Robin J. Ziegler

Sanofi (France) — France

Medicine · Health Sciences

23h-index2.2kcitations39works0.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(25), Virus-based gene therapy research(12), Cellular transport and secretion(7), RNA Interference and Gene Delivery(7), Cytomegalovirus and herpesvirus research(7)

Publications39 total

Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice
Journal of Pharmacology and Experimental Therapeutics·2023· 10 citedOpen Access
Secreted acid sphingomyelinase as a potential gene therapy for limb girdle muscular dystrophy 2B
Journal of Clinical Investigation·2022· 43 citedOpen Access
Miglustat does not enhance alglucosidase alfa or avalglucosidase alfa efficacy in Pompe mice
Molecular Genetics and Metabolism·2021
The ΔF508 CFTR defect: molecular mechanism of suppressor mutation V510D and the contribution of transmembrane helix unraveling
2020· 1 citedOpen Access
Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
Molecular Genetics and Metabolism·2018
Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
Journal of Inherited Metabolic Disease·2017· 10 cited
Reversibility of neuropathology in Tay–Sachs-related diseases
Human Molecular Genetics·2013· 57 citedOpen Access
Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow
Molecular Therapy·2012· 76 citedOpen Access
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
Human Molecular Genetics·2011· 47 citedOpen Access
Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
Experimental Neurology·2011· 33 cited
Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
Molecular Therapy·2011· 54 citedOpen Access
Adeno‐associated virus‐mediated expression of acid sphingomyelinase decreases atherosclerotic lesion formation in apolipoprotein E<sup>−/−</sup> mice
The Journal of Gene Medicine·2011· 17 cited
Muscle Regeneration and Repair in the Pompe Mouse
Clinical Therapeutics·2011
Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
Molecular Therapy·2010· 134 citedOpen Access
Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease
Experimental Neurology·2010· 35 cited
Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice
Molecular Therapy·2010· 21 citedOpen Access
Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease
Molecular Therapy·2009· 169 citedOpen Access
Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann–Pick disease
Molecular Genetics and Metabolism·2009· 13 cited
Systemic Insulin-like Growth Factor-1 Reverses Hypoalgesia and Improves Mobility in a Mouse Model of Diabetic Peripheral Neuropathy
Molecular Therapy·2008· 48 citedOpen Access
Ability of Adeno-Associated Virus Serotype 8-Mediated Hepatic Expression of Acid α-Glucosidase to Correct the Biochemical and Motor Function Deficits of Presymptomatic and Symptomatic Pompe Mice
Human Gene Therapy·2008· 61 cited
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Frequent Co-authors

Seng H. Cheng(27), Ronald K. Scheule(12), Nelson S. Yew(11), Scott D. Bercury(9), John Marshall(7), Joseph W. Foley(7), Chester Li(6), Maribeth Cherry(6), Robert J. Desnick(6), Jennifer B. Nietupski(5), Timothy M. Cox(5), Donna Armentano(5), Christine M. Barbon(4), Lamya S. Shihabuddin(4), Bradley L. Hodges(4), Canwen Jiang(3), María Begoña Cachón-González(3), Susan Z. Wang(3), Gregory D Hurlbut(3), Malgorzata Przybylska(3)