Mark J Osborn
Affiliation: University of Minnesota
- Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type IMark 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...
- Synthetic zinc finger nuclease design and rapid assemblyMark 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...
- Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene productMark 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...
- Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosaJakub 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...
- 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....
- A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter systemMark 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....
- Sarcoma derived from cultured mesenchymal stem cellsJakub 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...
- Short-term inhibition of p53 combined with keratinocyte growth factor improves thymic epithelial cell recovery and enhances T-cell reconstitution after murine bone marrow transplantationRyan 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...
- Overexpression of murine TSLP impairs lymphopoiesis and myelopoiesisMark 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...
- TALEN-based gene correction for epidermolysis bullosaMark J Osborn
Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA
Mol Ther 21:1151-9. 2013....
- Bone marrow transplantation for recessive dystrophic epidermolysis bullosaJohn 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....