Romuald Corbau

Biochemistry, Genetics and Molecular Biology · Life Sciences

16h-index912citations38works6.02yr avg
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Research Topics

Virus-based gene therapy research(14), HIV/AIDS drug development and treatment(9), HIV Research and Treatment(8), CRISPR and Genetic Engineering(8), Lysosomal Storage Disorders Research(8)

Publications38 total

FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1
Molecular Therapy·2025· 6 citedOpen Access
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Gene Therapy·2023· 36 citedOpen Access
Differences in wild-type– and R338L-tenase complex formation are at the root of R338L-factor IX assay discrepancies
Blood Advances·2022· 13 citedOpen Access
Development of a GLA nAb assay with a fully-human, neutralizing IgG4 positive control to characterize antibody response in Fabry disease patients
Molecular Genetics and Metabolism·2021
FLT201: An AAV-mediated gene therapy for type 1 Gaucher disease designed to target difficult to reach tissues
Molecular Genetics and Metabolism·2021· 3 cited
Generation of β-Glucocerebrosidase variants with increased half-life in human plasma for liver directed AAV gene therapy aimed at the treatment of Gaucher disease type 1
Molecular Genetics and Metabolism·2021· 2 cited
One-off liver directed AAV gene therapy achieves long term uptake of acid beta-glucocerebrosidase by macrophages of affected tissues in Gaucher disease
Molecular Genetics and Metabolism·2020
First-in-human study of a liver-directed AAV gene therapy (FLT190) in Fabry disease
Molecular Genetics and Metabolism·2020· 12 cited
Liver-Directed AAV Gene Therapy for Gaucher Disease
Blood·2019· 5 cited
Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate
Blood·2019
Liver-directed gene therapy corrects Fabry disease in mice
Molecular Genetics and Metabolism·2019· 8 cited
Liver directed AAV gene therapy to treat Gaucher disease
Molecular Genetics and Metabolism·2019· 1 cited
Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Blood·2018· 6 cited
Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B
Blood·2018· 4 cited
A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX
Blood·2018· 2 cited
RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1
Annals of Neurology·2016· 53 citedOpen Access
185. Safety Study by Validated Immunoassays in a Phase III Study of Subjects with Inherited Retinal Dystrophy Due to Mutations in the Gene Encoding Human Retinal Pigment Epithelium-Specific Protein 65 (RPE65) Injected with Adeno-Associated Viral Vectors
Molecular Therapy·2016· 1 citedOpen Access
ANALYSIS OF THE MODE OF ACTION OF A PSEUDOMONAS AERUGINOSA ANTI-PERSISTER COMPOUND.
PubMed·2015
Pharmacokinetics and Safety in Cynomolgus Monkeys of a Monoclonal Antibody Targeting Human Scavenger Receptor Class B Type-1 for Hepatitis C Treatment
Antiviral Therapy·2013· 2 cited
Identification of novel FLT3 kinase inhibitors
European Journal of Medicinal Chemistry·2013· 6 cited
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Frequent Co-authors

Rose Sheridan(12), Amit C. Nathwani(9), Jey M Jeyakumar(8), Azadeh Kia(8), Jenny McIntosh(8), Charles E. Mowbray(7), Manos Perros(7), Jean Rommelaere(7), Elisa Chisari(7), Carlos J. Miranda(7), Clément Cocita(6), Jonathan H. Foley(6), Erald Shehu(6), Jalpa Pandya(6), Rob Webster(5), Jürg P. F. Nüesch(5), Petya Kalcheva(5), Miriam Canavese(5), Jaminder Khinder(5), Allison Dane(5)