activity of engineered TAL effector proteins...
  20. pmc Bone marrow transplantation for recessive dystrophic epidermolysis bullosa
    John E Wagner
    Blood and Marrow Transplant Program, Department of Pediatrics, University of Minnesota, Minneapolis, MN 55455, USA
    N Engl J Med 363:629-39. 2010
    ....