List of works - John Marshall

A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

scientific article published on 16 May 2007

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

scientific article published in June 2006

Antitumor activity of cationic lipid complexed with immunostimulatory DNA

scientific article published on 01 October 2001

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.

scientific article published on 07 March 2016

Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia

scientific article published on 01 January 1998

Contribution of Plasmid DNA to Inflammation in the Lung after Administration of Cationic Lipid:pDNA Complexes

scientific article published on 01 January 1999

Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease

scientific article

Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease

scientific article

Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.

scientific article published in February 2012

Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy

scientific article published on 25 March 2010

Increased duration of transgene expression in the lung with plasmid DNA vectors harboring adenovirus E4 open reading frame 3.

scientific article published on July 1999

Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease.

scientific article published on 5 May 2010

Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease.

scientific article published on 29 July 2010

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A ⬇️

scientific article published on July 24, 2012

Monoclonal antibodies to novel erythroid differentiation antigens reveal specific effects of oncogenes on the leukaemic cell phenotype.

scientific article

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

scientific article published on 24 August 2010

Primitive series embryonic chick erythrocytes express the transferrin receptor

scientific article published on 01 May 1986

Production of cystic fibrosis transmembrane conductance regulator in the milk of transgenic mice

scientific article published on 01 January 1992

Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease

scientific article (publication date: 24 November 2010)

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

scientific article published on 28 June 2011

List of works by John Marshall

A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

Antitumor activity of cationic lipid complexed with immunostimulatory DNA

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.

Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia

Contribution of Plasmid DNA to Inflammation in the Lung after Administration of Cationic Lipid:pDNA Complexes

Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease

Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease

Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann-Pick C mice.

Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy

Increased duration of transgene expression in the lung with plasmid DNA vectors harboring adenovirus E4 open reading frame 3.

Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease.

Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease.

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A ⬇️

Monoclonal antibodies to novel erythroid differentiation antigens reveal specific effects of oncogenes on the leukaemic cell phenotype.

Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy

Primitive series embryonic chick erythrocytes express the transferrin receptor

Production of cystic fibrosis transmembrane conductance regulator in the milk of transgenic mice

Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

α-Galactosidase A knockout mice: progressive organ pathology resembles the type 2 later-onset phenotype of Fabry disease