Ronald K. Scheule

Sanofi (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

42h-index5.5kcitations130works0.02yr avg
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Research Topics

RNA Interference and Gene Delivery(44), Virus-based gene therapy research(37), Lysosomal Storage Disorders Research(25), Neonatal Respiratory Health Research(16), Viral gastroenteritis research and epidemiology(16)

Publications130 total

Liposome delivery systems
Gene Therapy·2020· 2 cited
The ΔF508 CFTR defect: molecular mechanism of suppressor mutation V510D and the contribution of transmembrane helix unraveling
2020· 1 citedOpen Access
Patient information sheets
2016
Dose preparation sheet
2016
Results of mechanistic substudy
2016
Methods: clinical trial design and outcome measures
2016
Safety and adverse events
2016· 1 cited
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis
Efficacy and Mechanism Evaluation·2016· 45 citedOpen Access
Results of clinical efficacy outcomes
2016
Cystic Fibrosis Questionnaire – Revised quality-of-life questionnaire
2016
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
The Lancet Respiratory Medicine·2015· 417 citedOpen Access
Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease
Molecular Genetics and Metabolism·2015· 20 cited
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
Molecular Medicine·2015· 99 citedOpen Access
Results of a Phase IIb non-viral gene therapy trial from the UK CF Gene Therapy Consortium
Human Gene Therapy·2014
Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
Molecular Therapy — Nucleic Acids·2014· 70 citedOpen Access
The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
Biomaterials·2013· 30 citedOpen Access
Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
PLoS ONE·2013· 34 citedOpen Access
A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
Molecular Genetics and Metabolism·2013
Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease
PLoS ONE·2012· 78 citedOpen Access
Rapid identification of novel functional promoters for gene therapy
Journal of Molecular Medicine·2012· 8 cited
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Frequent Co-authors

Seng H. Cheng(24), John Marshall(14), Simon J. Eastman(9), Nelson S. Yew(8), Jennifer B. Nietupski(8), Bradley L. Hodges(7), Robin J. Ziegler(6), Jennifer D. Tousignant(6), Karen M. Ashe(6), Craig Siegel(5), Eric W.F.W. Alton(5), Uta Griesenbach(5), Scott D. Bercury(5), Diane Copeland(5), Edward R. Lee(4), David J. Harris(4), Qiuming Chu(4), Kristin M. Taylor(4), Rodney J. Moreland(4), Mario A. Cabrera-Salazar(4)