Oncology Division
Alphabetical list (active faculty):   
Mark S. Sands

Mark S. Sands, PhD


Department of Medicine

Oncology Division

Stem Cell Biology

Department of Genetics

Research Interests

  • Lysosomal storage diseases
  • Neurodegenerative disorders
  • Gene therapy
  • Enzyme replacement
  • Hematopoiesis
  • Bone marrow transplantation


  • 314-362-5494 (office)
  • 314-362-5496 (lab)
  • 314-747-5770 (fax)
  • Division of Oncology
    Mail Stop 8007-0066-05
    Washington University
    660 South Euclid Avenue
    St. Louis, MO 63110
  • Room 5509, 4444 Forest Park (office)
  • Room 5510, 4444 Forest Park (lab)

Peer-reviewed Manuscripts

  • Preclinical Studies in Krabbe Disease: A Model for the Investigation of Novel Combination Therapies for Lysosomal Storage Diseases
    Heller G, Bradbury AM, Sands MS, Bongarzone ER
    Mol Ther 2023 Jan 4;31(1):7-23
  • Cross-Species Efficacy of Enzyme Replacement Therapy for CLN1 Disease in Mice and Sheep
    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, Greenhalgh SN, Tennant P, Gregson R, Clutton E, Nixon J, Proudfoot C, Guido S, Lillico SG, Whitelaw CBA, Lu JY, Hofmann SL, Ekins S, Sands MS, Wishart TM, Cooper JD
    J Clin Invest 2022 Oct 17;132(20):e163107
  • Effects of Chronic Cannabidiol in a Mouse Model of Naturally Occurring Neuroinflammation, Neurodegeneration, and Spontaneous Seizures
    Dearborn JT, Nelvagal HR, Rensing NR, Takahashi K, Hughes SM, Wishart TM, Cooper JD, Wong M, Sands MS
    Sci Rep 2022 Jul 4;12(1):11286
  • CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine Beta-Galactosyl-Ceramidase Gene Re-Capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease
    Rebiai R, Rue E, Zaldua S, Nguyen D, Scesa G, Jastrzebski M, Foster R, Wang B, Jiang X, Tai L, Brady ST, van Breemen R, Givogri MI, Sands MS, Bongarzone ER
    Front Mol Neurosci 2022 May 10;15:896314
  • Substrate Reduction Therapy for Krabbe Disease and Metachromatic Leukodystrophy Using a Novel Ceramide Galactosyltransferase Inhibitor
    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, Shen Y, Bhagwat S, Sands MS, Crawford BE
    Sci Rep 2021 Jul 14;11(1):14486
  • Biochemical Evaluation of Intracerebroventricular RhNAGLU-IGF2 Enzyme Replacement Therapy in Neonatal Mice With Sanfilippo B Syndrome
    Kan SH, Elsharkawi I, Le SQ, Prill H, Mangini L, Cooper JD, Lawrence R, Sands MS, Crawford BE, Dickson PI
    Mol Genet Metab 2021 Jun;133(2):185-192
  • Waning Efficacy in a Long-Term AAV-Mediated Gene Therapy Study in the Murine Model of Krabbe Disease
    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
    Mol Ther 2021 May 5;29(5):1883-1902
  • Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
    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
    Mol Ther 2021 Feb 3;29(2):691-701
  • Spinal Manifestations of CLN1 Disease Start During the Early Postnatal Period
    Nelvagal HR, Dearborn JT, Ostergaard JR, Sands MS, Cooper JD
    Neuropathol Appl Neurobiol 2021 Feb;47(2):251-267
  • Comparative Proteomic Profiling Reveals Mechanisms for Early Spinal Cord Vulnerability in CLN1 Disease
    Nelvagal HR, Hurtado ML, Eaton SL, Kline RA, Lamont DJ, Sands MS, Wishart TM, Cooper JD
    Sci Rep 2020 Sep 16;10(1):15157
  • Macrophage Secretion of miR-106b-5p Causes Renin-Dependent Hypertension
    Oh J, Matkovich SJ, Riek AE, Bindom SM, Shao JS, Head RD, Barve RA, Sands MS, Carmeliet G, Osei-Owusu P, Knutsen RH, Zhang H, Blumer KJ, Nichols CG, Mecham RP, Baldan A, Benitez BA, Sequeira-Lopez ML, Gomez RA, Bernal-Mizrachi C
    Nat Commun 2020 Sep 23;11(1):4798
  • Krabbe Disease Successfully Treated Via Monotherapy of Intrathecal Gene Therapy
    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, Shelton GD, Bongarzone ER, Gray SJ, Vite CH
    J Clin Invest 2020 Sep 1;130(9):4906-4920
  • Cell-Autonomous Expression of the Acid Hydrolase Galactocerebrosidase
    Mikulka CR, Dearborn JT, Benitez BA, Strickland A, Liu L, Milbrandt J, Sands MS
    Proc Natl Acad Sci U S A 2020 Apr 21;117(16):9032-9041
  • Genetic Ablation of Acid Ceramidase in Krabbe Disease Confirms the Psychosine Hypothesis and Identifies a New Therapeutic Target
    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, Vogler CA, Medin JA, Crawford BE, Sands MS
    Proc Natl Acad Sci U S A 2019 Oct 1;116(40):20097-20103
  • Cell-Type-Specific Profiling of Alternative Translation Identifies Regulated Protein Isoform Variation in the Mouse Brain
    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 Rep 2019 Jan 15;26(3):594-607
  • Compromised Astrocyte Function and Survival Negatively Impact Neurons in Infantile Neuronal Ceroid Lipofuscinosis
    Lange J, Haslett LJ, Lloyd-Evans E, Pocock JM, Sands MS, Williams BP, Cooper JD
    Acta Neuropathol Commun 2018 Aug 8;6(1):74
  • A HILIC-MS/MS Method for Simultaneous Quantification of the Lysosomal Disease Markers Galactosylsphingosine and Glucosylsphingosine in Mouse Serum
    Sidhu R, Mikulka CR, Fujiwara H, Sands MS, Schaffer JE, Ory DS, Jiang X
    Biomed Chromatogr 2018 Jul;32(7):e4235
  • A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I
    Le SQ, Kan SH, Clarke D, Sanghez V, Egeland M, Vondrak KN, Doherty TM, Vera MU, Iacovino M, Cooper JD, Sands MS, Dickson PI
    Mol Ther Methods Clin Dev 2017 Oct 5;8:42-51
  • Widespread Expression of a Membrane-Tethered Version of the Soluble Lysosomal Enzyme Palmitoyl Protein Thioesterase-1
    Shyng C, Macauley SL, Dearborn JT, Sands MS
    JIMD Rep 2017;36:85-92
  • Synergistic Effects of Treating the Spinal Cord and Brain in CLN1 Disease
    Shyng C, Nelvagal HR, Dearborn JT, Tyynela J, Schmidt RE, Sands MS, Cooper JD
    Proc Natl Acad Sci U S A 2017 Jul 18;114(29):E5920-E5929
  • Primary Fibroblasts From CSPalpha Mutation Carriers Recapitulate Hallmarks of the Adult Onset Neuronal Ceroid Lipofuscinosis
    Benitez BA, Sands MS
    Sci Rep 2017 Jul 24;7(1):6332
  • Hematopoietic Stem Cell Transplantation and Lentiviral Vector-Based Gene Therapy for Krabbe's Disease: Present Convictions and Future Prospects
    Hu P, Li Y, Nikolaishvili-Feinberg N, Scesa G, Bi Y, Pan D, Moore D, Bongarzone ER, Sands MS, Miller R, Kafri T
    J Neurosci Res 2016 Nov;94(11):1152-68
  • Behavioral Deficits and Cholinergic Pathway Abnormalities in Male Sanfilippo B Mice
    Kan SH, Le SQ, Bui QD, Benedict B, Cushman J, Sands MS, Dickson PI
    Behav Brain Res 2016 Oct 1;312:265-71
  • Clinical Course of Sly Syndrome (Mucopolysaccharidosis Type VII)
    Montano AM, Lock-Hock N, Steiner RD, Graham BH, Szlago M, Greenstein R, Pineda M, Gonzalez-Meneses A, Coker M, Bartholomew D, Sands MS, Wang R, Giugliani R, Macaya A, Pastores G, Ketko AK, Ezgu F, Tanaka A, Arash L, Beck M, Falk RE, Bhattacharya K, Franco J, White KK, Mitchell GA, Cimbalistiene L, Holtz M, Sly WS
    J Med Genet 2016 Jun;53(6):403-18
  • Histochemical Localization of Palmitoyl Protein Thioesterase-1 Activity
    Dearborn JT, Ramachandran S, Shyng C, Lu JY, Thornton J, Hofmann SL, Sands MS
    Mol Genet Metab 2016 Feb;117(2):210-6
  • Clinically Early-Stage CSPalpha Mutation Carrier Exhibits Remarkable Terminal Stage Neuronal Pathology With Minimal Evidence of Synaptic Loss
    Benitez BA, Cairns NJ, Schmidt RE, Morris JC, Norton JB, Cruchaga C, Sands MS
    Acta Neuropathol Commun 2015 Nov 26;3(1):73
  • Generation of a Stable Packaging Cell Line Producing High-Titer PPT-Deleted Integration-Deficient Lentiviral Vectors
    Hu P, Li Y, Sands MS, McCown T, Kafri T
    Mol Ther Methods Clin Dev 2015 Jul 22;2:15025 eCollection 2015
  • Comprehensive Functional Characterization of Murine Infantile Batten Disease Including Parkinson-Like Behavior and Dopaminergic Markers
    Dearborn JT, Harmon SK, Fowler SC, O'Malley KL, Taylor GT, Sands MS, Wozniak DF
    Sci Rep 2015 Aug 4;5:12752
  • Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy
    Hawkins-Salsbury JA, Shea L, Jiang X, Hunter DA, Guzman AM, Reddy AS, Qin EY, Li Y, Gray SJ, Ory DS, Sands MS
    J Neurosci 2015 Apr 22;35(16):6495-505
  • An Anti-Neuroinflammatory That Targets Dysregulated Glia Enhances the Efficacy of CNS-Directed Gene Therapy in Murine Infantile Neuronal Ceroid Lipofuscinosis
    Macauley SL, Wong AM, Shyng C, Augner DP, Dearborn JT, Pearse Y, Roberts MS, Fowler SC, Cooper JD, Watterson DM, Sands MS
    J Neurosci 2014 Sep 24;34(39):13077-82
  • Adeno-Associated Virus Serotypes 9 and Rh10 Mediate Strong Neuronal Transduction of the Dog Brain
    Swain GP, Prociuk M, Bagel JH, O'Donnell P, Berger K, Drobatz K, Gurda BL, Haskins ME, Sands MS, Vite CH
    Gene Ther 2014 Jan;21(1):28-36
  • Psychosine, the Cytotoxic Sphingolipid That Accumulates in Globoid Cell Leukodystrophy, Alters Membrane Architecture
    Hawkins-Salsbury JA, Parameswar AR, Jiang X, Schlesinger PH, Bongarzone E, Ory DS, Demchenko AV, Sands MS
    J Lipid Res 2013 Dec;54(12):3303-11
  • Disease Correction by Combined Neonatal Intracranial AAV and Systemic Lentiviral Gene Therapy in Sanfilippo Syndrome Type B Mice
    Heldermon CD, Qin EY, Ohlemiller KK, Herzog ED, Brown JR, Vogler C, Hou W, Orrock JL, Crawford BE, Sands MS
    Gene Ther 2013 Sep;20(9):913-21
  • The Sphingolipid Psychosine Inhibits Fast Axonal Transport in Krabbe Disease by Activation of GSK3beta and Deregulation of Molecular Motors
    Cantuti Castelvetri L, Givogri MI, Hebert A, Smith B, Song Y, Kaminska A, Lopez-Rosas A, Morfini G, Pigino G, Sands M, Brady ST, Bongarzone ER
    J Neurosci 2013 Jun 12;33(24):10048-56
  • Central Nervous System Pathology Progresses Independently of KC and CXCR2 in Globoid-Cell Leukodystrophy
    Reddy AS, Patel JR, Vogler C, Klein RS, Sands MS
    PLoS One 2013 Jun 3;8(6):e64647
  • Oxidative Stress as a Therapeutic Target in Globoid Cell Leukodystrophy
    Hawkins-Salsbury JA, Qin EY, Reddy AS, Vogler CA, Sands MS
    Exp Neurol 2012 Oct;237(2):444-52
  • Bone Marrow Transplantation Increases Efficacy of Central Nervous System-Directed Enzyme Replacement Therapy in the Murine Model of Globoid Cell Leukodystrophy
    Qin EY, Hawkins-Salsbury JA, Jiang X, Reddy AS, Farber NB, Ory DS, Sands MS
    Mol Genet Metab 2012 Sep;107(1-2):186-96
  • Background Mutations in Parental Cells Account for Most of the Genetic Heterogeneity of Induced Pluripotent Stem Cells
    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, Wendl MC, Sands MS, Mardis ER, Wilson RK, Townes TM, Ley TJ
    Cell Stem Cell 2012 May 4;10(5):570-82
  • Synergistic Effects of Central Nervous System-Directed Gene Therapy and Bone Marrow Transplantation in the Murine Model of Infantile Neuronal Ceroid Lipofuscinosis
    Macauley SL, Roberts MS, Wong AM, McSloy F, Reddy AS, Cooper JD, Sands MS
    Ann Neurol 2012 Jun;71(6):797-804
  • Combination Small Molecule PPT1 Mimetic and CNS-Directed Gene Therapy as a Treatment for Infantile Neuronal Ceroid Lipofuscinosis
    Roberts MS, Macauley SL, Wong AM, Yilmas D, Hohm S, Cooper JD, Sands MS
    J Inherit Metab Dis 2012 Sep;35(5):847-57
  • Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy
    Reddy AS, Wozniak DF, Farber NB, Dearborn JT, Fowler SC, Sands MS
    J Clin Med 2012 Jan 19;1(1):1-14
  • Exome-Sequencing Confirms DNAJC5 Mutations as Cause of Adult Neuronal Ceroid-Lipofuscinosis
    Benitez BA, Alvarado D, Cai Y, Mayo K, Chakraverty S, Norton J, Morris JC, Sands MS, Goate A, Cruchaga C
    PLoS One 2011;6(11):e26741
  • The Role of Attenuated Astrocyte Activation in Infantile Neuronal Ceroid Lipofuscinosis
    Macauley SL, Pekny M, Sands MS
    J Neurosci 2011 Oct 26;31(43):15575-85
  • Lentiviral-Mediated Gene Transfer to the Sheep Brain: Implications for Gene Therapy in Batten Disease
    Linterman KS, Palmer DN, Kay GW, Barry LA, Mitchell NL, McFarlane RG, Black MA, Sands MS, Hughes SM
    Hum Gene Ther 2011 Aug;22(8):1011-20
  • 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
    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
    J Neurosci 2011 Jul 6;31(27):9945-9957
  • Oligodendrocyte Degeneration and Recovery After Focal Cerebral Ischemia
    McIver SR, Muccigrosso M, Gonzales ER, Lee JM, Roberts MS, Sands MS, Goldberg MP
    Neuroscience 2010 Sep 1;169(3):1364-1375
  • Concise Synthesis of the Unnatural Sphingosine and Psychosine Enantiomer
    Parameswar AR, Hawkins JA, Mydock LK, Sands MS, Demchenko AV
    European J Org Chem 2010 Jun;2010(17):3269-74
  • Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-Mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB
    Heldermon CD, Ohlemiller KK, Herzog ED, Vogler C, Qin E, Wozniak DF, Tan Y, Orrock JL, Sands MS
    Mol Ther 2010 May;18(5):873-80
  • Diffusion Tensor Imaging Detects Axonal Injury and Demyelination in the Spinal Cord and Cranial Nerves of a Murine Model of Globoid Cell Leukodystrophy
    Hofling AA, Kim JH, Fantz CR, Sands MS, Song SK
    NMR Biomed 2009 Dec;22(10):1100-1106
  • Metabolic Adaptations to Interrupted Glycosaminoglycan Recycling
    Woloszynek JC, Kovacs A, Ohlemiller KK, Roberts M, Sands MS
    J Biol Chem 2009 Oct 23;284(43):29684-91
  • Molecular Correlates of Axonal and Synaptic Pathology in Mouse Models of Batten Disease
    Kielar C, Wishart TM, Palmer A, Dihanich S, Wong AM, Macauley SL, Chan CH, Sands MS, Pearce DA, Cooper JD, Gillingwater TH
    Hum Mol Genet 2009 Nov 1;18(21):4066-80
  • Cerebellar Pathology and Motor Deficits in the Palmitoyl Protein Thioesterase 1-Deficient Mouse
    Macauley SL, Wozniak DF, Kielar C, Tan Y, Cooper JD, Sands MS
    Exp Neurol 2009 May;217(1):124-35
  • Central Amygdala Glucocorticoid Receptor Action Promotes Fear-Associated CRH Activation and Conditioning
    Kolber BJ, Roberts MS, Howell MP, Wozniak DF, Sands MS, Muglia LJ
    Proc Natl Acad Sci U S A 2008 Aug 19;105(33):12004-9
  • Lentiviral-Transduced Human Mesenchymal Stem Cells Persistently Express Therapeutic Levels of Enzyme in a Xenotransplantation Model of Human Disease
    Meyerrose TE, Roberts M, Ohlemiller KK, Vogler CA, Wirthlin L, Nolta JA, Sands MS
    Stem Cells 2008 Jul;26(7):1713-22
  • Niemann-Pick C1 Protects Against Atherosclerosis in Mice Via Regulation of Macrophage Intracellular Cholesterol Trafficking
    Zhang JR, Coleman T, Langmade SJ, Scherrer DE, Lane L, Lanier MH, Feng C, Sands MS, Schaffer JE, Semenkovich CF, Ory DS
    J Clin Invest 2008 Jun;118(6):2281-90
  • A Murine Model of Infantile Neuronal Ceroid Lipofuscinosis-Ultrastructural Evaluation of Storage in the Central Nervous System and Viscera
    Galvin N, Vogler C, Levy B, Kovacs A, Griffey M, Sands MS
    Pediatr Dev Pathol 2008 May-Jun;11(3):185-92
  • Widespread Nonhematopoietic Tissue Distribution by Transplanted Human Progenitor Cells With High Aldehyde Dehydrogenase Activity
    Hess DA, Craft TP, Wirthlin L, Hohm S, Zhou P, Eades WC, Creer MH, Sands MS, Nolta JA
    Stem Cells 2008 Mar;26(3):611-20
  • Lysosomal Dysfunction Results in Altered Energy Balance
    Woloszynek JC, Coleman T, Semenkovich CF, Sands MS
    J Biol Chem 2007 Dec 7;282(49):35765-71
  • Development of Sensory, Motor and Behavioral Deficits in the Murine Model of Sanfilippo Syndrome Type B
    Heldermon CD, Hennig AK, Ohlemiller KK, Ogilvie JM, Herzog ED, Breidenbach A, Vogler C, Wozniak DF, Sands MS
    PLoS ONE 2007 Aug 22;2(1):e772
  • AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma
    Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS
    Science 2007 Jul 27;317(5837):477
  • Clinical Response to Persistent, Low-Level Beta-Glucuronidase Expression in the Murine Model of Mucopolysaccharidosis Type VII
    Donsante A, Levy B, Vogler C, Sands MS
    J Inherit Metab Dis 2007 Apr;30(2):227-38
  • Successive Neuron Loss in the Thalamus and Cortex in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis
    Kielar C, Maddox L, Bible E, Pontikis CC, Macauley SL, Griffey MA, Wong M, Sands MS, Cooper JD
    Neurobiol Dis 2007 Jan;25(1):150-62
  • In Vivo Distribution of Human Adipose-Derived Mesenchymal Stem Cells in Novel Xenotransplantation Models
    Meyerrose TE, De Ugarte DA, Hofling AA, Herrbrich PE, Cordonnier TD, Shultz LD, Eagon JC, Wirthlin L, Sands MS, Hedrick MA, Nolta JA
    Stem Cells 2007 Jan;25(1):220-7
  • Central Nervous System-Directed AAV2/5-Mediated Gene Therapy Synergizes With Bone Marrow Transplantation in the Murine Model of Globoid-Cell Leukodystrophy
    Lin D, Donsante A, Macauley S, Levy B, Vogler C, Sands MS
    Mol Ther 2007 Jan;15(1):44-52
  • Gene Targeting in Vivo by Adeno-Associated Virus Vectors
    Miller DG, Wang PR, Petek LM, Hirata RK, Sands MS, Russell DW
    Nat Biotechnol 2006 Aug;24(8):1022-6
  • The Origin and in Vivo Significance of Murine and Human Culture-Expanded Endothelial Progenitor Cells
    Sharpe EE 3rd, Teleron AA, Li B, Price J, Sands MS, Alford K, Young PP
    Am J Pathol 2006 May;168(5):1710-21
  • CNS-Directed AAV2-Mediated Gene Therapy Ameliorates Functional Deficits in a Murine Model of Infantile Neuronal Ceroid Lipofuscinosis
    Griffey MA, Wozniak D, Wong M, Bible E, Johnson K, Rothman SM, Wentz AE, Cooper JD, Sands MS
    Mol Ther 2006 Mar;13(3):538-47
  • Critical Role of Beta3 Integrin in Experimental Postmenopausal Osteoporosis
    Zhao H, Kitaura H, Sands MS, Ross FP, Teitelbaum SL, Novack DV
    J Bone Miner Res 2005 Dec;20(12):2116-23
  • Lentiviral Transduction of Murine Oligodendrocytes in Vivo
    McIver SR, Lee CS, Lee JM, Green SH, Sands MS, Snider BJ, Goldberg MP
    J Neurosci Res 2005 Nov 1;82(3):397-403
  • AAV2-Mediated Ocular Gene Therapy for Infantile Neuronal Ceroid Lipofuscinosis
    Griffey M, Macauley SL, Ogilvie JM, Sands MS
    Mol Ther 2005 Sep;12(3):413-21
  • AAV2/5 Vector Expressing Galactocerebrosidase Ameliorates CNS Disease in the Murine Model of Globoid-Cell Leukodystrophy More Efficiently Than AAV2
    Lin D, Fantz CR, Levy B, Rafi MA, Vogler C, Wenger DA, Sands MS
    Mol Ther 2005 Sep;12(3):422-30
  • Marrow Stromal Cells and Osteoclast Precursors Differentially Contribute to TNF-Alpha-Induced Osteoclastogenesis in Vivo
    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
    J Immunol 2004 Oct 15;173(8):4838-46
  • Collection of a Mobilized Peripheral Blood Apheresis Product From a Patient With Mucopolysaccharidosis Type VII and Subsequent CD34+ Cell Isolation
    Hofling AA, Sands MS, Lublin DM, Bauer G, Devine S
    J Clin Apher 2004;19(3):151-3
  • In Vivo Transduction of Hematopoietic Stem Cells After Neonatal Intravenous Injection of an Amphotropic Retroviral Vector in Mice
    Xu L, O'Malley T, Sands MS, Wang B, Meyerrose T, Haskins ME, Ponder KP
    Mol Ther 2004 Jul;10(1):37-44
  • AAV-Mediated Intravitreal Gene Therapy Reduces Lysosomal Storage in the Retinal Pigmented Epithelium and Improves Retinal Function in Adult MPS VII Mice
    Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS
    Mol Ther 2004 Jul;10(1):106-16
  • Human CD34+ Hematopoietic Progenitor Cell-Directed Lentiviral-Mediated Gene Therapy in a Xenotransplantation Model of Lysosomal Storage Disease
    Hofling AA, Devine S, Vogler C, Sands MS
    Mol Ther 2004 Jun;9(6):856-65
  • Adeno-Associated Virus 2-Mediated Gene Therapy Decreases Autofluorescent Storage Material and Increases Brain Mass in a Murine Model of Infantile Neuronal Ceroid Lipofuscinosis
    Griffey M, Bible E, Vogler C, Levy B, Gupta P, Cooper J, Sands MS
    Neurobiol Dis 2004 Jul;16(2):360-9
  • 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
    Young PP, Fantz CR, Sands MS
    Exp Neurol 2004 Jul;188(1):104-14
  • Neonatal Retroviral Vector-Mediated Hepatic Gene Therapy Reduces Bone, Joint, and Cartilage Disease in Mucopolysaccharidosis VII Mice and Dogs
    Mango RL, Xu L, Sands MS, Vogler C, Seiler G, Schwarz T, Haskins ME, Ponder KP
    Mol Genet Metab 2004 May;82(1):4-19
  • Numerous Transcriptional Alterations in Liver Persist After Short-Term Enzyme-Replacement Therapy in a Murine Model of Mucopolysaccharidosis Type VII
    Woloszynek JC, Roberts M, Coleman T, Vogler C, Sly W, Semenkovich CF, Sands MS
    Biochem J 2004 Apr 15;379(Pt 2):461-9
  • Neonatal or Hepatocyte Growth Factor-Potentiated Adult Gene Therapy With a Retroviral Vector Results in Therapeutic Levels of Canine Factor IX for Hemophilia B
    Xu L, Gao C, Sands MS, Cai SR, Nichols TC, Bellinger DA, Raymer RA, McCorquodale S, Ponder KP
    Blood 2003 May 15;101(10):3924-32
  • Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice
    Hennig AK, Levy B, Ogilvie JM, Vogler CA, Galvin N, Bassnett S, Sands MS
    J Neurosci 2003 Apr 15;23(8):3302-7
  • 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
    Hofling AA, Vogler C, Creer MH, Sands MS
    Blood 2003 Mar 1;101(5):2054-63
  • Biodistribution and Efficacy of Donor T Lymphocytes in a Murine Model of Lysosomal Storage Disease
    Young PP, Vogler C, Hofling AA, Sands MS
    Mol Ther 2003 Jan;7(1):52-61
  • Evaluation of Pathological Manifestations of Disease in Mucopolysaccharidosis VII Mice After Neonatal Hepatic Gene Therapy
    Xu L, Mango RL, Sands MS, Haskins ME, Ellinwood NM, Ponder KP
    Mol Ther 2002 Dec;6(6):745-58
  • VEGF Increases Engraftment of Bone Marrow-Derived Endothelial Progenitor Cells (EPCs) Into Vasculature of Newborn Murine Recipients
    Young PP, Hofling AA, Sands MS
    Proc Natl Acad Sci U S A 2002 Sep 3;99(18):11951-6
  • Inner Ear Pathology in the Mucopolysaccharidosis VII Mouse
    Ohlemiller KK, Hennig AK, Lett JM, Heidbreder AF, Sands MS
    Hear Res 2002 Jul;169(1-2):69-84
  • Abnormal Osteoclast Morphology and Bone Remodeling in a Murine Model of a Lysosomal Storage Disease
    Monroy MA, Ross FP, Teitelbaum SL, Sands MS
    Bone 2002 Feb;30(2):352-9
  • Biodistribution, Kinetics, and Efficacy of Highly Phosphorylated and Non-Phosphorylated Beta-Glucuronidase in the Murine Model of Mucopolysaccharidosis VII
    Sands MS, Vogler CA, Ohlemiller KK, Roberts MS, Grubb JH, Levy B, Sly WS
    J Biol Chem 2001 Nov 16;276(46):43160-5
  • Murine Mucopolysaccharidosis VII:  Impact of Therapies on the Phenotype, Clinical Course and Pathology in a Model of Lysosomal Storage Disease
    Vogler CA, Barker JE, Sands MS, Levy B, Galvin B, Sly WS
    Ped Dev Path 2001;4:421
  • Observed Incidence of Tumorigenesis in Long-Term Rodent Studies of RAAV Vectors
    Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS
    Gene Ther 2001 Sep;8(17):1343-6
  • Prevention of Systemic Clinical Disease in MPS VII Mice Following AAV-Mediated Neonatal Gene Transfer
    Daly TM, Ohlemiller KK, Roberts MS, Vogler CA, Sands MS
    Gene Ther 2001 Sep;8(17):1291-8
  • 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
    Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, Sands MS, Parker Ponder K
    Hum Gene Ther 2001 Mar 20;12(5):563-73
  • Intracranial Injection of Recombinant Adeno-Associated Virus Improves Cognitive Function in a Murine Model of Mucopolysaccharidosis Type VII
    Frisella WA, O'Connor LH, Vogler CA, Roberts M, Walkley S, Levy B, Daly TM, Sands MS
    Mol Ther 2001 Mar;3(3):351-8
  • 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
    Vogler C, Levy B, Galvin N, Sands MS, Birkenmeier EH, Sly WS, Barker J
    Pediatr Res 2001 Mar;49(3):342-8
  • Gene Transfer of Low Levels of Beta-Glucuronidase Corrects Hepatic Lysosomal Storage in a Large Animal Model of Mucopolysaccharidosis VII
    Wolfe JH, Sands MS, Harel N, Weil MA, Parente MK, Polesky AC, Reilly JJ, Hasson C, Weimelt S, Haskins ME
    Mol Ther 2000 Dec;2(6):552-61
  • Delivery of a Retroviral Vector Expressing Human Beta-Glucuronidase to the Liver and Spleen Decreases Lysosomal Storage in Mucopolysaccharidosis VII Mice
    Gao C, Sands MS, Haskins ME, Ponder KP
    Mol Ther 2000 Sep;2(3):233-44
  • Treatment of a Lysosomal Storage Disease, Mucopolysaccharidosis VII, With Microencapsulated Recombinant Cells
    Ross CJ, Bastedo L, Maier SA, Sands MS, Chang PL
    Hum Gene Ther 2000 Oct 10;11(15):2117-27
  • Retinal Function Is Improved in a Murine Model of a Lysosomal Storage Disease Following Bone Marrow Transplantation
    Ohlemiller KK, Vogler CA, Roberts M, Galvin N, Sands MS
    Exp Eye Res 2000 Nov;71(5):469-81
  • Abnormal Immune Function in Vivo in a Murine Model of Lysosomal Storage Disease
    Daly TM, Lorenz RG, Sands MS
    Pediatr Res 2000 Jun;47(6):757-62
  • Behavior and Therapeutic Efficacy of Beta-Glucuronidase-Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII
    Freeman BJ, Roberts MS, Vogler CA, Nicholes A, Hofling AA, Sands MS
    Blood 1999 Sep 15;94(6):2142-50
  • Enzyme Replacement in Murine Mucopolysaccharidosis Type VII: Neuronal and Glial Response to Beta-Glucuronidase Requires Early Initiation of Enzyme Replacement Therapy
    Vogler C, Levy B, Galvin NJ, Thorpe C, Sands MS, Barker JE, Baty J, Birkenmeier EH, Sly WS
    Pediatr Res 1999 Jun;45(6):838-44
  • Percutaneous Intravenous Injection in Neonatal Mice
    Sands MS, Barker JE
    Lab Anim Sci 1999 Jun;49(3):328-30
  • Neonatal Gene Transfer Leads to Widespread Correction of Pathology in a Murine Model of Lysosomal Storage Disease
    Daly TM, Vogler C, Levy B, Haskins ME, Sands MS
    Proc Natl Acad Sci U S A 1999 Mar 2;96(5):2296-300
  • 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
    Daly TM, Okuyama T, Vogler C, Haskins ME, Muzyczka N, Sands MS
    Hum Gene Ther 1999 Jan 1;10(1):85-94
  • 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
    Gwynn B, Lueders K, Sands MS, Birkenmeier EH
    Mol Cell Biol 1998 Nov;18(11):6474-81
  • Murine Mucopolysaccharidosis Type VII: The Impact of Therapies on the Clinical Course and Pathology in a Murine Model of Lysosomal Storage Disease
    Vogler C, Sands MS, Galvin N, Levy B, Thorpe C, Barker J, Sly WS
    J Inherit Metab Dis 1998 Aug;21(5):575-86
  • Enzyme Replacement Therapy for Murine Mucopolysaccharidosis Type VII Leads to Improvements in Behavior and Auditory Function
    O'Connor LH, Erway LC, Vogler CA, Sly WS, Nicholes A, Grubb J, Holmberg SW, Levy B, Sands MS
    J Clin Invest 1998 Apr 1;101(7):1394-400
  • Gene Therapy for Murine Mucopolysaccharidosis Type VII
    Sands MS, Wolfe JH, Birkenmeier EH, Barker JE, Vogler C, Sly WS, Okuyama T, Freeman B, Nicholes A, Muzyczka N, Chang PL, Axelrod HR
    Neuromuscul Disord 1997 Jul;7(5):352-60
  • Murine Mucopolysaccharidosis Type VII: Long Term Therapeutic Effects of Enzyme Replacement and Enzyme Replacement Followed by Bone Marrow Transplantation
    Sands MS, Vogler C, Torrey A, Levy B, Gwynn B, Grubb J, Sly WS, Birkenmeier EH
    J Clin Invest 1997 Apr 1;99(7):1596-605
  • Successful Expression of Human Factor IX Following Repeat Administration of Adenoviral Vector in Mice
    Walter J, You Q, Hagstrom JN, Sands M, High KA
    Proc Natl Acad Sci U S A 1996 Apr 2;93(7):3056-61
  • 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
    Vogler C, Sands MS, Levy B, Galvin N, Birkenmeier EH, Sly WS
    Pediatr Res 1996 Jun;39(6):1050-4
  • Syngeneic Bone Marrow Transplantation Reduces the Hearing Loss Associated With Murine Mucopolysaccharidosis Type VII
    Sands MS, Erway LC, Vogler C, Sly WS, Birkenmeier EH
    Blood 1995 Sep 1;86(5):2033-40
  • High Level Expression and Export of Beta-Glucuronidase From Murine Mucopolysaccharidosis VII Cells Corrected by a Double-Copy Retrovirus Vector
    Wolfe JH, Kyle JW, Sands MS, Sly WS, Markowitz DG, Parente MK
    Gene Ther 1995 Jan;2(1):70-8
  • Behavioral Consequences of Bone Marrow Transplantation in the Treatment of Murine Mucopolysaccharidosis Type VII
    Bastedo L, Sands MS, Lambert DT, Pisa MA, Birkenmeier E, Chang PL
    J Clin Invest 1994 Sep;94(3):1180-6
  • Enzyme Replacement Therapy for Murine Mucopolysaccharidosis Type VII
    Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, Birkenmeier EH
    J Clin Invest 1994 Jun;93(6):2324-31
  • Enzyme Replacement With Recombinant Beta-Glucuronidase in the Newborn Mucopolysaccharidosis Type VII Mouse
    Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, Sly WS
    Pediatr Res 1993 Dec;34(6):837-40
  • A Single-Base-Pair Deletion in the Beta-Glucuronidase Gene Accounts for the Phenotype of Murine Mucopolysaccharidosis Type VII
    Sands MS, Birkenmeier EH
    Proc Natl Acad Sci U S A 1993 Jul 15;90(14):6567-71
  • Treatment of Murine Mucopolysaccharidosis Type VII by Syngeneic Bone Marrow Transplantation in Neonates
    Sands MS, Barker JE, Vogler C, Levy B, Gwynn B, Galvin N, Sly WS, Birkenmeier E
    Lab Invest 1993 Jun;68(6):676-86
  • Reversal of Pathology in Murine Mucopolysaccharidosis Type VII by Somatic Cell Gene Transfer
    Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, Vogler CA, Birkenmeier EH
    Nature 1992 Dec 24-31;360(6406):749-53
  • Binding of TFIIIA to Derivatives of 5S RNA Containing Sequence Substitutions or Deletions Defines a Minimal TFIIIA Binding Site
    Bogenhagen DF, Sands MS
    Nucleic Acids Res 1992 Jun 11;20(11):2639-45
  • Two Zinc Finger Proteins From Xenopus Laevis Bind the Same Region of 5S RNA but With Different Nuclease Protection Patterns
    Sands MS, Bogenhagen DF
    Nucleic Acids Res 1991 Apr 25;19(8):1797-803
  • The Carboxyterminal Zinc Fingers of TFIIIA Interact With the Tip of Helix V of 5S RNA in the 7S Ribonucleoprotein Particle
    Sands MS, Bogenhagen DF
    Nucleic Acids Res 1991 Apr 25;19(8):1791-6
  • Local Blood-Brain Barrier Penetration Following Systemic Contrast Medium Administration. A Case Report and an Experimental Study
    Utz R, Ekholm SE, Isaac L, Sands M, Fonte D
    Acta Radiol 1988 Mar-Apr;29(2):237-42
  • TFIIIA Binds to Different Domains of 5S RNA and the Xenopus Borealis 5S RNA Gene
    Sands MS, Bogenhagen DF
    Mol Cell Biol 1987 Nov;7(11):3985-93
  • Computed Tomographic Enhancement of Liver and Spleen in the Dog With Iodipamide Ethyl Ester Particulate Suspensions
    Sands MS, Violante MR, Gadeholt G
    Invest Radiol 1987 May;22(5):408-16
  • Postoperative Radiographic Appearance of Intracranial Hemostatic Gelatin Sponge
    Maurer PK, Ekholm SE, McDonald JV, Sands M, Kido DK
    Surg Neurol 1986 Dec;26(6):562-6
  • Lumbar Myelography With Iohexol and Metrizamide. A Double-Blind Clinical Trial
    Hindmarsh T, Ekholm SE, Kido DK, Sahler L, Sands M
    Acta Radiol [Diagn] (Stockh) 1984;25(5):365-8
  • Iohexol Lumbar Myelography: Clinical Study
    Holder JC, Binet EF, Kido DK, Belanger G, Sands MS
    AJNR Am J Neuroradiol 1984 Jul-Aug;5(4):399-402
  • Radioisotope Imaging for Detection of an Ectopic Spleen in a Macaque
    Allen J, Sands M, Baggs R
    J Am Vet Med Assoc 1982 Dec 1;181(11):1428-9
  • Cost-Effective Use of Stannous Pyrophosphate [Letter]
    Sands M, Owunwanne A
    J Nucl Med 1981 Oct;22(10):932

Invited Reviews and Book Chapters

  • Krabbe Disease: New Hope for an Old Disease
    Bradbury AM, Bongarzone ER, Sands MS
    Neurosci Lett 2021 May 1;752:135841
  • Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights From Animal Models
    Chandler RJ, Sands MS, Venditti CP
    Hum Gene Ther 2017 Apr;28(4):314-322
  • Treatment for Krabbe's Disease: Finding the Combination
    Mikulka CR, Sands MS
    J Neurosci Res 2016 Nov;94(11):1126-37
  • Overcoming the Next Barriers to Successful Therapy
    Cohen IJ, Baris H, Mistry PK, Sands MS
    Pediatr Endocrinol Rev 2016 Jun;13 Suppl 1:629
  • Insights Into the Pathogenesis and Treatment of Krabbe Disease
    Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS
    Pediatr Endocrinol Rev 2016 Jun;13 Suppl 1:689-96
  • Mucopolysaccharidosis Type VII: A Powerful Experimental System and Therapeutic Challenge
    Sands MS
    Pediatr Endocrinol Rev 2014 Sep;12 Suppl 1:159-65
  • Treatment for LSDs: Real Options for Several Diseases. Forward
    Cohen IJ, Baris HN, Mistry PK, Sands MS
    Pediatr Endocrinol Rev 2014 Sep;12 Suppl 1:71
  • A Hitchhiker's Guide to the Blood-Brain Barrier: In Trans Delivery of a Therapeutic Enzyme
    Sands MS
    Mol Ther 2014 Mar;22(3):483-4
  • Pathogenesis and Therapies for Infantile Neuronal Ceroid Lipofuscinosis (Infantile CLN1 Disease)
    Hawkins-Salsbury JA, Cooper JD, Sands MS
    Biochim Biophys Acta 2013 Nov;1832(11):1906-9
  • Considerations for the Treatment of Infantile Neuronal Ceroid Lipofuscinosis (Infantile Batten Disease)
    Sands MS
    J Child Neurol 2013 Sep;28(9):1151-8
  • Farber Disease: Understanding a Fatal Childhood Disorder and Dissecting Ceramide Biology
    Sands MS
    EMBO Mol Med 2013 Jun;5(6):799-801
  • AAV-Mediated Liver-Directed Gene Therapy
    Sands MS
    In: Methods in Molecular Biology (RO Snyder, ed), Humana Press Inc., Totowa, NJ 807:141; 2011
  • Combination Therapies for Lysosomal Storage Disease: Is the Whole Greater Than the Sum of Its Parts?
    Hawkins-Salsbury JA, Reddy AS, Sands MS
    Hum Mol Genet 2011 Apr 15;20(R1):R54-60
  • Promising CNS-Directed Enzyme Replacement Therapy for Lysosomal Storage Diseases
    Macauley SL, Sands MS
    Exp Neurol 2009 Jul;218(1):5-8
  • CNS-Directed Gene Therapy for Lysosomal Storage Diseases
    Sands MS, Haskins ME
    Acta Paediatr Suppl 2008 Apr;97(457):22-7
  • Gene Therapy for Lysosomal Storage Diseases
    Sands MS, Davidson BL
    Mol Ther 2006 May;13(5):839-49
  • Pre-Clinical Basis for Gene Therapy Studies
    Sands MS, Nolta JA, Bauer G, Ferkol T
    In: Translational and Experimental Medicine (Schuster D, Powers W, eds), Lippincott Publishing; 2005
  • Gene Therapy for Lysosomal Storage Diseases Affecting the Brain
    Sands MS
    In: Lysosomal Storage Diseases Affecting the Central Nervous System (Platt F, Walkley SU, eds), Oxford University Press, Oxford, UK; 2004
  • Safety of Adeno-Associated Virus Gene Therapy Vectors: A Current Evaluation
    Monahan PE, Jooss K, Sands MS
    Expert Opin Drug Saf 2002 May;1(1):79-91
  • Preventing Sensory Loss in a Mouse Model of Lysosomal Storage Disease
    Ohlemiller KK, Vogler CA, Daly TM, Sands MS
    In: Handbook of mouse auditory research: from behavior to molecular biology (JF Willot, ed), CRC Press, New York; 2001
  • Gene Therapy for Lysosomal Storage Diseases
    Daly TM, Sands MS
    Expert Opin Investig Drugs 1998 Oct;7(10):1673-82
  • Murine Mucopolysaccharidosis Type VII: A Model System for Somatic Gene Therapy of the Central Nervous System
    Wolfe JH, Sands MS
    In: Gene Transfer into Neurones, Towards Gene Therapy of Neurological Disorders (P Lowenstein, L Enquist, eds), J Wiley & Sons Ltd, Essex, England, p 263; 1996
  • Domains of the 5S RNA Gene Control Region
    Bogenhagen DF, Sands MS, McConkey GA
    16th Annual Steenbock Symposium (Reznikoff W et al, eds), Madison, Wisconsin, p 219; 1986
  • Maximizing Hepatic Contrast Enhancement With a Particulate Contrast Agent in Computed Tomography
    Violante MR, Fischer HW, Mare KO, Boxmann HB, Owunwanne A, Zack AG, Harmon P, Case K, Sands M
    Contrast Media in Computed Tomography, International Workshop, Berlin, January, 1981