Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II.

[1]  W. Low,et al.  Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome , 2023, Molecular genetics and metabolism reports.

[2]  L. Belur,et al.  Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS after the Development of Cognitive Dysfunction. , 2022, Human gene therapy.

[3]  L. Belur,et al.  Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells , 2022, Human gene therapy.

[4]  B. Burton,et al.  First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B. , 2022, Molecular therapy : the journal of the American Society of Gene Therapy.

[5]  Hiroshi Kobayashi,et al.  A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy , 2022, Gene Therapy.

[6]  J. Medin,et al.  Autologous, lentivirus‐modified, T‐rapa cell “micropharmacies” for lysosomal storage disorders , 2022, EMBO molecular medicine.

[7]  C. Hampe,et al.  Differences in MPS I and MPS II Disease Manifestations , 2021, International journal of molecular sciences.

[8]  Paweł Zapolnik,et al.  Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities , 2021, International journal of molecular sciences.

[9]  J. Eisengart,et al.  The natural history of neurocognition in MPS disorders: A review. , 2021, Molecular genetics and metabolism.

[10]  K. Minami,et al.  Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice , 2021, Molecular therapy : the journal of the American Society of Gene Therapy.

[11]  T. Ishimoto,et al.  Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation , 2020, Molecular therapy. Methods & clinical development.

[12]  A. M. Watabe,et al.  Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy. , 2020, Molecular genetics and metabolism.

[13]  J. Nolta,et al.  Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector , 2020, The journal of gene medicine.

[14]  Dong Ryul Lee,et al.  T Lymphocyte Development and Activation in Humanized Mouse Model , 2019, Development & reproduction.

[15]  J. Segovia,et al.  Advances in the gene therapy of monogenic blood cell diseases , 2019, Clinical genetics.

[16]  P. Guest,et al.  The Y-Maze for Assessment of Spatial Working and Reference Memory in Mice. , 2018, Methods in molecular biology.

[17]  A. Bradley,et al.  Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements , 2018, Nature Biotechnology.

[18]  C. O’Leary,et al.  Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms , 2018, EMBO molecular medicine.

[19]  S. S. St Martin,et al.  Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.

[20]  W. Low,et al.  Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer. , 2017, Human gene therapy.

[21]  Lothar Hennighausen,et al.  CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome , 2017, Nature Communications.

[22]  A. Biffi.  Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[23]  C. O’Leary,et al.  Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II , 2017, PloS one.

[24]  Wieslawa I. Mentzen,et al.  Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy , 2016, Stem cells translational medicine.

[25]  L. Shultz,et al.  Improved B cell development in humanized NOD‐scid IL2Rγnull mice transgenically expressing human stem cell factor, granulocyte‐macrophage colony‐stimulating factor and interleukin‐3 , 2016, Immunity, inflammation and disease.

[26]  V. Haurigot,et al.  CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome). , 2016, JCI insight.

[27]  H. Azari,et al.  Transplantation of Defined Populations of Differentiated Human Neural Stem Cell Progeny , 2016, Scientific Reports.

[28]  D. Greiner,et al.  Humanized Mouse Models for Transplant Immunology , 2016, American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons.

[29]  R. McIvor,et al.  373. Expression of Human Iduronidase from Sleeping Beauty Engineered Human B Lymphocytes as a Cellular Therapy for Mucopolysaccharidosis Type I , 2015 .

[30]  J. Wolfe,et al.  Ex Vivo Gene Therapy Using Patient iPSC-Derived NSCs Reverses Pathology in the Brain of a Homologous Mouse Model , 2015, Stem cell reports.

[31]  Y. Eto,et al.  Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II. , 2015, Human gene therapy.

[32]  J. Jester,et al.  A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I , 2015, Molecular therapy. Methods & clinical development.

[33]  M. Poe,et al.  Natural Progression of Neurological Disease in Mucopolysaccharidosis Type II , 2011, Pediatrics.

[34]  M. Lieber,et al.  The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway. , 2010, Annual review of biochemistry.

[35]  I. Weissman,et al.  Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis. , 2009, Cell stem cell.

[36]  L. de Meirleir,et al.  Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy , 2007, European Journal of Pediatrics.

[37]  J. Muenzer,et al.  The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) , 2007, Journal of Inherited Metabolic Disease.

[38]  C. Eng,et al.  A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) , 2006, Genetics in Medicine.

[39]  A. Ballabio,et al.  Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery. , 2006, Human molecular genetics.

[40]  M. Kotb,et al.  Human Lymphoid and Myeloid Cell Development in NOD/LtSz-scid IL2Rγnull Mice Engrafted with Mobilized Human Hemopoietic Stem Cells 12 , 2004, The Journal of Immunology.

[41]  A. Amalfitano,et al.  Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model , 2004, Gene Therapy.

[42]  M. Sands,et al.  Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

[43]  M. Sands,et al.  Engraftment of human CD34+ cells leads to widespread distribution of donor-derived cells and correction of tissue pathology in a novel murine xenotransplantation model of lysosomal storage disease. , 2003, Blood.

[44]  J. DaCosta,et al.  Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report , 2002, Acta paediatrica (Oslo, Norway : 1992). Supplement.

[45]  O. V. van Diggelen,et al.  A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease) , 2001, Journal of Inherited Metabolic Disease.

[46]  P. Meikle,et al.  Diagnosis of lysosomal storage disorders: evaluation of lysosome-associated membrane protein LAMP-1 as a diagnostic marker. , 1997, Clinical chemistry.

[47]  R. Latchaw,et al.  Retroviral-mediated transfer of the iduronate-2-sulfatase gene into lymphocytes for treatment of mild Hunter syndrome (mucopolysaccharidosis type II). , 1996, Human gene therapy.

[48]  S. Braun,et al.  Preclinical studies of lymphocyte gene therapy for mild Hunter syndrome (mucopolysaccharidosis type II). , 1996, Human gene therapy.

[49]  C. Barnes.  Memory deficits associated with senescence: a neurophysiological and behavioral study in the rat. , 1979, Journal of comparative and physiological psychology.

[50]  R. Giugliani,et al.  The mucopolysaccharidoses. , 1976, Journal of medical genetics.