Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

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

[2]  Yasuyuki Suzuki,et al.  Growth impairment in mucopolysaccharidoses. , 2018, Molecular genetics and metabolism.

[3]  Hiroshi Kobayashi,et al.  Chaperone effect of sulfated disaccharide from heparin on mutant iduronate-2-sulfatase in mucopolysaccharidosis type II. , 2017, Molecular genetics and metabolism.

[4]  T. Ishimoto,et al.  Co-deteriorations of anisotropic extracellular matrix arrangement and intrinsic mechanical property in c-src deficient osteopetrotic mouse femur. , 2017, Bone.

[5]  C. Hendriksz,et al.  Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients , 2017, Molecular genetics and metabolism reports.

[6]  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.

[7]  S. Byers,et al.  Reversal of established bone pathology in MPS VII mice following lentiviral-mediated gene therapy. , 2016, Molecular genetics and metabolism.

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

[9]  Y. Eto,et al.  A method for measuring disease-specific iduronic acid from the non-reducing end of glycosaminoglycan in mucopolysaccharidosis type II mice. , 2016, Molecular genetics and metabolism.

[10]  M. Schweizer,et al.  Impaired bone remodeling and its correction by combination therapy in a mouse model of mucopolysaccharidosis-I. , 2015, Human molecular genetics.

[11]  T. Bateman,et al.  Single‐Limb Irradiation Induces Local and Systemic Bone Loss in a Murine Model , 2015, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

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

[13]  S. Byers,et al.  Lentiviral-mediated gene therapy results in sustained expression of β-glucuronidase for up to 12 months in the gus(mps/mps) and up to 18 months in the gus(tm(L175F)Sly) mouse models of mucopolysaccharidosis type VII. , 2014, Human gene therapy.

[14]  H. Nakauchi,et al.  Enzyme augmentation therapy enhances the therapeutic efficacy of bone marrow transplantation in mucopolysaccharidosis type II mice. , 2014, Molecular genetics and metabolism.

[15]  T. Martin,et al.  Historically significant events in the discovery of RANK/RANKL/OPG. , 2013, World journal of orthopedics.

[16]  L. Hofbauer,et al.  The promotion of osteoclastogenesis by sulfated hyaluronan through interference with osteoprotegerin and receptor activator of NF-κB ligand/osteoprotegerin complex formation. , 2013, Biomaterials.

[17]  C. von Kalle,et al.  Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy , 2013, Science.

[18]  Luca Biasco,et al.  Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome , 2013, Science.

[19]  R. Steiner,et al.  Skeletal abnormalities in lysosomal storage diseases. , 2013, Pediatric endocrinology reviews : PER.

[20]  W. Sly,et al.  Assessment of bone dysplasia by micro-CT and glycosaminoglycan levels in mouse models for mucopolysaccharidosis type I, IIIA, IVA, and VII , 2013, Journal of Inherited Metabolic Disease.

[21]  Yohei Sato,et al.  Massive Accumulation of Glycosaminoglycans in the Aortic Valve of a Patient With Hunter Syndrome During Enzyme Replacement Therapy , 2013, Pediatric Cardiology.

[22]  S. Gerson,et al.  Chemoselection of allogeneic HSC after murine neonatal transplantation without myeloablation or post-transplant immunosuppression. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[23]  Y. Eto,et al.  Administration of anti-CD3 antibodies modulates the immune response to an infusion of α-glucosidase in mice. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[24]  Y. Eto,et al.  Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II. , 2012, Molecular genetics and metabolism.

[25]  B. Bigger,et al.  Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice. , 2012, Molecular therapy : the journal of the American Society of Gene Therapy.

[26]  S. Byers,et al.  Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII. , 2012, Molecular genetics and metabolism.

[27]  R. Petty,et al.  Musculoskeletal manifestations of mucopolysaccharidoses. , 2011, Rheumatology.

[28]  K. White Orthopaedic aspects of mucopolysaccharidoses. , 2011, Rheumatology.

[29]  N. Anagnou,et al.  The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia , 2011, Stem cells international.

[30]  A. Ficcadenti,et al.  Brain and spine MRI features of Hunter disease: frequency, natural evolution and response to therapy , 2011, Journal of Inherited Metabolic Disease.

[31]  C. Eng,et al.  Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome , 2011, Genetics in Medicine.

[32]  L. Naldini,et al.  Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. , 2010, Blood.

[33]  M. Saito,et al.  Raloxifene ameliorates detrimental enzymatic and nonenzymatic collagen cross-links and bone strength in rabbits with hyperhomocysteinemia , 2010, Osteoporosis International.

[34]  M. Saito,et al.  Collagen cross-links as a determinant of bone quality: a possible explanation for bone fragility in aging, osteoporosis, and diabetes mellitus , 2010, Osteoporosis International.

[35]  Manfred Schmidt,et al.  Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy , 2009, Science.

[36]  M. Cosma,et al.  IDS crossing of the blood-brain barrier corrects CNS defects in MPSII mice. , 2009, American journal of human genetics.

[37]  T. Nakano,et al.  The combination therapy with alfacalcidol and risedronate improves the mechanical property in lumbar spine by affecting the material properties in an ovariectomized rat model of osteoporosis , 2009, BMC musculoskeletal disorders.

[38]  Y. Bertrand,et al.  Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17 years. , 2009, The Journal of pediatrics.

[39]  D. Chappard,et al.  Bone mass and microarchitecture of irradiated and bone marrow-transplanted mice: influences of the donor strain , 2009, Osteoporosis International.

[40]  E. Mayatepek,et al.  Neurological findings in Hunter disease: Pathology and possible therapeutic effects reviewed , 2008, Journal of Inherited Metabolic Disease.

[41]  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.

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

[43]  Yan Cui,et al.  Lentiviral vectors with CMV or MHCII promoters administered in vivo: immune reactivity versus persistence of expression. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

[44]  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.

[45]  P. Delmas,et al.  Bone quality--the material and structural basis of bone strength and fragility. , 2006, The New England journal of medicine.

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

[47]  A. Tessitore,et al.  Scientific category: CHEMOKINES Chemokine-induced recruitment of genetically modified bone marrow cells into the CNS of GM1-gangliosidosis mice corrects neuronal pathology , 2013 .

[48]  R. Moats,et al.  Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.

[49]  Y. Tabata,et al.  Unique alignment and texture of biological apatite crystallites in typical calcified tissues analyzed by microbeam X-ray diffractometer system. , 2002, Bone.

[50]  C. Elliger,et al.  Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

[51]  Susan R. Johnson,et al.  Osteoporosis prevention, diagnosis, and therapy. , 2001, JAMA.

[52]  E. Young,et al.  Long-term follow-up following bone marrow transplantation for Hunter disease , 1999, Journal of Inherited Metabolic Disease.

[53]  T Jämsä,et al.  Comparison of three-point bending test and peripheral quantitative computed tomography analysis in the evaluation of the strength of mouse femur and tibia. , 1998, Bone.

[54]  J. Rutledge,et al.  Bone marrow transplantation in Hunter syndrome. , 1996, The Journal of pediatrics.

[55]  L. Lockman,et al.  Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation , 1995, Journal of Inherited Metabolic Disease.

[56]  O. Gabrielli,et al.  Bone marrow transplantation in Hunter syndrome , 1995, Journal of Inherited Metabolic Disease.

[57]  M. Drezner,et al.  Bone histomorphometry: Standardization of nomenclature, symbols, and units: Report of the asbmr histomorphometry nomenclature committee , 1987, Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research.

[58]  M. Valsecchi,et al.  Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I. , 2015, Blood.

[59]  Yasuhiro Kobayashi,et al.  Roles of Wnt signals in bone resorption during physiological and pathological states , 2012, Journal of Molecular Medicine.

[60]  Y. Eto,et al.  Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II). , 2010, Molecular genetics and metabolism.