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Mark S. Sands - Publications

Affiliations: 
Washington University, Saint Louis, St. Louis, MO 
Area:
Molecular genetics
Website:
http://dbbs.wustl.edu/DBBS/website.nsf/RIB/5EE729EE796AEAE086256D4E005B2DAF

119 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2019 Li Y, Xu Y, Benitez BA, Nagree MS, Dearborn JT, Jiang X, Guzman MA, Woloszynek JC, Giaramita A, Yip BK, Elsbernd J, Babcock MC, Lo M, Fowler SC, Wozniak DF, ... ... Sands MS, et al. Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target. Proceedings of the National Academy of Sciences of the United States of America. PMID 31527255 DOI: 10.1073/pnas.1912108116  0.52
2018 Lange J, Haslett LJ, Lloyd-Evans E, Pocock JM, Sands MS, Williams BP, Cooper JD. Compromised astrocyte function and survival negatively impact neurons in infantile neuronal ceroid lipofuscinosis. Acta Neuropathologica Communications. 6: 74. PMID 30089511 DOI: 10.1186/s40478-018-0575-4  0.4
2018 Sidhu R, Mikulka CR, Fujiwara H, Sands MS, Schaffer JE, Ory DS, Jiang X. A HILIC-MS/MS Method for Simultaneous Quantification of the Lysosomal Disease Markers Galactosylsphingosine and Glucosylsphingosine in Mouse Serum. Biomedical Chromatography : Bmc. PMID 29516569 DOI: 10.1002/bmc.4235  0.52
2018 Le SQ, Kan SH, Clarke D, Sanghez V, Egeland M, Vondrak KN, Doherty TM, Vera MU, Iacovino M, Cooper JD, Sands MS, Dickson PI. A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I. Molecular Therapy. Methods & Clinical Development. 8: 42-51. PMID 29159202 DOI: 10.1016/j.omtm.2017.09.008  0.4
2017 Shyng C, Nelvagal HR, Dearborn JT, Tyynelä J, Schmidt RE, Sands MS, Cooper JD. Synergistic effects of treating the spinal cord and brain in CLN1 disease. Proceedings of the National Academy of Sciences of the United States of America. PMID 28673981 DOI: 10.1073/pnas.1701832114  1
2017 Shyng C, Macauley SL, Dearborn JT, Sands MS. Widespread Expression of a Membrane-Tethered Version of the Soluble Lysosomal Enzyme Palmitoyl Protein Thioesterase-1. Jimd Reports. PMID 28213849 DOI: 10.1007/8904_2017_1  1
2015 Benitez BA, Cairns NJ, Schmidt RE, Morris JC, Norton JB, Cruchaga C, Sands MS. Clinically early-stage CSPα mutation carrier exhibits remarkable terminal stage neuronal pathology with minimal evidence of synaptic loss. Acta Neuropathologica Communications. 3: 73. PMID 26610600 DOI: 10.1186/s40478-015-0256-5  0.32
2015 Dearborn JT, Ramachandran S, Shyng C, Lu JY, Thornton J, Hofmann SL, Sands MS. Histochemical localization of palmitoyl protein thioesterase-1 activity. Molecular Genetics and Metabolism. PMID 26597320 DOI: 10.1016/j.ymgme.2015.11.004  1
2015 Dearborn JT, Harmon SK, Fowler SC, O'Malley KL, Taylor GT, Sands MS, Wozniak DF. Comprehensive functional characterization of murine infantile Batten disease including Parkinson-like behavior and dopaminergic markers. Scientific Reports. 5: 12752. PMID 26238334 DOI: 10.1038/srep12752  1
2015 Hawkins-Salsbury JA, Shea L, Jiang X, Hunter DA, Guzman AM, Reddy AS, Qin EY, Li Y, Gray SJ, Ory DS, Sands MS. Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 35: 6495-505. PMID 25904800 DOI: 10.1523/JNEUROSCI.4199-14.2015  1
2014 Sands MS. Mucopolysaccharidosis type VII: A powerful experimental system and therapeutic challenge. Pediatric Endocrinology Reviews : Per. 12: 159-65. PMID 25345098  1
2014 Cohen IJ, Baris HN, Mistry PK, Sands MS. Treatment for Lsds: real options for several diseases. Forward. Pediatric Endocrinology Reviews : Per. 12: 71. PMID 25345087  1
2014 Macauley SL, Wong AM, Shyng C, Augner DP, Dearborn JT, Pearse Y, Roberts MS, Fowler SC, Cooper JD, Watterson DM, Sands MS. An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 34: 13077-82. PMID 25253854 DOI: 10.1523/JNEUROSCI.2518-14.2014  1
2014 Li Y, Sands MS. Experimental therapies in the murine model of globoid cell leukodystrophy. Pediatric Neurology. 51: 600-6. PMID 25240259 DOI: 10.1016/j.pediatrneurol.2014.08.003  1
2014 Shyng C, Sands MS. Astrocytosis in infantile neuronal ceroid lipofuscinosis: friend or foe? Biochemical Society Transactions. 42: 1282-5. PMID 25233404 DOI: 10.1042/BST20140188  1
2014 Sands MS. A Hitchhiker's guide to the blood-brain barrier: in trans delivery of a therapeutic enzyme. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 483-4. PMID 24584077 DOI: 10.1038/mt.2014.12  1
2014 Swain GP, Prociuk M, Bagel JH, O'Donnell P, Berger K, Drobatz K, Gurda BL, Haskins ME, Sands MS, Vite CH. Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain. Gene Therapy. 21: 28-36. PMID 24131981 DOI: 10.1038/gt.2013.54  1
2014 Reddy AS, Patel JR, Vogler C, Klein RS, Sands MS. Central nervous system pathology progresses independently of KC and CXCR2 in globoid-cell leukodystrophy. Plos One. 8: e64647. PMID 23755134 DOI: 10.1371/journal.pone.0064647  1
2013 Sands MS. Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease). Journal of Child Neurology. 28: 1151-8. PMID 24014510 DOI: 10.1177/0883073813495960  1
2013 Hawkins-Salsbury JA, Parameswar AR, Jiang X, Schlesinger PH, Bongarzone E, Ory DS, Demchenko AV, Sands MS. Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture. Journal of Lipid Research. 54: 3303-11. PMID 24006512 DOI: 10.1194/jlr.M039610  1
2013 Hawkins-Salsbury JA, Cooper JD, Sands MS. Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease). Biochimica Et Biophysica Acta. 1832: 1906-9. PMID 23747979 DOI: 10.1016/j.bbadis.2013.05.026  1
2013 Sands MS. Farber disease: understanding a fatal childhood disorder and dissecting ceramide biology. Embo Molecular Medicine. 5: 799-801. PMID 23666771 DOI: 10.1002/emmm.201302781  1
2013 Heldermon CD, Qin EY, Ohlemiller KK, Herzog ED, Brown JR, Vogler C, Hou W, Orrock JL, Crawford BE, Sands MS. Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. Gene Therapy. 20: 913-21. PMID 23535899 DOI: 10.1038/gt.2013.14  1
2012 Reddy AS, Wozniak DF, Farber NB, Dearborn JT, Fowler SC, Sands MS. Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy. Journal of Clinical Medicine. 1: 1-14. PMID 24013457 DOI: 10.3390/jcm1010001  0.88
2012 Hawkins-Salsbury JA, Qin EY, Reddy AS, Vogler CA, Sands MS. Oxidative stress as a therapeutic target in globoid cell leukodystrophy. Experimental Neurology. 237: 444-52. PMID 22849820 DOI: 10.1016/j.expneurol.2012.07.013  1
2012 Qin EY, Hawkins-Salsbury JA, Jiang X, Reddy AS, Farber NB, Ory DS, Sands MS. Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy. Molecular Genetics and Metabolism. 107: 186-96. PMID 22704480 DOI: 10.1016/j.ymgme.2012.05.021  1
2012 Young MA, Larson DE, Sun CW, George DR, Ding L, Miller CA, Lin L, Pawlik KM, Chen K, Fan X, Schmidt H, Kalicki-Veizer J, Cook LL, Swift GW, Demeter RT, ... ... Sands MS, et al. Background mutations in parental cells account for most of the genetic heterogeneity of induced pluripotent stem cells. Cell Stem Cell. 10: 570-82. PMID 22542160 DOI: 10.1016/j.stem.2012.03.002  1
2012 Macauley SL, Roberts MS, Wong AM, McSloy F, Reddy AS, Cooper JD, Sands MS. Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis. Annals of Neurology. 71: 797-804. PMID 22368049 DOI: 10.1002/ana.23545  1
2012 Roberts MS, Macauley SL, Wong AM, Yilmas D, Hohm S, Cooper JD, Sands MS. Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis. Journal of Inherited Metabolic Disease. 35: 847-57. PMID 22310926 DOI: 10.1007/s10545-011-9446-x  1
2011 Benitez BA, Alvarado D, Cai Y, Mayo K, Chakraverty S, Norton J, Morris JC, Sands MS, Goate A, Cruchaga C. Exome-sequencing confirms DNAJC5 mutations as cause of adult neuronal ceroid-lipofuscinosis. Plos One. 6: e26741. PMID 22073189 DOI: 10.1371/journal.pone.0026741  1
2011 Sands MS. AAV-mediated liver-directed gene therapy. Methods in Molecular Biology (Clifton, N.J.). 807: 141-57. PMID 22034029 DOI: 10.1007/978-1-61779-370-7_6  1
2011 Macauley SL, Pekny M, Sands MS. The role of attenuated astrocyte activation in infantile neuronal ceroid lipofuscinosis. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 31: 15575-85. PMID 22031903 DOI: 10.1523/JNEUROSCI.3579-11.2011  1
2011 Reddy AS, Kim JH, Hawkins-Salsbury JA, Macauley SL, Tracy ET, Vogler CA, Han X, Song SK, Wozniak DF, Fowler SC, Klein RS, Sands MS. Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 31: 9945-57. PMID 21734286 DOI: 10.1523/JNEUROSCI.1802-11.2011  1
2011 Linterman KS, Palmer DN, Kay GW, Barry LA, Mitchell NL, McFarlane RG, Black MA, Sands MS, Hughes SM. Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease. Human Gene Therapy. 22: 1011-20. PMID 21595499 DOI: 10.1089/hum.2011.026  1
2011 Hawkins-Salsbury JA, Reddy AS, Sands MS. Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts? Human Molecular Genetics. 20: R54-60. PMID 21421999 DOI: 10.1093/hmg/ddr112  1
2010 Parameswar AR, Hawkins JA, Mydock LK, Sands MS, Demchenko AV. Concise Synthesis of the Unnatural Sphingosine and Psychosine Enantiomer. European Journal of Organic Chemistry. 2010. PMID 24371429 DOI: 10.1002/ejoc.201000024  1
2010 McIver SR, Muccigrosso M, Gonzales ER, Lee JM, Roberts MS, Sands MS, Goldberg MP. Oligodendrocyte degeneration and recovery after focal cerebral ischemia. Neuroscience. 169: 1364-75. PMID 20621643 DOI: 10.1016/j.neuroscience.2010.04.070  1
2010 Heldermon CD, Ohlemiller KK, Herzog ED, Vogler C, Qin E, Wozniak DF, Tan Y, Orrock JL, Sands MS. Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 873-80. PMID 20179679 DOI: 10.1038/mt.2010.17  1
2010 Sands MS. Combination therapy for globoid cell leukodystrophy International Journal of Clinical Pharmacology and Therapeutics. 48: S33-S34.  1
2009 Woloszynek JC, Kovacs A, Ohlemiller KK, Roberts M, Sands MS. Metabolic adaptations to interrupted glycosaminoglycan recycling. The Journal of Biological Chemistry. 284: 29684-91. PMID 19700765 DOI: 10.1074/jbc.M109.020818  1
2009 Hofling AA, Kim JH, Fantz CR, Sands MS, Song SK. Diffusion tensor imaging detects axonal injury and demyelination in the spinal cord and cranial nerves of a murine model of globoid cell leukodystrophy. Nmr in Biomedicine. 22: 1100-6. PMID 19650072 DOI: 10.1002/nbm.1420  1
2009 Kielar C, Wishart TM, Palmer A, Dihanich S, Wong AM, Macauley SL, Chan CH, Sands MS, Pearce DA, Cooper JD, Gillingwater TH. Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease. Human Molecular Genetics. 18: 4066-80. PMID 19640925 DOI: 10.1093/hmg/ddp355  1
2009 Macauley SL, Wozniak DF, Kielar C, Tan Y, Cooper JD, Sands MS. Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouse. Experimental Neurology. 217: 124-35. PMID 19416667 DOI: 10.1016/j.expneurol.2009.01.022  1
2009 Macauley SL, Sands MS. Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases. Experimental Neurology. 218: 5-8. PMID 19361502 DOI: 10.1016/j.expneurol.2009.03.040  1
2008 Kolber BJ, Roberts MS, Howell MP, Wozniak DF, Sands MS, Muglia LJ. Central amygdala glucocorticoid receptor action promotes fear-associated CRH activation and conditioning. Proceedings of the National Academy of Sciences of the United States of America. 105: 12004-9. PMID 18695245 DOI: 10.1073/pnas.0803216105  1
2008 Zhang JR, Coleman T, Langmade SJ, Scherrer DE, Lane L, Lanier MH, Feng C, Sands MS, Schaffer JE, Semenkovich CF, Ory DS. Niemann-Pick C1 protects against atherosclerosis in mice via regulation of macrophage intracellular cholesterol trafficking. The Journal of Clinical Investigation. 118: 2281-90. PMID 18483620 DOI: 10.1172/JCI32561  1
2008 Meyerrose TE, Roberts M, Ohlemiller KK, Vogler CA, Wirthlin L, Nolta JA, Sands MS. Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease. Stem Cells (Dayton, Ohio). 26: 1713-22. PMID 18436861 DOI: 10.1634/stemcells.2008-0008  1
2008 Sands MS, Haskins ME. CNS-directed gene therapy for lysosomal storage diseases. Acta Paediatrica (Oslo, Norway : 1992). Supplement. 97: 22-7. PMID 18339183 DOI: 10.1111/j.1651-2227.2008.00660.x  1
2008 Hess DA, Craft TP, Wirthlin L, Hohm S, Zhou P, Eades WC, Creer MH, Sands MS, Nolta JA. Widespread nonhematopoietic tissue distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity. Stem Cells (Dayton, Ohio). 26: 611-20. PMID 18055447 DOI: 10.1634/stemcells.2007-0429  1
2008 Galvin N, Vogler C, Levy B, Kovacs A, Griffey M, Sands MS. A murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera. Pediatric and Developmental Pathology : the Official Journal of the Society For Pediatric Pathology and the Paediatric Pathology Society. 11: 185-92. PMID 17990914 DOI: 10.2350/07-03-0242.1  1
2007 Woloszynek JC, Coleman T, Semenkovich CF, Sands MS. Lysosomal dysfunction results in altered energy balance. The Journal of Biological Chemistry. 282: 35765-71. PMID 17911106 DOI: 10.1074/jbc.M705124200  1
2007 Heldermon CD, Hennig AK, Ohlemiller KK, Ogilvie JM, Herzog ED, Breidenbach A, Vogler C, Wozniak DF, Sands MS. Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B. Plos One. 2: e772. PMID 17712420 DOI: 10.1371/journal.pone.0000772  1
2007 Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS. AAV vector integration sites in mouse hepatocellular carcinoma. Science (New York, N.Y.). 317: 477. PMID 17656716 DOI: 10.1126/science.1142658  1
2007 Donsante A, Levy B, Vogler C, Sands MS. Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII. Journal of Inherited Metabolic Disease. 30: 227-38. PMID 17308887 DOI: 10.1007/s10545-007-0483-4  1
2007 Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands MS. Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 44-52. PMID 17164774 DOI: 10.1038/sj.mt.6300026  1
2007 Kielar C, Maddox L, Bible E, Pontikis CC, Macauley SL, Griffey MA, Wong M, Sands MS, Cooper JD. Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis. Neurobiology of Disease. 25: 150-62. PMID 17046272 DOI: 10.1016/j.nbd.2006.09.001  1
2007 Meyerrose TE, De Ugarte DA, Hofling AA, Herrbrich PE, Cordonnier TD, Shultz LD, Eagon JC, Wirthlin L, Sands MS, Hedrick MA, Nolta JA. In vivo distribution of human adipose-derived mesenchymal stem cells in novel xenotransplantation models. Stem Cells (Dayton, Ohio). 25: 220-7. PMID 16960135 DOI: 10.1634/stemcells.2006-0243  1
2006 Miller DG, Wang PR, Petek LM, Hirata RK, Sands MS, Russell DW. Gene targeting in vivo by adeno-associated virus vectors. Nature Biotechnology. 24: 1022-6. PMID 16878127 DOI: 10.1038/nbt1231  1
2006 Sharpe EE, Teleron AA, Li B, Price J, Sands MS, Alford K, Young PP. The origin and in vivo significance of murine and human culture-expanded endothelial progenitor cells. The American Journal of Pathology. 168: 1710-21. PMID 16651636 DOI: 10.2353/ajpath.2006.050556  1
2006 Sands MS, Davidson BL. Gene therapy for lysosomal storage diseases. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 839-49. PMID 16545619 DOI: 10.1016/j.ymthe.2006.01.006  1
2006 Griffey MA, Wozniak D, Wong M, Bible E, Johnson K, Rothman SM, Wentz AE, Cooper JD, Sands MS. CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 538-47. PMID 16364693 DOI: 10.1016/j.ymthe.2005.11.008  1
2005 Zhao H, Kitaura H, Sands MS, Ross FP, Teitelbaum SL, Novack DV. Critical role of beta3 integrin in experimental postmenopausal osteoporosis. Journal of Bone and Mineral Research : the Official Journal of the American Society For Bone and Mineral Research. 20: 2116-23. PMID 16294265 DOI: 10.1359/JBMR.050724  1
2005 McIver SR, Lee CS, Lee JM, Green SH, Sands MS, Snider BJ, Goldberg MP. Lentiviral transduction of murine oligodendrocytes in vivo. Journal of Neuroscience Research. 82: 397-403. PMID 16158420 DOI: 10.1002/jnr.20626  1
2005 Lin D, Fantz CR, Levy B, Rafi MA, Vogler C, Wenger DA, Sands MS. AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 422-30. PMID 15996520 DOI: 10.1016/j.ymthe.2005.04.019  1
2005 Griffey M, Macauley SL, Ogilvie JM, Sands MS. AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 413-21. PMID 15979943 DOI: 10.1016/j.ymthe.2005.04.018  1
2004 Hofling AA, Sands MS, Lublin DM, Bauer G, Devine S. Collection of a mobilized peripheral blood apheresis product from a patient with mucopolysaccharidosis type VII and subsequent CD34+ cell isolation. Journal of Clinical Apheresis. 19: 151-3. PMID 15493049 DOI: 10.1002/jca.20018  1
2004 Kitaura H, Sands MS, Aya K, Zhou P, Hirayama T, Uthgenannt B, Wei S, Takeshita S, Novack DV, Silva MJ, Abu-Amer Y, Ross FP, Teitelbaum SL. Marrow stromal cells and osteoclast precursors differentially contribute to TNF-alpha-induced osteoclastogenesis in vivo. Journal of Immunology (Baltimore, Md. : 1950). 173: 4838-46. PMID 15470024  1
2004 Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS. AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 106-16. PMID 15233947 DOI: 10.1016/j.ymthe.2004.03.018  1
2004 Xu L, O'Malley T, Sands MS, Wang B, Meyerrose T, Haskins ME, Ponder KP. In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 37-44. PMID 15233940 DOI: 10.1016/j.ymthe.2004.04.010  1
2004 Hofling AA, Devine S, Vogler C, Sands MS. Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. 9: 856-65. PMID 15194052 DOI: 10.1016/j.ymthe.2004.03.013  1
2004 Griffey M, Bible E, Vogler C, Levy B, Gupta P, Cooper J, Sands MS. Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis. Neurobiology of Disease. 16: 360-9. PMID 15193292 DOI: 10.1016/j.nbd.2004.03.005  1
2004 Young PP, Fantz CR, Sands MS. VEGF disrupts the neonatal blood-brain barrier and increases life span after non-ablative BMT in a murine model of congenital neurodegeneration caused by a lysosomal enzyme deficiency. Experimental Neurology. 188: 104-14. PMID 15191807 DOI: 10.1016/j.expneurol.2004.03.007  1
2004 Mango RL, Xu L, Sands MS, Vogler C, Seiler G, Schwarz T, Haskins ME, Ponder KP. Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. Molecular Genetics and Metabolism. 82: 4-19. PMID 15110316 DOI: 10.1016/j.ymgme.2004.01.015  1
2004 Woloszynek JC, Roberts M, Coleman T, Vogler C, Sly W, Semenkovich CF, Sands MS. Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII. The Biochemical Journal. 379: 461-9. PMID 14705966 DOI: 10.1042/BJ20031048  1
2003 Hennig AK, Levy B, Ogilvie JM, Vogler CA, Galvin N, Bassnett S, Sands MS. Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 23: 3302-7. PMID 12716937  1
2003 Young PP, Vogler C, Hofling AA, Sands MS. Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. 7: 52-61. PMID 12573618 DOI: 10.1016/S1525-0016(02)00016-5  1
2003 Xu L, Gao C, Sands MS, Cai SR, Nichols TC, Bellinger DA, Raymer RA, McCorquodale S, Ponder KP. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood. 101: 3924-32. PMID 12531787 DOI: 10.1182/blood-2002-10-3050  1
2003 Hofling AA, Vogler C, Creer MH, Sands MS. 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. Blood. 101: 2054-63. PMID 12406886 DOI: 10.1182/blood-2002-08-2597  1
2002 Monahan PE, Jooss K, Sands MS. Safety of adeno-associated virus gene therapy vectors: a current evaluation. Expert Opinion On Drug Safety. 1: 79-91. PMID 12904163 DOI: 10.1517/14740338.1.1.79  1
2002 Xu L, Mango RL, Sands MS, Haskins ME, Ellinwood NM, Ponder KP. Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 745-58. PMID 12498771 DOI: 10.1006/mthe.2002.0809  1
2002 Young PP, Hofling AA, Sands MS. VEGF increases engraftment of bone marrow-derived endothelial progenitor cells (EPCs) into vasculature of newborn murine recipients. Proceedings of the National Academy of Sciences of the United States of America. 99: 11951-6. PMID 12195016 DOI: 10.1073/pnas.182215799  1
2002 Ohlemiller KK, Hennig AK, Lett JM, Heidbreder AF, Sands MS. Inner ear pathology in the mucopolysaccharidosis VII mouse. Hearing Research. 169: 69-84. PMID 12121741 DOI: 10.1016/S0378-5955(02)00341-6  1
2002 Monroy MA, Ross FP, Teitelbaum SL, Sands MS. Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease. Bone. 30: 352-9. PMID 11856642 DOI: 10.1016/S8756-3282(01)00679-2  1
2001 Vogler C, Barker J, Sands MS, Levy B, Galvin N, Sly WS. Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease. Pediatric and Developmental Pathology : the Official Journal of the Society For Pediatric Pathology and the Paediatric Pathology Society. 4: 421-33. PMID 11779044 DOI: 10.1007/s10024001-0079-1  1
2001 Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Therapy. 8: 1343-6. PMID 11571571 DOI: 10.1038/sj.gt.3301541  1
2001 Daly TM, Ohlemiller KK, Roberts MS, Vogler CA, Sands MS. Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Therapy. 8: 1291-8. PMID 11571565 DOI: 10.1038/sj.gt.3301420  1
2001 Sands MS, Vogler CA, Ohlemiller KK, Roberts MS, Grubb JH, Levy B, Sly WS. Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII. The Journal of Biological Chemistry. 276: 43160-5. PMID 11562370 DOI: 10.1074/jbc.M107778200  1
2001 Frisella WA, O'Connor LH, Vogler CA, Roberts M, Walkley S, Levy B, Daly TM, Sands MS. Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Molecular Therapy : the Journal of the American Society of Gene Therapy. 3: 351-8. PMID 11273777 DOI: 10.1006/mthe.2001.0274  1
2001 Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, Sands MS, Parker Ponder K. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Human Gene Therapy. 12: 563-73. PMID 11268288 DOI: 10.1089/104303401300042500  1
2001 Vogler C, Levy B, Galvin N, Sands MS, Birkenmeier EH, Sly WS, Barker J. A novel model of murine mucopolysaccharidosis type VII due to an intracisternal a particle element transposition into the beta-glucuronidase gene: clinical and pathologic findings. Pediatric Research. 49: 342-8. PMID 11228259 DOI: 10.1203/00006450-200103000-00007  1
2000 Wolfe JH, Sands MS, Harel N, Weil MA, Parente MK, Polesky AC, Reilly JJ, Hasson C, Weimelt S, Haskins ME. Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII. Molecular Therapy : the Journal of the American Society of Gene Therapy. 2: 552-61. PMID 11124056 DOI: 10.1006/mthe.2000.0202  1
2000 Ross CJ, Bastedo L, Maier SA, Sands MS, Chang PL. Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells. Human Gene Therapy. 11: 2117-27. PMID 11044913 DOI: 10.1089/104303400750001426  1
2000 Ohlemiller KK, Vogler CA, Roberts M, Galvin N, Sands MS. Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation. Experimental Eye Research. 71: 469-81. PMID 11040082 DOI: 10.1006/exer.2000.0897  1
2000 Gao C, Sands MS, Haskins ME, Ponder KP. Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 2: 233-44. PMID 10985954 DOI: 10.1006/mthe.2000.0121  1
2000 Sands MS, Barker JE. Percutaneous intravenous injection in neonatal mice. Comparative Medicine. 50: 107. PMID 10857000  1
2000 Daly TM, Lorenz RG, Sands MS. Abnormal immune function in vivo in a murine model of lysosomal storage disease. Pediatric Research. 47: 757-62. PMID 10832733  1
2000 Young PP, Sands MS. Bone marrow endothelial progenitor cells give rise to neovasculature in a nonablative newborn murine transplant model Blood. 96: 276a.  1
2000 Alex Hofling A, Haug JS, Sands MS. Engraftment of human hematopoietic stem cells in a novel murine xenotransplantation system Blood. 96: 71a.  1
2000 Sands MS, Barker JE. Letter to the editor Comparative Medicine. 50: 107.  1
1999 Freeman BJ, Roberts MS, Vogler CA, Nicholes A, Hofling AA, Sands MS. Behavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII. Blood. 94: 2142-50. PMID 10477745  1
1999 Sands MS, Barker JE. Percutaneous intravenous injection in neonatal mice. Laboratory Animal Science. 49: 328-30. PMID 10403452  1
1999 Vogler C, Levy B, Galvin NJ, Thorpe C, Sands MS, Barker JE, Baty J, Birkenmeier EH, Sly WS. Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy. Pediatric Research. 45: 838-44. PMID 10367775 DOI: 10.1203/00006450-199906000-00010  1
1999 Daly TM, Vogler C, Levy B, Haskins ME, Sands MS. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proceedings of the National Academy of Sciences of the United States of America. 96: 2296-300. PMID 10051635 DOI: 10.1073/pnas.96.5.2296  1
1999 Daly TM, Okuyama T, Vogler C, Haskins ME, Muzyczka N, Sands MS. Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. Human Gene Therapy. 10: 85-94. PMID 10022533 DOI: 10.1089/10430349950019219  1
1998 Daly TM, Sands MS. Gene therapy for lysosomal storage diseases. Expert Opinion On Investigational Drugs. 7: 1673-82. PMID 15991909 DOI: 10.1517/13543784.7.10.1673  1
1998 Gwynn B, Lueders K, Sands MS, Birkenmeier EH. Intracisternal A-particle element transposition into the murine beta-glucuronidase gene correlates with loss of enzyme activity: a new model for beta-glucuronidase deficiency in the C3H mouse. Molecular and Cellular Biology. 18: 6474-81. PMID 9774663  1
1998 Vogler C, Sands MS, Galvin N, Levy B, Thorpe C, Barker J, Sly WS. Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease. Journal of Inherited Metabolic Disease. 21: 575-86. PMID 9728337 DOI: 10.1023/A:1005423222927  1
1998 O'Connor LH, Erway LC, Vogler CA, Sly WS, Nicholes A, Grubb J, Holmberg SW, Levy B, Sands MS. Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function. The Journal of Clinical Investigation. 101: 1394-400. PMID 9525982 DOI: 10.1172/JCI1773  0.44
1997 Sands MS, Wolfe JH, Birkenmeier EH, Barker JE, Vogler C, Sly WS, Okuyama T, Freeman B, Nicholes A, Muzyczka N, Chang PL, Axelrod HR. Gene therapy for murine mucopolysaccharidosis type VII. Neuromuscular Disorders : Nmd. 7: 352-60. PMID 9267850 DOI: 10.1016/S0960-8966(97)00061-8  1
1997 Sands MS, Vogler C, Torrey A, Levy B, Gwynn B, Grubb J, Sly WS, Birkenmeier EH. Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation. The Journal of Clinical Investigation. 99: 1596-605. PMID 9120003 DOI: 10.1172/JCI119322  1
1996 Vogler C, Sands MS, Levy B, Galvin N, Birkenmeier EH, Sly WS. Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival. Pediatric Research. 39: 1050-4. PMID 8725268 DOI: 10.1203/00006450-199606000-00019  1
1995 Wolfe JH, Kyle JW, Sands MS, Sly WS, Markowitz DG, Parente MK. High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector. Gene Therapy. 2: 70-8. PMID 7712336  1
1995 Sands MS, Erway LC, Vogler C, Sly WS, Birkenmeier EH. Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII. Blood. 86: 2033-40. PMID 7655032  1
1994 Wolfe JH, Taylor RM, Sands MS, Haskins ME, Patterson DF, Snyder EY. Mucopolysaccharidosis type VII as a model system for gene transfer to the central nervous system. Gene Therapy. 1: S55. PMID 8542400  1
1994 Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, Birkenmeier EH. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. The Journal of Clinical Investigation. 93: 2324-31. PMID 8200966 DOI: 10.1172/JCI117237  1
1994 Bastedo L, Sands MS, Lambert DT, Pisa MA, Birkenmeier E, Chang PL. Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII. The Journal of Clinical Investigation. 94: 1180-6. PMID 8083358 DOI: 10.1172/JCI117434  1
1993 Sands MS, Barker JE, Vogler C, Levy B, Gwynn B, Galvin N, Sly WS, Birkenmeier E. Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Laboratory Investigation; a Journal of Technical Methods and Pathology. 68: 676-86. PMID 8515654  1
1993 Sands MS, Birkenmeier EH. A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII. Proceedings of the National Academy of Sciences of the United States of America. 90: 6567-71. PMID 8101990  1
1992 Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, Vogler CA, Birkenmeier EH. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature. 360: 749-53. PMID 1465145 DOI: 10.1038/360749a0  1
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