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List of works by Mark S Sands

A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum

scientific article published on 26 April 2018

A Hitchhiker's guide to the blood-brain barrier: in trans delivery of a therapeutic enzyme.

scientific article published on March 2014

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I.

scientific article published on 5 October 2017

A murine model of infantile neuronal ceroid lipofuscinosis-ultrastructural evaluation of storage in the central nervous system and viscera.

scientific article published on 23 May 2007

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.

scientific article published in March 2001

A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

scientific article

AAV vector integration sites in mouse hepatocellular carcinoma.

scientific article published in July 2007

AAV-Mediated Liver-Directed Gene Therapy ⬇️

scientific article published on January 1, 2011

AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.

scientific article published in July 2004

AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis.

scientific article

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.

scientific article

Abnormal immune function in vivo in a murine model of lysosomal storage disease

scientific article published on 01 June 2000

Abnormal osteoclast morphology and bone remodeling in a murine model of a lysosomal storage disease

scientific article published in February 2002

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

scientific article

Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain.

scientific article published on 17 October 2013

An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis

scientific article

Astrocytosis in infantile neuronal ceroid lipofuscinosis: friend or foe?

scientific article published in October 2014

Behavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII.

scientific article published in September 1999

Behavioral consequences of bone marrow transplantation in the treatment of murine mucopolysaccharidosis type VII.

scientific article

Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice

scientific article published on 20 June 2016

Binding of TFIIIA to derivatives of 5S RNA containing sequence substitutions or deletions defines a minimal TFIIIA binding site

scientific article

Biodistribution and efficacy of donor T lymphocytes in a murine model of lysosomal storage disease.

scientific article published in January 2003

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.

scientific article

Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine Model of Globoid-Cell Leukodystrophy.

scientific article published on January 2012

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

scientific article

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy

scientific article

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

scientific article published in March 2001

CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.

scientific article published on 20 December 2005

CNS-directed gene therapy for lysosomal storage diseases

scientific article published in April 2008

Central amygdala glucocorticoid receptor action promotes fear-associated CRH activation and conditioning

scholarly article

Central nervous system pathology progresses independently of KC and CXCR2 in globoid-cell leukodystrophy

scientific article

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

scientific article published in January 2007

Cerebellar pathology and motor deficits in the palmitoyl protein thioesterase 1-deficient mouse

scientific article published on 10 February 2009

Clinical course of sly syndrome (mucopolysaccharidosis type VII)

scientific article published in June 2016

Clinical response to persistent, low-level beta-glucuronidase expression in the murine model of mucopolysaccharidosis type VII.

scientific article published on 16 February 2007

Clinically early-stage CSPα mutation carrier exhibits remarkable terminal stage neuronal pathology with minimal evidence of synaptic loss

scientific article

Collection of a mobilized peripheral blood apheresis product from a patient with mucopolysaccharidosis type VII and subsequent CD34+ cell isolation

scientific article

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

scientific article

Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

scientific article

Comprehensive functional characterization of murine infantile Batten disease including Parkinson-like behavior and dopaminergic markers.

scientific article

Computed tomographic enhancement of liver and spleen in the dog with iodipamide ethyl ester particulate suspensions

scientific article published on 01 May 1987

Concise Synthesis of the Unnatural Sphingosine and Psychosine Enantiomer

scientific article published on June 2010

Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease)

scientific article published on September 2013

Cost-effective use of stannous pyrophosphate

scientific article published on 01 October 1981

Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice

scientific article published on 01 September 2000

Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B

scientific journal article

Diffusion tensor imaging detects axonal injury and demyelination in the spinal cord and cranial nerves of a murine model of globoid cell leukodystrophy.

scientific article

Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.

scientific article published on 28 March 2013

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.

scientific article published on 24 October 2002

Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy

scientific article

Enzyme replacement therapy for murine mucopolysaccharidosis type VII

scientific article

Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function

scientific article published on April 1998

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

scientific article published on 01 June 1996

Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse

scientific article published on 01 December 1993

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

scientific article published on December 2002

Experimental therapies in the murine model of globoid cell leukodystrophy.

scientific article

Farber disease: understanding a fatal childhood disorder and dissecting ceramide biology ⬇️

scientific article published on May 13, 2013

Gene targeting in vivo by adeno-associated virus vectors.

scientific article published on 30 July 2006

Gene therapy for lysosomal storage diseases

scientific article published on 01 October 1998

Gene therapy for lysosomal storage diseases.

scientific article

Gene therapy for murine mucopolysaccharidosis type VII.

scientific article published on July 1997

Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

scientific article published in December 2000

Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors

scientific article published on 22 July 2015

Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.

scientific article

High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector.

scientific article published on January 1995

Histochemical localization of palmitoyl protein thioesterase-1 activity

scientific article published on 11 November 2015

Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.

scientific article published in June 2004

In vivo distribution of human adipose-derived mesenchymal stem cells in novel xenotransplantation models

scientific article

In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice

scientific article published in July 2004

Inner ear pathology in the mucopolysaccharidosis VII mouse

scientific article

Insights into the Pathogenesis and Treatment of Krabbe Disease

scientific article

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

scientific article

Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

scientific article published in March 2001

Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice.

scientific article published in April 2003

Iohexol lumbar myelography: clinical study

scientific article published on 01 July 1984

Lentiviral transduction of murine oligodendrocytes in vivo.

scientific article published in November 2005

Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.

scientific article

Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease

scientific article published on 24 April 2008

Local blood-brain barrier penetration following systemic contrast medium administration. A case report and an experimental study

scientific article

Lumbar myelography with iohexol and metrizamide. A double-blind clinical trial

scientific article published on 01 January 1984

Lysosomal dysfunction results in altered energy balance

scientific article published on 2 October 2007

Marrow Stromal Cells and Osteoclast Precursors Differentially Contribute to TNF- -Induced Osteoclastogenesis In Vivo

scientific article published on 01 October 2004

Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy

scientific article published on April 2015

Metabolic adaptations to interrupted glycosaminoglycan recycling.

scientific article published on 21 August 2009

Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease

scientific article published on 29 July 2009

Mucopolysaccharidosis type VII as a model system for gene transfer to the central nervous system

scientific article published in January 1994

Mucopolysaccharidosis type VII: A powerful experimental system and therapeutic challenge

scientific article

Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease

scientific article

Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

scientific article published on April 1997

Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease

scientific article

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

scientific article published on March 1999

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.

scientific article published in January 1999

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

scientific article published on 16 January 2003

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

scientific article published in May 2004

Niemann-Pick C1 protects against atherosclerosis in mice via regulation of macrophage intracellular cholesterol trafficking

scientific article

Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.

scientific article published in April 2004

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

scientific article published in September 2001

Oligodendrocyte degeneration and recovery after focal cerebral ischemia.

scientific article published on 31 May 2010

Overcoming the Next Barriers to Successful Therapy

Oxidative stress as a therapeutic target in globoid cell leukodystrophy.

scientific article published on 28 July 2012

Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).

scientific article

Percutaneous intravenous injection in neonatal mice

scientific article published on 01 April 2000

Percutaneous intravenous injection in neonatal mice

scientific article published on 01 June 1999

Postoperative radiographic appearance of intracranial hemostatic gelatin sponge.

scientific article published in December 1986

Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

scientific article published in September 2001

Primary fibroblasts from CSPα mutation carriers recapitulate hallmarks of the adult onset neuronal ceroid lipofuscinosis

scientific article

Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases

scientific article published on 8 April 2009

Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture.

scientific article

Radioisotope imaging for detection of an ectopic spleen in a macaque

scientific article published on 01 December 1982

Retinal function is improved in a murine model of a lysosomal storage disease following bone marrow transplantation

scientific article published on 01 November 2000

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

scientific journal article

Safety of adeno-associated virus gene therapy vectors: a current evaluation

scientific article

Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

scientific article published on April 1996

Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis

scientific article published on 12 October 2006

Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

scientific article

Synergistic effects of treating the spinal cord and brain in CLN1 disease.

scientific article

Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII.

scientific article published in September 1995

TFIIIA binds to different domains of 5S RNA and the Xenopus borealis 5S RNA gene

scientific article published on November 1987

The carboxyterminal zinc fingers of TFIIIA interact with the tip of helix V of 5S RNA in the 7S ribonucleoprotein particle

scientific article published on April 1991

The origin and in vivo significance of murine and human culture-expanded endothelial progenitor cells

scientific article published on May 2006

The role of attenuated astrocyte activation in infantile neuronal ceroid lipofuscinosis

scientific article published on October 2011

The sphingolipid psychosine inhibits fast axonal transport in Krabbe disease by activation of GSK3β and deregulation of molecular motors

scientific article published on June 2013

Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

scientific article

Treatment for Krabbe's disease: Finding the combination.

scientific article published on November 2016

Treatment for Lsds: real options for several diseases. Forward

scientific article published on 01 September 2014

Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

scientific article

Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates

scientific article published in June 1993

Two zinc finger proteins from Xenopus laevis bind the same region of 5S RNA but with different nuclease protection patterns

scientific article

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.

scientific article published in July 2004

VEGF increases engraftment of bone marrow-derived endothelial progenitor cells (EPCs) into vasculature of newborn murine recipients.

scientific article

Widespread Expression of a Membrane-Tethered Version of the Soluble Lysosomal Enzyme Palmitoyl Protein Thioesterase-1.

scientific article published on 18 February 2017

Widespread nonhematopoietic tissue distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity.

scientific article

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