Gernoux, Gwladys

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Mueller, C., Berry, J. D., McKenna-Yasek, D. M., Gernoux, G., Owegi, M. A., Pothier, L. M., Douthwright, C. L., Gelevski, D., Luppino, S. D., Blackwood, M., et al. (2020) · New England Journal of Medicine

Amyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Research

Cited by 264

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

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 227

AAV gene therapy for Tay-Sachs disease

Flotte, T. R., Cataltepe, O., Puri, A., Batista, A. R., Moser, R., McKenna-Yasek, D., Douthwright, C., Gernoux, G., Blackwood, M., Mueller, C., et al. (2022) · Nature Medicine

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 143

5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency

Mueller, C., Gernoux, G., Gruntman, A. M., Borel, F., Reeves, E. P., Calcedo, R., Rouhani, F. N., Yachnis, A., Humphries, M., Campbell-Thompson, M., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 112

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

Pfister, E. L., DiNardo, N., Mondo, E., Borel, F., Conroy, F., Fraser, C., Gernoux, G., Han, X., Hu, D., Johnson, E., et al. (2018) · Human Gene Therapy

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 95