Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy.
暂无分享,去创建一个
M. Haskins | K. Ponder | R. Herati | P. O'donnell | M. D’Angelo | V. Knox
[1] M. Haskins,et al. Mechanism of glycosaminoglycan-mediated bone and joint disease: implications for the mucopolysaccharidoses and other connective tissue diseases. , 2008, The American journal of pathology.
[2] J. Muenzer,et al. The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome) , 2007, Journal of Inherited Metabolic Disease.
[3] C. Vite,et al. Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[4] W. Sly,et al. Murine model (Galns(tm(C76S)slu)) of MPS IVA with missense mutation at the active site cysteine conserved among sulfatase proteins. , 2007, Molecular genetics and metabolism.
[5] R. Hoft,et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. , 2007, Molecular genetics and metabolism.
[6] Durval Damiani,et al. Bomba de infusão de insulina em diabetes melito tipo 1 , 2006 .
[7] C. Vite,et al. Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs. , 2006, Molecular genetics and metabolism.
[8] M. Haskins,et al. Joint and Bone Disease in Mucopolysaccharidoses VI and VII: Identification of New Therapeutic Targets and BioMarkers Using Animal Models , 2005, Pediatric Research.
[9] S. Cai,et al. Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[10] M. Poe,et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. , 2004, The New England journal of medicine.
[11] M. Haskins,et al. Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. , 2004, Molecular genetics and metabolism.
[12] S. Schneider,et al. Craniovertebral Instability With Spinal Cord Compression in a 17-Month-Old Boy With Sly Syndrome (Mucopolysaccharidosis Type VII): A Surgical Dilemma , 2004, Spine.
[13] S. Khan,et al. Cervical Cord Compression in an Elderly Patient with Hurler’s Syndrome: A Case Report , 2003, Spine.
[14] S. Ryazantsev,et al. Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB , 2003, Proceedings of the National Academy of Sciences of the United States of America.
[15] G. Aguirre,et al. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs , 2002, Proceedings of the National Academy of Sciences of the United States of America.
[16] T. Koob,et al. Isolation and Identification of the Major Heparan Sulfate Proteoglycans in the Developing Bovine Rib Growth Plate* , 2002, The Journal of Biological Chemistry.
[17] Cuihua Gao,et al. Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.
[18] J. Belmont,et al. Enzyme-replacement therapy in mucopolysaccharidosis I. , 2001, The New England journal of medicine.
[19] M. Fanselow,et al. Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[20] Peter Finamore,et al. A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome). , 1999, Glycobiology.
[21] R. A. Wevers,et al. The frequency of lysosomal storage diseases in The Netherlands , 1999, Human Genetics.
[22] R. Desnick,et al. Identification and characterization of the molecular lesion causing mucopolysaccharidosis type I in cats. , 1999, Molecular genetics and metabolism.
[23] K. Ng,et al. Murine MPS I: insights into the pathogenesis of Hurler syndrome , 1998, Clinical genetics.
[24] J. Fyfe,et al. Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells. , 1998, Genomics.
[25] W. Sly,et al. Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation , 1998, Bone Marrow Transplantation.
[26] F. Jirik,et al. Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene. , 1997, Human molecular genetics.
[27] J. B. Williamson,et al. Spinal problems in mucopolysaccharidosis I (Hurler syndrome). , 1996, The Journal of bone and joint surgery. British volume.
[28] C. Peters,et al. Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VI. , 1996, Proceedings of the National Academy of Sciences of the United States of America.
[29] T. Nakano,et al. A study of the chemical composition of the proximal tibial articular cartilage and growth plate of broiler chickens. , 1995, Poultry science.
[30] M. Mcentee,et al. Enzyme replacement in a canine model of Hurler syndrome. , 1994, Proceedings of the National Academy of Sciences of the United States of America.
[31] W. Sly,et al. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. , 1994, The Journal of clinical investigation.
[32] M. Sands,et al. A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII. , 1993, Proceedings of the National Academy of Sciences of the United States of America.
[33] W. Sly,et al. Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. , 1993, Laboratory investigation; a journal of technical methods and pathology.
[34] E. Neufeld,et al. Architecture of the canine IDUA gene and mutation underlying canine mucopolysaccharidosis I. , 1992, Genomics.
[35] J. Hopwood,et al. Immunolocation analysis of glycosaminoglycans in the human growth plate. , 1992, The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society.
[36] J. Hopwood,et al. Growth-plate chondrocyte cultures for reimplantation into growth-plate defects in sheep. Characterization of cultures. , 1990, Clinical orthopaedics and related research.
[37] M. Davisson,et al. Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency. , 1989, The Journal of clinical investigation.
[38] G. Constantopoulos,et al. Long-term effects of bone marrow transplantation in dogs with mucopolysaccharidosis I. , 1989, The American journal of pathology.
[39] P. Pizzutillo,et al. Atlantoaxial instability in mucopolysaccharidosis type VII. , 1989, Journal of pediatric orthopedics.
[40] R. Shull,et al. Radiographic findings in a canine model of mucopolysaccharidosis I. Changes associated with bone marrow transplantation. , 1988, Investigative radiology.
[41] L. Rosenberg,et al. Localization of a dermatan sulfate proteoglycan (DS-PGII) in cartilage and the presence of an immunologically related species in other tissues. , 1986, The journal of histochemistry and cytochemistry : official journal of the Histochemistry Society.
[42] R. Desnick,et al. β-Glucuronidase Deficiency in a Dog: a Model of Human Mucopolysaccharidosis VII , 1984, Pediatric Research.
[43] C. Dietrich,et al. Chondroitin sulfates of the epiphysial cartilages of different mammals. , 1979, Comparative biochemistry and physiology. B, Comparative biochemistry.
[44] K. Hirschhorn,et al. Spastic quadriparesis due to C1-C2 subluxation in Hurler syndrome. , 1978, The Journal of pediatrics.
[45] R. Stevenson,et al. The iduronidase-deficient mucopolysaccharidoses: clinical and roentgenorgraphic features. , 1976, Pediatrics.
[46] W. Sly,et al. Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis. , 1973, The Journal of pediatrics.
[47] L. Swischuk. The beaked, notched, or hooked vertebra: its significance in infants and young children. , 1970, Radiology.