Lista de obras - Kim M. Hemsley - Dominio Público Uruguay

A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome

artículo científico publicado en 2016

A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome

artículo científico publicado en 2017

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

scientific article published on 10 December 2019

Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

artículo científico publicado en 2017

Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice

artículo científico publicado en 2010

Atropine acts in the ventral striatum to reduce raclopride-induced catalepsy.

artículo científico publicado en 2001

Atropine reduces raclopride-induced muscle rigidity by acting in the ventral region of the striatum

artículo científico publicado en 2002

Axonal dystrophy in the brain of mice with Sanfilippo syndrome

artículo científico publicado en 2017

Behavioural characterisation of the alpha-mannosidosis guinea pig.

artículo científico publicado en 2007

Changes in muscle tone are regulated by D1 and D2 dopamine receptors in the ventral striatum and D1 receptors in the substantia nigra.

artículo científico publicado en 2001

Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA.

artículo científico publicado en 2006

Continual Low-Dose Infusion of Sulfamidase Is Superior to Intermittent High-Dose Delivery in Ameliorating Neuropathology in the MPS IIIA Mouse Brain

scientific article published on December 2015

Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods

artículo científico publicado en 2014

Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice

artículo científico publicado en 2015

Development of cerebellar pathology in the canine model of mucopolysaccharidosis type IIIA (MPS IIIA).

artículo científico publicado en 2014

Development of motor deficits in a murine model of mucopolysaccharidosis type IIIA (MPS-IIIA).

artículo científico publicado en 2005

Directed differentiation and characterization of genetically modified embryonic stem cells for therapy

artículo científico publicado en 2006

Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease

artículo científico publicado en 2014

Dopamine receptors in the subthalamic nucleus are involved in the regulation of muscle tone in the rat.

artículo científico publicado en 2002

Early disease course is unaltered in mucopolysaccharidosis type IIIA (MPS IIIA) mice lacking α-synuclein

scientific article published on 25 April 2019

Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study.

artículo científico publicado en 2009

Embryonic stem cell-derived glial precursors as a vehicle for sulfamidase production in the MPS-IIIA mouse brain.

artículo científico publicado en 2010

Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.

artículo científico publicado en 2011

Endo-lysosomal and autophagic dysfunction: a driving factor in Alzheimer's disease?

artículo científico publicado en 2016

Enzyme replacement reduces neuropathology in MPS IIIA dogs

artículo científico publicado en 2011

Evaluation of Disease Lesions in the Developing Canine MPS IIIA Brain

artículo científico publicado en 2018

Evaluation of enzyme dose and dose-frequency in ameliorating substrate accumulation in MPS IIIA Huntaway dog brain

artículo científico publicado en 2014

Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease.

artículo científico publicado en 2009

Exocytosis is impaired in mucopolysaccharidosis IIIA mouse chromaffin cells

artículo científico publicado en 2012

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

artículo científico publicado en 2007

Genetic manipulation of murine embryonic stem cells with enhanced green fluorescence protein and sulfatase-modifying factor I genes.

artículo científico publicado en 2010

Glycosphingolipid analysis in a naturally occurring ovine model of acute neuronopathic Gaucher disease.

artículo científico publicado en 2016

Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder

artículo científico publicado en 2011

Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA.

artículo científico publicado en 2011

In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA.

artículo científico publicado en 2004

Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice.

artículo científico publicado en 2006

Intracerebral gene therapy for mucopolysaccharidosis type IIIB syndrome

artículo científico publicado en 2017

Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA.

artículo científico publicado en 2004

Intracisternal enzyme replacement therapy in lysosomal storage diseases: dispersal pathways, regional enzyme concentrations and the effect of posttreatment posture.

artículo científico publicado en 2013

Intracisternal enzyme replacement therapy in lysosomal storage diseases: routes of absorption into brain

scientific article published on 01 June 2011

Is the eye a window to the brain in Sanfilippo syndrome?

scientific article published on 17 November 2020

Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.

scientific article published on 07 May 2010

Longitudinal Magnetic Resonance Spectroscopy and Diffusion Tensor Imaging in Sheep (Ovis aries) With Quinolinic Acid Lesions of the Striatum: Time-Dependent Recovery of N-Acetylaspartate and Fractional Anisotropy

artículo científico publicado en 2020

Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder

artículo científico publicado en 2015

Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA.

artículo científico publicado en 2016

Lysosomal Dysregulation in the Murine AppNL-G-F/NL-G-F Model of Alzheimer's Disease

artículo científico publicado en 2020

MPS-IIIA mice acquire autistic behaviours with age.

artículo científico publicado en 2018

Neonatal Bone Marrow Transplantation in MPS IIIA Mice

artículo científico publicado en 2012

Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA.

artículo científico publicado en 2018

Open field locomotor activity and anxiety-related behaviors in mucopolysaccharidosis type IIIA mice

artículo científico publicado en 2008

Primary culture of neural cells isolated from the cerebellum of newborn and adult mucopolysaccharidosis type IIIA mice

artículo científico publicado en 2008

Raclopride and chlorpromazine, but not clozapine, increase muscle rigidity in the rat: relationship with D2 dopamine receptor occupancy

artículo científico publicado en 1999

Reduction in open field activity in the absence of memory deficits in the AppNL-G-F knock-in mouse model of Alzheimer's disease

artículo científico publicado en 2017

SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors

artículo científico publicado en 2010

Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric-onset neurodegenerative disease, MPS IIIA.

artículo científico publicado en 2016

Survival and engraftment of mouse embryonic stem cell-derived implants in the guinea pig brain

artículo científico publicado en 2005

Synthetic Disaccharide Standards Enable Quantitative Analysis of Stored Heparan Sulfate in MPS IIIA Murine Brain Regions

scientific article published on 18 July 2019

The effects of an irreversible dopamine receptor antagonist, N-ethoxycarbonyl-2-ethoxy-1,2-dihydroquinoline (EEDQ), on the regulation of muscle tone in the rat: the role of the substantia nigra

artículo científico publicado en 1998

Treatment of canine fucosidosis by intracisternal enzyme infusion

scientific article published on 06 May 2011

Validation of a heparan sulfate-derived disaccharide as a marker of accumulation in murine mucopolysaccharidosis type IIIA.

artículo científico publicado en 2005

Variables influencing fluorimetric N-sulfoglucosamine sulfohydrolase (SGSH) activity measurement in brain homogenates

artículo científico publicado en 2015

β-Mannosidosis in German Shepherd Dogs

scientific article published on 14 April 2019

Lista de obras de Kim M. Hemsley

A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome

A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration

Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice

Atropine acts in the ventral striatum to reduce raclopride-induced catalepsy.

Atropine reduces raclopride-induced muscle rigidity by acting in the ventral region of the striatum

Axonal dystrophy in the brain of mice with Sanfilippo syndrome

Behavioural characterisation of the alpha-mannosidosis guinea pig.

Changes in muscle tone are regulated by D1 and D2 dopamine receptors in the ventral striatum and D1 receptors in the substantia nigra.

Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA.

Continual Low-Dose Infusion of Sulfamidase Is Superior to Intermittent High-Dose Delivery in Ameliorating Neuropathology in the MPS IIIA Mouse Brain

Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods

Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice

Development of cerebellar pathology in the canine model of mucopolysaccharidosis type IIIA (MPS IIIA).

Development of motor deficits in a murine model of mucopolysaccharidosis type IIIA (MPS-IIIA).

Directed differentiation and characterization of genetically modified embryonic stem cells for therapy

Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease

Dopamine receptors in the subthalamic nucleus are involved in the regulation of muscle tone in the rat.

Early disease course is unaltered in mucopolysaccharidosis type IIIA (MPS IIIA) mice lacking α-synuclein

Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study.

Embryonic stem cell-derived glial precursors as a vehicle for sulfamidase production in the MPS-IIIA mouse brain.

Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.

Endo-lysosomal and autophagic dysfunction: a driving factor in Alzheimer's disease?

Enzyme replacement reduces neuropathology in MPS IIIA dogs

Evaluation of Disease Lesions in the Developing Canine MPS IIIA Brain

Evaluation of enzyme dose and dose-frequency in ameliorating substrate accumulation in MPS IIIA Huntaway dog brain

Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease.

Exocytosis is impaired in mucopolysaccharidosis IIIA mouse chromaffin cells

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

Genetic manipulation of murine embryonic stem cells with enhanced green fluorescence protein and sulfatase-modifying factor I genes.

Glycosphingolipid analysis in a naturally occurring ovine model of acute neuronopathic Gaucher disease.

Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder

Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA.

In vitro characterization of genetically modified embryonic stem cells as a therapy for murine mucopolysaccharidosis type IIIA.

Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice.

Intracerebral gene therapy for mucopolysaccharidosis type IIIB syndrome

Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA.

Intracisternal enzyme replacement therapy in lysosomal storage diseases: dispersal pathways, regional enzyme concentrations and the effect of posttreatment posture.

Intracisternal enzyme replacement therapy in lysosomal storage diseases: routes of absorption into brain

Is the eye a window to the brain in Sanfilippo syndrome?

Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.

Longitudinal Magnetic Resonance Spectroscopy and Diffusion Tensor Imaging in Sheep (Ovis aries) With Quinolinic Acid Lesions of the Striatum: Time-Dependent Recovery of N-Acetylaspartate and Fractional Anisotropy

Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder

Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA.

Lysosomal Dysregulation in the Murine AppNL-G-F/NL-G-F Model of Alzheimer's Disease

MPS-IIIA mice acquire autistic behaviours with age.

Neonatal Bone Marrow Transplantation in MPS IIIA Mice

Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA.

Open field locomotor activity and anxiety-related behaviors in mucopolysaccharidosis type IIIA mice

Primary culture of neural cells isolated from the cerebellum of newborn and adult mucopolysaccharidosis type IIIA mice

Raclopride and chlorpromazine, but not clozapine, increase muscle rigidity in the rat: relationship with D2 dopamine receptor occupancy

Reduction in open field activity in the absence of memory deficits in the AppNL-G-F knock-in mouse model of Alzheimer's disease

SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors

Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric-onset neurodegenerative disease, MPS IIIA.

Survival and engraftment of mouse embryonic stem cell-derived implants in the guinea pig brain

Synthetic Disaccharide Standards Enable Quantitative Analysis of Stored Heparan Sulfate in MPS IIIA Murine Brain Regions

The effects of an irreversible dopamine receptor antagonist, N-ethoxycarbonyl-2-ethoxy-1,2-dihydroquinoline (EEDQ), on the regulation of muscle tone in the rat: the role of the substantia nigra

Treatment of canine fucosidosis by intracisternal enzyme infusion

Validation of a heparan sulfate-derived disaccharide as a marker of accumulation in murine mucopolysaccharidosis type IIIA.

Variables influencing fluorimetric N-sulfoglucosamine sulfohydrolase (SGSH) activity measurement in brain homogenates

β-Mannosidosis in German Shepherd Dogs