Gwladys Gernoux

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Julia F. Alterman, Bruno M.D.C. Godinho, Matthew Hassler et al. · Nature Biotechnology · 2019 · 220 citations

Molecular Neuroscience, Signal Transduction, Medicine +10

AAV gene therapy for Tay-Sachs disease

Terence R. Flotte, Oğuz Çataltepe, Ajit S Puri et al. · Nature Medicine · 2022 · 122 citations · Full text

Gene Therapies, Aav Gene Therapy, Pathogenesis +5

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

Christian Mueller, Gwladys Gernoux, Alisha M. Gruntman et al. · Molecular Therapy · 2017 · 105 citations · Full text

Inflammation, Primary Immunodeficiency, Disease Mechanism +10

Therapeutic rAAVrh10 Mediated <i>SOD1</i> Silencing in Adult <i>SOD1</i> ^G93A Mice and Nonhuman Primates

Florie Borel, Gwladys Gernoux, Brynn H. Cardozo et al. · Human Gene Therapy · 2015 · 103 citations · Full text

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Edith L. Pfister, Natalie DiNardo, Erica Mondo et al. · Human Gene Therapy · 2017 · 86 citations · Full text