PubRank

Mark S Sands

Author PubWeight™ 64.36‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 AAV vector integration sites in mouse hepatocellular carcinoma. Science 2007 3.73
2 Background mutations in parental cells account for most of the genetic heterogeneity of induced pluripotent stem cells. Cell Stem Cell 2012 2.11
3 Gene targeting in vivo by adeno-associated virus vectors. Nat Biotechnol 2006 2.03
4 Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis. Neurobiol Dis 2006 1.69
5 CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis. Mol Ther 2005 1.66
6 In vivo distribution of human adipose-derived mesenchymal stem cells in novel xenotransplantation models. Stem Cells 2006 1.56
7 Exome-sequencing confirms DNAJC5 mutations as cause of adult neuronal ceroid-lipofuscinosis. PLoS One 2011 1.51
8 Niemann-Pick C1 protects against atherosclerosis in mice via regulation of macrophage intracellular cholesterol trafficking. J Clin Invest 2008 1.51
9 Marrow stromal cells and osteoclast precursors differentially contribute to TNF-alpha-induced osteoclastogenesis in vivo. J Immunol 2004 1.50
10 Central amygdala glucocorticoid receptor action promotes fear-associated CRH activation and conditioning. Proc Natl Acad Sci U S A 2008 1.41
11 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 1.40
12 Widespread nonhematopoietic tissue distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity. Stem Cells 2007 1.30
13 Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease. Hum Mol Genet 2009 1.26
14 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 1.25
15 Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B. PLoS One 2007 1.23
16 AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2. Mol Ther 2005 1.20
17 Critical role of beta3 integrin in experimental postmenopausal osteoporosis. J Bone Miner Res 2005 1.17
18 Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB. Mol Ther 2010 1.16
19 Lysosomal dysfunction results in altered energy balance. J Biol Chem 2007 1.15
20 AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis. Mol Ther 2005 1.15
21 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 1.13
22 Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouse. Exp Neurol 2009 1.13
23 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. J Neurosci 2011 1.12
24 The role of attenuated astrocyte activation in infantile neuronal ceroid lipofuscinosis. J Neurosci 2011 1.12
25 Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease. Stem Cells 2008 1.12
26 Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts? Hum Mol Genet 2011 1.07
27 Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Mol Ther 2002 1.07
28 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 1.06
29 Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis. Ann Neurol 2012 1.05
30 A murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera. Pediatr Dev Pathol 2007 1.03
31 The origin and in vivo significance of murine and human culture-expanded endothelial progenitor cells. Am J Pathol 2006 1.03
32 Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. Mol Genet Metab 2004 1.02
33 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 2002 1.02
34 Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. Mol Ther 2004 0.99
35 Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice. J Neurosci 2003 0.98
36 Inner ear pathology in the mucopolysaccharidosis VII mouse. Hear Res 2002 0.98
37 Diffusion tensor imaging detects axonal injury and demyelination in the spinal cord and cranial nerves of a murine model of globoid cell leukodystrophy. NMR Biomed 2009 0.97
38 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 0.95
39 Safety of adeno-associated virus gene therapy vectors: a current evaluation. Expert Opin Drug Saf 2002 0.95
40 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 0.94
41 Lentiviral transduction of murine oligodendrocytes in vivo. J Neurosci Res 2005 0.94
42 Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy. Mol Genet Metab 2012 0.90
43 Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis. J Inherit Metab Dis 2012 0.89
44 In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice. Mol Ther 2004 0.88
45 Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases. Exp Neurol 2009 0.88
46 Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease). Biochim Biophys Acta 2013 0.87
47 Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture. J Lipid Res 2013 0.86
48 Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII. Biochem J 2004 0.86
49 Metabolic adaptations to interrupted glycosaminoglycan recycling. J Biol Chem 2009 0.83
50 Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy. J Clin Med 2012 0.81
51 Oxidative stress as a therapeutic target in globoid cell leukodystrophy. Exp Neurol 2012 0.80
52 Collection of a mobilized peripheral blood apheresis product from a patient with mucopolysaccharidosis type VII and subsequent CD34+ cell isolation. J Clin Apher 2004 0.80
53 Astrocytosis in infantile neuronal ceroid lipofuscinosis: friend or foe? Biochem Soc Trans 2014 0.79
54 Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease. Mol Ther 2003 0.79
55 Central nervous system pathology progresses independently of KC and CXCR2 in globoid-cell leukodystrophy. PLoS One 2013 0.79
56 Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease. Hum Gene Ther 2011 0.77
57 Mucopolysaccharidosis type VII: A powerful experimental system and therapeutic challenge. Pediatr Endocrinol Rev 2014 0.76
58 Treatment for Lsds: real options for several diseases. Forward. Pediatr Endocrinol Rev 2014 0.75
59 Overcoming the Next Barriers to Successful Therapy. Pediatr Endocrinol Rev 2016 0.75