| Year |
Citation |
Score |
| 2023 |
Takahashi K, Eultgen EM, Wang SH, Rensing NR, Nelvagal HR, Dearborn JT, Danos O, Buss N, Sands MS, Wong M, Cooper JD. Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model. The Journal of Clinical Investigation. PMID 37104037 DOI: 10.1172/JCI165908 |
0.332 |
|
| 2022 |
Nelvagal HR, Eaton SL, Wang SH, Eultgen EM, Takahashi K, Le SQ, Nesbitt R, Dearborn JT, Siano N, Puhl AC, Dickson PI, Thompson G, Murdoch F, Brennan PM, Gray M, ... ... Sands MS, et al. Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep. The Journal of Clinical Investigation. PMID 36040802 DOI: 10.1172/JCI163107 |
0.399 |
|
| 2021 |
Babcock MC, Mikulka CR, Wang B, Chandriani S, Chandra S, Xu Y, Webster K, Feng Y, Nelvagal HR, Giaramita A, Yip BK, Lo M, Jiang X, Chao Q, Woloszynek JC, ... ... Sands MS, et al. Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor. Scientific Reports. 11: 14486. PMID 34262084 DOI: 10.1038/s41598-021-93601-1 |
0.798 |
|
| 2021 |
Bradbury AM, Bongarzone ER, Sands MS. Krabbe Disease: New Hope for an Old Disease. Neuroscience Letters. 135841. PMID 33766733 DOI: 10.1016/j.neulet.2021.135841 |
0.317 |
|
| 2021 |
Heller GJ, Marshall MS, Issa Y, Marshall JN, Nguyen D, Rue E, Pathmasiri KC, Domowicz MS, van Breemen RB, Tai LM, Cologna SM, Crocker SJ, Givogri MI, Sands MS, Bongarzone ER. Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33508430 DOI: 10.1016/j.ymthe.2021.01.026 |
0.32 |
|
| 2020 |
Li Y, Miller CA, Shea LK, Jiang X, Guzman MA, Chandler RJ, Ramakrishnan SM, Smith SN, Venditti CP, Vogler CA, Ory DS, Ley TJ, Sands MS. Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33388420 DOI: 10.1016/j.ymthe.2020.12.031 |
0.386 |
|
| 2020 |
Nelvagal HR, Hurtado ML, Eaton SL, Kline RA, Lamont DJ, Sands MS, Wishart TM, Cooper JD. Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease. Scientific Reports. 10: 15157. PMID 32938982 DOI: 10.1038/s41598-020-72075-7 |
0.41 |
|
| 2020 |
Nelvagal HR, Dearborn JT, Ostergaard JR, Sands MS, Cooper JD. Spinal manifestations of CLN1 disease start during the early postnatal period. Neuropathology and Applied Neurobiology. PMID 32841420 DOI: 10.1111/nan.12658 |
0.35 |
|
| 2020 |
Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, et al. Krabbe disease successfully treated via monotherapy of intrathecal gene therapy. The Journal of Clinical Investigation. PMID 32773406 DOI: 10.1172/Jci133953 |
0.666 |
|
| 2020 |
Mikulka CR, Dearborn JT, Benitez BA, Strickland A, Liu L, Milbrandt J, Sands MS. Cell-autonomous expression of the acid hydrolase galactocerebrosidase. Proceedings of the National Academy of Sciences of the United States of America. PMID 32253319 DOI: 10.1073/Pnas.1917675117 |
0.43 |
|
| 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 |
0.811 |
|
| 2019 |
Sapkota D, Lake AM, Yang W, Yang C, Wesseling H, Guise A, Uncu C, Dalal JS, Kraft AW, Lee JM, Sands MS, Steen JA, Dougherty JD. Cell-Type-Specific Profiling of Alternative Translation Identifies Regulated Protein Isoform Variation in the Mouse Brain. Cell Reports. 26: 594-607.e7. PMID 30650354 DOI: 10.1016/J.Celrep.2018.12.077 |
0.329 |
|
| 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 |
0.352 |
|
| 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 |
0.342 |
|
| 2018 |
Nelvagal HR, Shyng C, Sands MS, Cooper JD. Early gait abnormalities relate to brainstem and spinal cord pathology in CLN1 disease Molecular Genetics and Metabolism. 123: S104. DOI: 10.1016/J.Ymgme.2017.12.275 |
0.765 |
|
| 2018 |
Mikulka CR, Sands MS. Eliminating cross-correction allows for cell-specific expression of the lysosomal enzyme galactocerebrosidase in the twitcher mouse Molecular Genetics and Metabolism. 123: S95. DOI: 10.1016/j.ymgme.2017.12.249 |
0.335 |
|
| 2017 |
Benitez BA, Sands MS. Primary fibroblasts from CSPα mutation carriers recapitulate hallmarks of the adult onset neuronal ceroid lipofuscinosis. Scientific Reports. 7: 6332. PMID 28740222 DOI: 10.1038/S41598-017-06710-1 |
0.335 |
|
| 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 |
0.794 |
|
| 2017 |
Chandler RJ, Sands M, Venditti CP. rAAV integration and genotoxicity: insights from animal models. Human Gene Therapy. PMID 28293963 DOI: 10.1089/Hum.2017.009 |
0.391 |
|
| 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 |
0.79 |
|
| 2017 |
Sanghez V, Kan S, Le SQ, Clarke D, Vondrak KN, Vera M, Iacovino M, Sands M, Dickson P. Characterization on the cellular distribution of enzyme replacement therapy (ERT) under the influence of humoral immune response in MPS I mouse model Molecular Genetics and Metabolism. 120: S118. DOI: 10.1016/j.ymgme.2016.11.305 |
0.337 |
|
| 2016 |
Hu P, Li Y, Nikolaishvili-Feinberg N, Scesa G, Bi Y, Pan D, Moore D, Bongarzone ER, Sands MS, Miller R, Kafri T. Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects. Journal of Neuroscience Research. 94: 1152-68. PMID 27638600 DOI: 10.1002/Jnr.23847 |
0.44 |
|
| 2016 |
Mikulka CR, Sands MS. Treatment for Krabbe's disease: Finding the combination. Journal of Neuroscience Research. 94: 1126-37. PMID 27638598 DOI: 10.1002/jnr.23822 |
0.452 |
|
| 2016 |
Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS. Insights into the Pathogenesis and Treatment of Krabbe Disease. Pediatric Endocrinology Reviews : Per. 13: 689-96. PMID 27491217 |
0.626 |
|
| 2016 |
Kan SH, Le SQ, Bui QD, Benedict B, Cushman J, Sands MS, Dickson PI. Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behavioural Brain Research. PMID 27340089 DOI: 10.1016/J.Bbr.2016.06.023 |
0.34 |
|
| 2016 |
Montaño AM, Lock-Hock N, Steiner RD, Graham BH, Szlago M, Greenstein R, Pineda M, Gonzalez-Meneses A, Çoker M, Bartholomew D, Sands MS, Wang R, Giugliani R, Macaya A, Pastores G, et al. Clinical course of sly syndrome (mucopolysaccharidosis type VII). Journal of Medical Genetics. PMID 26908836 DOI: 10.1136/Jmedgenet-2015-103322 |
0.332 |
|
| 2016 |
Sanghez V, Kan S, Le SQ, Clarke D, Vondrak K, Vera MU, Iacovino M, Sands MS, Dickson PI. 430. Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type I Molecular Therapy. 24: S170-S171. DOI: 10.1016/S1525-0016(16)33239-7 |
0.355 |
|
| 2016 |
Shyng C, Nelvagal HR, Dearborn JT, Cooper JD, Sands MS. 27. Therapeutic Efficacy of Intracranial and Intrathecal AAV2/9-PPT1 in Infantile Batten Disease Molecular Therapy. 24: S12-S13. DOI: 10.1016/S1525-0016(16)32836-2 |
0.81 |
|
| 2016 |
Li Y, Shea LK, Jiang X, Gray SJ, Ory DS, Sands MS. Combination therapy increases lifespan and improves clinicobehavioral performance in the murine model of globoid cell leukodystrophy Molecular Genetics and Metabolism. 117: S73. DOI: 10.1016/J.Ymgme.2015.12.338 |
0.615 |
|
| 2016 |
Cooper J, Dmytrus J, Sri S, Dearborn J, Sands M, Lu J, Wang L, Hofmann S, Nelvagal H. Progressive neuropathology in the spinal cord of a mouse model of CLN1 disease and assessing the efficacy of intrathecal enzyme replacement therapy (ERT) Molecular Genetics and Metabolism. 117: S36. DOI: 10.1016/J.Ymgme.2015.12.226 |
0.379 |
|
| 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 |
0.314 |
|
| 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 |
0.817 |
|
| 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 |
0.401 |
|
| 2015 |
Hu P, Li Y, Sands MS, McCown T, Kafri T. Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors. Molecular Therapy. Methods & Clinical Development. 2: 15025. PMID 26229972 DOI: 10.1038/Mtm.2015.25 |
0.311 |
|
| 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 |
0.748 |
|
| 2014 |
Sands MS. Mucopolysaccharidosis type VII: A powerful experimental system and therapeutic challenge. Pediatric Endocrinology Reviews : Per. 12: 159-65. PMID 25345098 |
0.361 |
|
| 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 |
0.815 |
|
| 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 |
0.459 |
|
| 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 |
0.809 |
|
| 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 |
0.389 |
|
| 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 |
0.429 |
|
| 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 |
0.637 |
|
| 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 |
0.422 |
|
| 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: 10.1016/J.Ymgme.2012.11.108 |
0.336 |
|
| 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 |
0.47 |
|
| 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 |
0.426 |
|
| 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 |
0.415 |
|
| 2013 |
Heldermon CD, Hennig AK, Ohlemiller KK, Ogilvie JM, Herzog ED, Breidenbach A, Vogler C, Wozniak DF, Sands MS. Older MPS IIIB mice are impaired on the rocking rotarod. Plos One. DOI: 10.1371/Journal.Pone.0000772.G001 |
0.304 |
|
| 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 |
0.577 |
|
| 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 |
0.549 |
|
| 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 |
0.641 |
|
| 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 |
0.613 |
|
| 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 |
0.431 |
|
| 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 |
0.315 |
|
| 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 |
0.396 |
|
| 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 |
0.438 |
|
| 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 |
0.608 |
|
| 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 |
0.466 |
|
| 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 |
0.593 |
|
| 2011 |
Macauley S, Roberts M, Hohms S, Reddy A, Cooper J, Sands M. Therapeutic approaches for the treatment of Infantile Neuronal Ceroid Lipofuscinosis (INCL) Molecular Genetics and Metabolism. 102: S27. DOI: 10.1016/j.ymgme.2010.11.090 |
0.47 |
|
| 2011 |
Hawkins J, Reddy AS, Sands MS. Oxidative stress as a therapeutic target in GLD Molecular Genetics and Metabolism. 102: S20. DOI: 10.1016/J.Ymgme.2010.11.069 |
0.605 |
|
| 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 |
0.566 |
|
| 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 |
0.416 |
|
| 2010 |
Hawkins J, Parameswar A, Mydock L, Demchenko A, Han X, Sands M. 57. Mechanisms of psychosine toxicity in globoid cell leukodystrophy Molecular Genetics and Metabolism. 99: S19-S20. DOI: 10.1016/J.Ymgme.2009.10.074 |
0.557 |
|
| 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 |
0.793 |
|
| 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 |
0.77 |
|
| 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 |
0.319 |
|
| 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 |
0.411 |
|
| 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 |
0.382 |
|
| 2009 |
Sands M, Woloszynek J, Ohlemiller K, Kovacs A. 120. Metabolic and clinical adaptations to interrupted lysosomal recycling Molecular Genetics and Metabolism. 96: S38. DOI: 10.1016/J.Ymgme.2008.11.121 |
0.746 |
|
| 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 |
0.334 |
|
| 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 |
0.445 |
|
| 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 |
0.46 |
|
| 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 |
0.374 |
|
| 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 |
0.799 |
|
| 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 |
0.799 |
|
| 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 |
0.407 |
|
| 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 |
0.713 |
|
| 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 |
0.747 |
|
| 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 |
0.778 |
|
| 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 |
0.758 |
|
| 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 |
0.797 |
|
| 2007 |
Heldermon C, Sands M. Encouragement of Transplacental Stem Cell Transplantation. Blood. 110: 1202-1202. DOI: 10.1182/blood.v110.11.1202.1202 |
0.407 |
|
| 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 |
0.363 |
|
| 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 |
0.337 |
|
| 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 |
0.387 |
|
| 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 |
0.796 |
|
| 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 |
0.333 |
|
| 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 |
0.439 |
|
| 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 |
0.785 |
|
| 2005 |
Hess DA, Wirthlin L, Craft TC, Herrbrich PE, Roberts M, Creer MH, Sands MS, Nolta JA. Transplanted Human Aldehyde Dehydrogenase Expressing Stem Cells Circulate and Engraft Nonhematopoietic Tissues. Blood. 106: 1693-1693. DOI: 10.1182/Blood.V106.11.1693.1693 |
0.394 |
|
| 2005 |
Hess DA, Craft TP, Wirthlin L, Herrbrich PE, Hofling AA, Sands MS, Nolta JA. 460. Transplantation of Human Aldehyde Dehydrogenase Expressing Cells Leads to Widespread Tissue Distribution of Donor Cells in the Pancreas and Liver of NOD/SCID/MPSVII Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.468 |
0.429 |
|
| 2005 |
Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands M. 424. Intracranial AAV5 Synergizes with Non-Myeloablative Bone Marrow Transplantation in the Murine Model of Globoid-Cell Leukodystrophy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.426 |
0.778 |
|
| 2005 |
Herrbrich PE, Meyerrose TE, Hofling AA, Sands M, Nolta JA. 243. Transplantation of Neonatal NOD/SCID/MPSVII Mice with Human Mesenchymal Stem Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.246 |
0.45 |
|
| 2005 |
Donsante A, Levy B, Vogler C, Sands M. 237. Evaluation of Low-Level, Constitutive Expression of |[beta]|-glucuronidase on the Clinical Manifestations of Mucopolysaccharidosis Type VII Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.240 |
0.761 |
|
| 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 |
0.788 |
|
| 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 DOI: 10.4049/Jimmunol.173.8.4838 |
0.325 |
|
| 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 |
0.418 |
|
| 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 |
0.445 |
|
| 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 |
0.817 |
|
| 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 |
0.794 |
|
| 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 |
0.434 |
|
| 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 |
0.409 |
|
| 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 |
0.823 |
|
| 2004 |
Hess DA, Craft TP, Wirthlin L, Herrbrich PE, Hofling AA, Sands MS, Nolta JA. Transplantation of Human Aldehyde Dehydrogenase Expressing Cells Leads to Widespread Tissue Distribution of Donor Cells in the Novel NOD/SCID/MPSVII Xenotransplantation Model. Blood. 104: 3601-3601. DOI: 10.1182/Blood.V104.11.3601.3601 |
0.803 |
|
| 2004 |
Griffey M, Wozniak D, Wong M, Rothman S, Bible E, Cooper J, Vogler C, Sands M. AAV-mediated gene therapy causes functional improvements in a murine model of infantile neuronal ceroid lipofuscinosis (INCL, Batten disease) Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.065 |
0.788 |
|
| 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 DOI: 10.1523/Jneurosci.23-08-03302.2003 |
0.453 |
|
| 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 |
0.807 |
|
| 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 |
0.395 |
|
| 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 |
0.809 |
|
| 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 |
0.352 |
|
| 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 |
0.472 |
|
| 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 |
0.767 |
|
| 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 |
0.351 |
|
| 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 |
0.383 |
|
| 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 |
0.474 |
|
| 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 |
0.731 |
|
| 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 |
0.347 |
|
| 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 |
0.344 |
|
| 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 |
0.385 |
|
| 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 |
0.357 |
|
| 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 |
0.445 |
|
| 2001 |
Frisella WA, O'Connor LH, Vogler CA, Roberts M, Walkley SU, Levy B, Daly TM, Sands MS. Erratum: Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII (Molecular Therapy (2001) vol. 3 (3) (351-358)) Molecular Therapy. 3. DOI: 10.1006/Mthe.2001.0349 |
0.309 |
|
| 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 |
0.402 |
|
| 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 |
0.391 |
|
| 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 |
0.374 |
|
| 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 DOI: 10.1203/00006450-200006000-00012 |
0.43 |
|
| 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 |
0.814 |
|
| 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 |
0.446 |
|
| 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 |
0.462 |
|
| 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 |
0.413 |
|
| 1999 |
Freeman BJ, Roberts MS, Vogler CA, Nicholes A, Hofling AA, Sands MS. Behavior and Therapeutic Efficacy of β-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII Blood. 94: 2142-2150. DOI: 10.1182/Blood.V94.6.2142.418K16_2142_2150 |
0.451 |
|
| 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 DOI: 10.1128/Mcb.18.11.6474 |
0.347 |
|
| 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 |
0.496 |
|
| 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 |
0.472 |
|
| 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 |
0.426 |
|
| 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 |
0.426 |
|
| 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 |
0.317 |
|
| 1996 |
Walter J, You Q, Hagstrom JN, Sands M, High KA. Successful expression of human factor IX following repeat administration of an adenoviral vector in mice Proceedings of the National Academy of Sciences of the United States of America. 93: 3056-3061. PMID 8610167 DOI: 10.1073/Pnas.93.7.3056 |
0.37 |
|
| 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 DOI: 10.1182/Blood.V86.5.2033.Bloodjournal8652033 |
0.404 |
|
| 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 |
0.441 |
|
| 1994 |
Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, Sly WS. Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediatric Research. 34: 837-40. PMID 8108204 DOI: 10.1203/00006450-199312000-00028 |
0.354 |
|
| 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 |
0.412 |
|
| 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 |
0.3 |
|
| 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 DOI: 10.1073/Pnas.90.14.6567 |
0.311 |
|
| 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 |
0.472 |
|
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