| SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS C Mueller, JD Berry, DM McKenna-Yasek, G Gernoux, MA Owegi, ... New England Journal of Medicine 383 (2), 151-158, 2020 | 174 | 2020 |
| A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system JF Alterman, BMDC Godinho, MR Hassler, CM Ferguson, D Echeverria, ... Nature biotechnology 37 (8), 884-894, 2019 | 150 | 2019 |
| Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1G93A Mice and Nonhuman Primates F Borel, G Gernoux, B Cardozo, JP Metterville, G T. Cabrera, L Song, ... Human gene therapy 27 (1), 19-31, 2016 | 118 | 2016 |
| Artificial miRNAs reduce human mutant Huntingtin throughout the striatum in a transgenic sheep model of Huntington's disease EL Pfister, N DiNardo, E Mondo, F Borel, F Conroy, C Fraser, G Gernoux, ... Human gene therapy 29 (6), 663-673, 2018 | 99 | 2018 |
| 5 Year expression and neutrophil defect repair after gene therapy in alpha-1 antitrypsin deficiency C Mueller, G Gernoux, AM Gruntman, F Borel, EP Reeves, R Calcedo, ... Molecular Therapy 25 (6), 1387-1394, 2017 | 98 | 2017 |
| Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates MK Chuah, I Petrus, P De Bleser, C Le Guiner, G Gernoux, O Adjali, ... Molecular Therapy 22 (9), 1605-1613, 2014 | 86 | 2014 |
| Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques F Borel, G Gernoux, H Sun, R Stock, M Blackwood, RH Brown Jr, ... Science Translational Medicine 10 (465), eaau6414, 2018 | 74 | 2018 |
| Survival advantage of both human hepatocyte xenografts and genome-edited hepatocytes for treatment of α-1 antitrypsin deficiency F Borel, Q Tang, G Gernoux, C Greer, Z Wang, A Barzel, MA Kay, ... Molecular Therapy 25 (11), 2477-2489, 2017 | 73 | 2017 |
| AAV gene therapy for Tay-Sachs disease TR Flotte, O Cataltepe, A Puri, AR Batista, R Moser, D McKenna-Yasek, ... Nature medicine 28 (2), 251-259, 2022 | 56 | 2022 |
| Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping W Ni, C Le Guiner, G Gernoux, M Penaud-Budloo, P Moullier, RO Snyder Gene therapy 18 (7), 709-718, 2011 | 53 | 2011 |
| Regulatory and exhausted T cell responses to AAV capsid G Gernoux, JM Wilson, C Mueller Human gene therapy 28 (4), 338-349, 2017 | 51 | 2017 |
| Five years of successful inducible transgene expression following locoregional adeno-associated virus delivery in nonhuman primates with no detectable immunity M Guilbaud, M Devaux, C Couzinie, J Le Duff, A Toromanoff, ... Human Gene Therapy 30 (7), 802-813, 2019 | 36 | 2019 |
| Muscle-directed delivery of an AAV1 vector leads to capsid-specific T cell exhaustion in nonhuman primates and humans G Gernoux, AM Gruntman, M Blackwood, M Zieger, TR Flotte, C Mueller Molecular Therapy 28 (3), 747-757, 2020 | 30 | 2020 |
| CHOP and c-JUN up-regulate the mutant Z α1-antitrypsin, exacerbating its aggregation and liver proteotoxicity S Attanasio, R Ferriero, G Gernoux, R De Cegli, A Carissimo, E Nusco, ... Journal of Biological Chemistry 295 (38), 13213-13223, 2020 | 17 | 2020 |
| Early interaction of adeno-associated virus serotype 8 vector with the host immune system following intramuscular delivery results in weak but detectable lymphocyte and … G Gernoux, M Guilbaud, L Dubreil, T Larcher, C Babarit, M Ledevin, ... Human gene therapy 26 (1), 1-13, 2015 | 16 | 2015 |
| Bridging from intramuscular to limb perfusion delivery of rAAV: optimization in a non-human primate study AM Gruntman, G Gernoux, Q Tang, GJ Ye, DR Knop, G Wang, J Benson, ... Molecular Therapy-Methods & Clinical Development 13, 233-242, 2019 | 11 | 2019 |
| AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation G Gernoux, M Guilbaud, M Devaux, M Journou, V Pichard, N Jaulin, ... Molecular Therapy-Methods & Clinical Development 20, 660-674, 2021 | 5 | 2021 |
| Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice M Zieger, F Borel, C Greer, G Gernoux, M Blackwood, TR Flotte, C Mueller Molecular Therapy-Methods & Clinical Development 25, 425-438, 2022 | 4 | 2022 |
| 24. Sustained Expression with Partial Correction of Neutrophil Defects 5 Years After Intramuscular rAAV1 Gene Therapy for Alpha-1 Antitrypsin Deficiency C Mueller, JD Chulay, NG McElvaney, G Gernoux, EP Reeves, ... Molecular Therapy 24, S11-S12, 2016 | 3 | 2016 |
| CHOP-c-JUN complex plays a critical role in liver proteotoxicity induced by mutant Z alpha-1 antitrypsin S Attanasio, G Gernoux, R Ferriero, R De Cegli, A Carissimo, E Nusco, ... bioRxiv, 2020.05. 04.076752, 2020 | 2 | 2020 |
