Mark S. Sands, Ph.D.

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 2008 Apr 24; [Epub ahead of print]

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.
J Clin Invest 2008 Jun;118(6):2281-90

Sands MS, Haskins ME
CNS-directed gene therapy for lysosomal storage diseases.
Acta Paediatr Suppl 2008 Apr;97(457):22-7

Galvin, N, Vogler CA, Levy B, Griffey M, Kovacs A, Sands MS
A murine model of infantile neuronal ceroid lipofuscinosis - Ultrastructural evaluation of storage in the central nervous system and viscera.
Ped Dev Path, in press

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 2008 Mar;26(3):611-20

Woloszynek JC, Coleman T, Semenkovich CF, Sands MS
Lysosomal dysfunction results in altered energy balance.
J Biol Chem 2007 Dec 7;282(49):35765-71

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 2007 Aug 22;2(1):e772

Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS
AAV vector integration sites in mouse hepatocellular carcinoma.
Science 2007 Jul 27;317(5837):477

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.
J Inherit Metab Dis 2007 Apr;30(2):227-38

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.
Neurobiol Dis 2007 Jan;25(1):150-62

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 2007 Jan;25(1):220-7

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.
Mol Ther 2007 Jan;15(1):44-52

Miller DG, Wang PR, Petek LM, Hirata RK, Sands MS, Russell DW
Gene targeting in vivo by adeno-associated virus vectors.
Nat Biotechnol 2006 Aug;24(8):1022-6

Sharpe EE 3rd, 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.
Am J Pathol 2006 May;168(5):1710-21

Sands MS, Davidson BL
Gene therapy for lysosomal storage diseases.
Mol Ther 2006 May;13(5):839-49

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.
Mol Ther 2006 Mar;13(3):538-47

Zhao H, Kitaura H, Sands MS, Ross FP, Teitelbaum SL, Novack DV
Critical role of beta3 integrin in experimental postmenopausal osteoporosis.
J Bone Miner Res 2005 Dec;20(12):2116-23

McIver SR, Lee CS, Lee JM, Green SH, Sands MS, Snider BJ, Goldberg MP
Lentiviral transduction of murine oligodendrocytes in vivo.
J Neurosci Res 2005 Nov 1;82(3):397-403

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.
Mol Ther 2005 Sep;12(3):422-30

Griffey M, Macauley SL, Ogilvie JM, Sands MS
AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.
Mol Ther 2005 Sep;12(3):413-21

Sands MS, Nolta JA, Bauer G, Ferkol T
Pre-clinical basis for gene therapy studies.
In: Translational and Experimental Medicine, D Schuster and W Powers, eds., Lippincott Publishing, 2005

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.
J Immunol 2004 Oct 15;173(8):4838-46

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.
J Clin Apher 2004;19(3):151-3

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.
Mol Ther 2004 Jul;10(1):37-44

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.
Mol Ther 2004 Jul;10(1):106-16

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.
Mol Ther 2004 Jun;9(6):856-65

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.
Neurobiol Dis 2004 Jul;16(2):360-9

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.
Exp Neurol 2004 Jul;188(1):104-14

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.
Mol Genet Metab 2004 May;82(1):4-19

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.
Biochem J 2004 Apr 15;379(Pt 2):461-9

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 2003 May 15;101(10):3924-32

Sands MS
Gene therapy for lysosomal storage diseases affecting the brain.
In: Lysosomal Storage Diseases Affecting the Central Nervous System, F. Platt, S.U. Walkley, eds., Oxford University Press, Oxford, UK, 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.
J Neurosci 2003 Apr 15;23(8):3302-7

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 2003 Mar 1;101(5):2054-63

Young PP, Vogler C, Hofling AA, Sands MS
Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease.
Mol Ther 2003 Jan;7(1):52-61

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.
Mol Ther 2002 Dec;6(6):745-58

Young PP, Hofling AA, Sands MS
VEGF increases engraftment of bone marrow-derived endothelial progenitor cells (EPCs) into vasculature of newborn murine recipients.
Proc Natl Acad Sci U S A 2002 Sep 3;99(18):11951-6

Monahan PE, Jooss K, Sands MS
Safety of adeno-associated virus gene therapy vectors: a current evaluation.
Expert Opin Drug Saf 2002 May;1(1):79-91

Ohlemiller KK, Hennig AK, Lett JM, Heidbreder AF, Sands MS
Inner ear pathology in the mucopolysaccharidosis VII mouse.
Hear Res 2002 Jul;169(1-2):69-84

Monroy MA, Ross FP, Teitelbaum SL, Sands MS
Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease.
Bone 2002 Feb;30(2):352-9

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.
J Biol Chem 2001 Nov 16;276(46):43160-5

Vogler CA, Barker JE, Sands MS, Levy B, Galvin B, Sly WS
Murine mucopolysaccharidosis VII:  Impact of therapies on the phenotype, clinical course and pathology in a model of lysosomal storage disease.
Ped Dev Path 2001;4:421

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 Ther 2001 Sep;8(17):1343-6

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 Ther 2001 Sep;8(17):1291-8

Ohlemiller KK, Vogler CA, Daly TM, Sands MS
Preventing sensory loss in a mouse model of lysosomal storage disease.
In:  Handbook of mouse auditory research:  From behavior to molecular biology.  JF Willot. Ed., CRC Press, New York, 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.
Hum Gene Ther 2001 Mar 20;12(5):563-73

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.
Mol Ther 2001 Mar;3(3):351-8

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.
Pediatr Res 2001 Mar;49(3):342-8

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.
Mol Ther 2000 Dec;2(6):552-61

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.
Mol Ther 2000 Sep;2(3):233-44

Ross CJ, Bastedo L, Maier SA, Sands MS, Chang PL
Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells.
Hum Gene Ther 2000 Oct 10;11(15):2117-27

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.
Exp Eye Res 2000 Nov;71(5):469-81

Daly TM, Lorenz RG, Sands MS
Abnormal immune function in vivo in a murine model of lysosomal storage disease.
Pediatr Res 2000 Jun;47(6):757-62

Sands MS, Barker JE
Percutaneous intravenous injection in neonatal mice.
Comp Med 2000 Apr;50(2):107

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 1999 Sep 15;94(6):2142-50

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.
Pediatr Res 1999 Jun;45(6):838-44

Sands MS, Barker JE
Percutaneous intravenous injection in neonatal mice.
Lab Anim Sci 1999 Jun;49(3):328-30

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.
Proc Natl Acad Sci U S A 1999 Mar 2;96(5):2296-300

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.
Hum Gene Ther 1999 Jan 1;10(1):85-94

Daly TM, Sands MS
Gene therapy for lysosomal storage diseases.
Expert Opin Investig Drugs 1998 Oct;7(10):1673-82

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.
Mol Cell Biol 1998 Nov;18(11):6474-81

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.
J Inherit Metab Dis 1998 Aug;21(5):575-86

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.
J Clin Invest 1998 Apr 1;101(7):1394-400

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.
Neuromuscul Disord 1997 Jul;7(5):352-60

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.
J Clin Invest 1997 Apr 1;99(7):1596-605

Walter J, You Q, Hagstrom JN, Sands M, High KA
Successful expression of human factor IX following repeat administration of adenoviral vector in mice.
Proc Natl Acad Sci U S A 1996 Apr 2;93(7):3056-61

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.
Pediatr Res 1996 Jun;39(6):1050-4

Wolfe JH, Sands M
Murine mucopolysaccharidosis type VII: a model system for somatic gene therapy of the central nervious system.
In: Gene Transfer into Neurones, Towards Gene Therapy of Neurological Disorders.  Lowenstein P and Enquist L, eds., J. Wiley & Sons Ltd., Essex, England, 1996, p. 263

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 1995 Sep 1;86(5):2033-40

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 Ther 1995 Jan;2(1):70-8

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.
J Clin Invest 1994 Sep;94(3):1180-6

Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, Birkenmeier EH
Enzyme replacement therapy for murine mucopolysaccharidosis type VII.
J Clin Invest 1994 Jun;93(6):2324-31

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.
Pediatr Res 1993 Dec;34(6):837-40

Sands MS, Birkenmeier EH
A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII.
Proc Natl Acad Sci U S A 1993 Jul 15;90(14):6567-71

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.
Lab Invest 1993 Jun;68(6):676-86

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 1992 Dec 24-31;360(6406):749-53

Bogenhagen DF, Sands MS
Binding of TFIIIA to derivatives of 5S RNA containing sequence substitutions or deletions defines a minimal TFIIIA binding site.
Nucleic Acids Res 1992 Jun 11;20(11):2639-45

Sands MS, Bogenhagen DF
Two zinc finger proteins from Xenopus laevis bind the same region of 5S RNA but with different nuclease protection patterns.
Nucleic Acids Res 1991 Apr 25;19(8):1797-803

Sands MS, Bogenhagen DF
The carboxyterminal zinc fingers of TFIIIA interact with the tip of helix V of 5S RNA in the 7S ribonucleoprotein particle.
Nucleic Acids Res 1991 Apr 25;19(8):1791-6

Utz R, Ekholm SE, Isaac L, Sands M, Fonte D
Local blood-brain barrier penetration following systemic contrast medium administration. A case report and an experimental study.
Acta Radiol 1988 Mar-Apr;29(2):237-42

Sands MS, Bogenhagen DF
TFIIIA binds to different domains of 5S RNA and the Xenopus borealis 5S RNA gene.
Mol Cell Biol 1987 Nov;7(11):3985-93

Sands MS, Violante MR, Gadeholt G
Computed tomographic enhancement of liver and spleen in the dog with iodipamide ethyl ester particulate suspensions.
Invest Radiol 1987 May;22(5):408-16

Bogenhagen DF, Sands M, McConkey GA
Domains of the 5S RNA gene control region.
16th Annual Steenbock Symposium, Reznikoff WS et al., eds., Madison, WI, 1986, p. 219

Maurer PK, Ekholm SE, McDonald JV, Sands M, Kido DK
Postoperative radiographic appearance of intracranial hemostatic gelatin sponge.
Surg Neurol 1986 Dec;26(6):562-6

Hindmarsh T, Ekholm SE, Kido DK, Sahler L, Sands M
Lumbar myelography with iohexol and metrizamide. A double-blind clinical trial.
Acta Radiol [Diagn] (Stockh) 1984;25(5):365-8

Holder JC, Binet EF, Kido DK, Belanger G, Sands MS
Iohexol lumbar myelography: clinical study.
AJNR Am J Neuroradiol 1984 Jul-Aug;5(4):399-402

Allen J, Sands M, Baggs R
Radioisotope imaging for detection of an ectopic spleen in a macaque.
J Am Vet Med Assoc 1982 Dec 1;181(11):1428-9

Sands M, Owunwanne A
Cost-effective use of stannous pyrophosphate [letter]
J Nucl Med 1981 Oct;22(10):932

Violante MR, Fischer HW, Mare KO, Bosmann, HB, Owunwanne A, Zack AG, Harmon P, Case K, Sands M
Maximizing hepatic contrast enhancement with a particular contrast agent in computed tomography.
Contrast Media in Computed Tomography, International Workshop, Berlin, 1981