| Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle M Penaud-Budloo, C Le Guiner, A Nowrouzi, A Toromanoff, Y Chérel, ... Journal of virology 82 (16), 7875-7885, 2008 | 302 | 2008 |
| The RNA-binding protein TIA-1 is a novel mammalian splicing regulator acting through intron sequences adjacent to a 5′ splice site F Del Gatto-Konczak, CF Bourgeois, C Le Guiner, L Kister, MC Gesnel, ... Molecular and cellular biology 20 (17), 6287-6299, 2000 | 240 | 2000 |
| Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy C Le Guiner, L Servais, M Montus, T Larcher, B Fraysse, S Moullec, ... Nature communications 8 (1), 16105, 2017 | 228 | 2017 |
| Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy T Larcher, A Lafoux, L Tesson, S Remy, V Thepenier, V François, ... PloS one 9 (10), e110371, 2014 | 173 | 2014 |
| In vivo gene regulation using tetracycline-regulatable systems K Stieger, B Belbellaa, C Le Guiner, P Moullier, F Rolling Advanced drug delivery reviews 61 (7-8), 527-541, 2009 | 170 | 2009 |
| TIA-1 and TIAR activate splicing of alternative exons with weak 5′ splice sites followed by a U-rich stretch on their own pre-mRNAs C Le Guiner, F Lejeune, D Galiana, L Kister, R Breathnach, J Stévenin, ... Journal of Biological Chemistry 276 (44), 40638-40646, 2001 | 165 | 2001 |
| Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients C Le Guiner, M Montus, L Servais, Y Cherel, V Francois, JL Thibaud, ... Molecular Therapy 22 (11), 1923-1935, 2014 | 133 | 2014 |
| Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs K Rouger, T Larcher, L Dubreil, JY Deschamps, C Le Guiner, G Jouvion, ... The American journal of pathology 179 (5), 2501-2518, 2011 | 127 | 2011 |
| Safety and efficacy of regional intravenous (ri) versus intramuscular (im) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle A Toromanoff, Y Chérel, M Guilbaud, M Penaud-Budloo, RO Snyder, ... Molecular Therapy 16 (7), 1291-1299, 2008 | 103 | 2008 |
| Serum profiling identifies novel muscle miRNA and cardiomyopathy-related miRNA biomarkers in Golden Retriever muscular dystrophy dogs and Duchenne muscular dystrophy patients L Jeanson-Leh, J Lameth, S Krimi, J Buisset, F Amor, C Le Guiner, ... The American journal of pathology 184 (11), 2885-2898, 2014 | 102 | 2014 |
| Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver A Nowrouzi, M Penaud-Budloo, C Kaeppel, U Appelt, C Le Guiner, ... Molecular Therapy 20 (6), 1177-1186, 2012 | 99 | 2012 |
| 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 |
| Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats J Hordeaux, L Dubreil, J Deniaud, F Iacobelli, S Moreau, M Ledevin, ... Gene therapy 22 (4), 316-324, 2015 | 73 | 2015 |
| Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle A Toromanoff, O Adjali, T Larcher, M Hill, L Guigand, P Chenuaud, ... Molecular Therapy 18 (1), 151-160, 2010 | 68 | 2010 |
| Establishment of two quantitative nested qPCR assays targeting the human EPO transgene EWI Neuberger, I Perez, C Le Guiner, D Moser, T Ehlert, M Allais, ... Gene therapy 23 (4), 330-339, 2016 | 54 | 2016 |
| 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 |
| Levels of inflammation and oxidative stress, and a role for taurine in dystropathology of the Golden Retriever Muscular Dystrophy dog model for Duchenne Muscular Dystrophy JR Terrill, MN Duong, R Turner, C Le Guiner, A Boyatzis, AJ Kettle, ... Redox biology 9, 276-286, 2016 | 52 | 2016 |
| Immune responses to gene product of inducible promoters CL Guiner, K Stieger, RO Snyder, F Rolling, P Moullier Current gene therapy 7 (5), 334-346, 2007 | 51 | 2007 |
| Transgene regulation using the tetracycline-inducible TetR-KRAB system after AAV-mediated gene transfer in rodents and nonhuman primates C Le Guiner, K Stieger, A Toromanoff, M Guilbaud, A Mendes-Madeira, ... PloS one 9 (9), e102538, 2014 | 45 | 2014 |
| AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A B Gautier, H Hajjar, S Soares, J Berthelot, M Deck, S Abbou, G Campbell, ... Nature communications 12 (1), 2356, 2021 | 44 | 2021 |
