Genomes and Genes
Affiliation: Cincinnati Children's Hospital Medical Center
- Improved gene transfer and normalized enzyme levels in primitive hematopoietic progenitors from patients with mucopolysaccharidosis type I using a bioreactorDao Pan
Gene Therapy Program, Department of Pediatrics, and Institute of Human Genetics, University of Minnesota, Minneapolis, MN, USA
J Gene Med 6:1293-303. 2004..Other inadequacies of current transduction protocols are related to their multi-step procedures, e.g., using tissue-culture flasks, roller bottles or gas-permeable bags for clinical application...
- Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndromeDao Pan
Molecular and Gene Therapy Program, Division of Experimental Hematology, Cincinnati Children s Research Foundation, Cincinnati, OH 45249, USA
Brain Res 1188:241-53. 2008..This study would also provide guidelines for the experimental designs of behavioral evaluation on innovative therapies for the treatment of MPS type I...
- Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidosesDao Pan
Molecular and Gene Therapy Program, Division of Experimental Hematology, Cincinnati Children s Hospital Medical Center, OH 45229, USA
Curr Pharm Biotechnol 12:884-96. 2011..Several neurological findings in CNS pathophysiology emerged with therapeutic investigation will also be discussed...
- Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase geneSeth D Hartung
Gene Therapy Program, Institute of Human Genetics, Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, MN 55455, USA
Mol Ther 9:866-75. 2004..We conclude that AAV-mediated transduction of the IDUA gene in newborn Idua(-/-) mice was sufficient to have a major curative impact on several of the most important parameters of the disease...
- Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndromeDaren Wang
Division of Experimental Hematology and Cancer Biology, Cincinnati Children s Hospital Medical Center, Cincinnati, OH 45229, USA
Proc Natl Acad Sci U S A 106:19958-63. 2009..This approach provides a paradigm for the utilization of RBC precursors as a depot for efficient and potentially safer systemic delivery of nonsecreted proteins by ex vivo HSC gene transfer...
- In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vectorD Nicole Worsham
Division of Experimental Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45249, USA
Mol Ther 14:514-24. 2006..This approach could lead to a novel application for treatment of human diseases...
- Co-expression of MGMT(P140K) and alpha-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correctionDaren Wang
Cell and Molecular Therapy Program, and Division of Experimental Hematology, Cincinnati Children s Hospital Medical Center, Cincinnati, OH, USA
J Gene Med 10:249-59. 2008..It has been shown that an O(6)-methylguanine-DNA-methyltransferase variant (MGMT(P140K)) mediated in vivo selection of transduced hematopoietic stem cells (HSC) in animals...
- Craniofacial abnormalities in a murine knock-out model of mucopolysaccharidosis I H: a computed tomography and anatomic studyPatrick Graupman
Department of Neurosurgery, University of Minnesota, 420 Delaware Street SE, Minneapolis, MN 55455, USA
J Craniofac Surg 15:392-8. 2004..This information now provides researchers with objective data from living Hurler syndrome-affected mice that will allow them to follow therapies directed at improving craniofacial outcomes for any therapy over time...
- K-Cl cotransporter gene expression during human and murine erythroid differentiationDao Pan
Molecular and Cell Therapy Program, Division of Experimental Hematology, Cincinnati Children s Hospital Medical Center, Cincinnati, Ohio 45229, USA
J Biol Chem 286:30492-503. 2011..The results suggest that KCC3 is the dominant isoform in erythrocytes, with variable expression of KCC1 and KCC4 among individuals that could result in modulation of KCC activity...
- In situ (in vivo) gene transfer into murine bone marrow stem cellsDao Pan
Division of Experimental Hematology, Cincinnati Children s Research Foundation, Cincinnati, OH, USA
Methods Mol Biol 506:159-69. 2009..This approach may provide a novel application for treatment of human diseases, and represent an interesting new tool to study adult stem cell plasticity and the nature of unperturbed hematopoiesis...
- Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrowDao Pan
Department of Pediatrics and Institute of Human Genetics, University of Minnesota, Minneapolis, Minnesota 55455, USA
Mol Ther 6:19-29. 2002..The observation of bone marrow transduction after intravenous vector administration suggests the possibility of an in vivo approach to stem cell gene therapy...
- Rac1 and Rac2 GTPases are necessary for early erythropoietic expansion in the bone marrow but not in the spleenTheodosia A Kalfa
Division of Hematology Oncology, Oncology, Cincinnati Children s Research Foundation, Cincinnati Children s Hospital Medical Center, MLC 7015 Cincinnati, OH 45229 3039, USA
Haematologica 95:27-35. 2010..The role of these Rac GTPases in erythropoiesis has not yet been fully elucidated...
- Genetic Therapy for CNS Manifestations in MPS I via BBB-targeted Protein DeliveryDao Pan; Fiscal Year: 2009..abstract_text> ..
- Genetic Therapy for CNS Manifestations in MPS I via BBB-targeted Protein DeliveryDao Pan; Fiscal Year: 2010..The studies described in this application will open the door to novel approaches for the treatment of neurological disorders-from MPS type I to major public health concerns such as stroke and Alzheimer's disease. ..
- In Vivo BM Stem Cell Gene Transfer for MPS type IDao Pan; Fiscal Year: 2006..Taken together, these data would not only open a door to a novel approach for treatment of human diseases, but also provide a new tool to study adult stem cell plasticity and the nature of hematopoiesis. ..