Development and Utility of an Internal Threshold Control (ITC) Real-Time PCR Assay for Exogenous DNA Detection

por Ni, Weiyi, Le Guiner, Caroline, Moullier, Philippe, Snyder, Richard O.
Publicado 2012

In vivo gene regulation using tetracycline-regulatable systems()

por Stieger, Knut, Belbellaa, Brahim, Le Guiner, Caroline, Moullier, Philippe, Rolling, Fabienne
Publicado 2009

Characterization of brain dystrophins absence and impact in dystrophin-deficient Dmd(mdx) rat model

por Caudal, Dorian, François, Virginie, Lafoux, Aude, Ledevin, Mireille, Anegon, Ignacio, Le Guiner, Caroline, Larcher, Thibaut, Huchet, Corinne
Publicado 2020

Gait characterization in golden retriever muscular dystrophy dogs using linear discriminant analysis

por Fraysse, Bodvaël, Barthélémy, Inès, Qannari, El Mostafa, Rouger, Karl, Thorin, Chantal, Blot, Stéphane, Le Guiner, Caroline, Chérel, Yan, Hogrel, Jean-Yves
Publicado 2017

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

por Mariot, Virginie, Le Guiner, Caroline, Barthélémy, Inès, Montus, Marie, Blot, Stéphane, Torelli, Silvia, Morgan, Jennifer, Muntoni, Francesco, Voit, Thomas, Dumonceaux, Julie
Publicado 2020

Adeno-Associated Viral Vector-Mediated Transgene Expression Is Independent of DNA Methylation in Primate Liver and Skeletal Muscle

por Léger, Adrien, Le Guiner, Caroline, Nickerson, Michael L., McGee Im, Kate, Ferry, Nicolas, Moullier, Philippe, Snyder, Richard O., Penaud-Budloo, Magalie
Publicado 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

por Terrill, Jessica R., Duong, Marisa N., Turner, Rufus, Le Guiner, Caroline, Boyatzis, Amber, Kettle, Anthony J., Grounds, Miranda D., Arthur, Peter G.
Publicado 2016

RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect

por Domenger, Claire, Allais, Marine, François, Virginie, Léger, Adrien, Lecomte, Emilie, Montus, Marie, Servais, Laurent, Voit, Thomas, Moullier, Philippe, Audic, Yann, Le Guiner, Caroline
Publicado 2017

AAV2/9-mediated gene transfer into murine lacrimal gland leads to a long-term targeted tear film modification

por Gautier, Benoit, Meneux, Léna, Feret, Nadège, Audrain, Christine, Hudecek, Laetitia, Kuony, Alison, Bourdon, Audrey, Le Guiner, Caroline, Blouin, Véronique, Delettre, Cécile, Michon, Frédéric
Publicado 2022

Transgene Regulation Using the Tetracycline-Inducible TetR-KRAB System after AAV-Mediated Gene Transfer in Rodents and Nonhuman Primates

por Le Guiner, Caroline, Stieger, Knut, Toromanoff, Alice, Guilbaud, Mickaël, Mendes-Madeira, Alexandra, Devaux, Marie, Guigand, Lydie, Cherel, Yan, Moullier, Philippe, Rolling, Fabienne, Adjali, Oumeya
Publicado 2014

Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

por Farrokhi, Vahid, Walsh, Jason, Palandra, Joe, Brodfuehrer, Joanne, Caiazzo, Teresa, Owens, Jane, Binks, Michael, Neelakantan, Srividya, Yong, Florence, Dua, Pinky, Le Guiner, Caroline, Neubert, Hendrik
Publicado 2021

Muscle Pathology in Dystrophic Rats and Zebrafish Is Unresponsive to Taurine Treatment, Compared to the mdx Mouse Model for Duchenne Muscular Dystrophy

por Terrill, Jessica R., Huchet, Corinne, Le Guiner, Caroline, Lafoux, Aude, Caudal, Dorian, Tulangekar, Ankita, Bryson-Richardson, Robert J., Sztal, Tamar E., Grounds, Miranda D., Arthur, Peter G.
Publicado 2023

AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation

por Gernoux, Gwladys, Guilbaud, Mickaël, Devaux, Marie, Journou, Malo, Pichard, Virginie, Jaulin, Nicolas, Léger, Adrien, Le Duff, Johanne, Deschamps, Jack-Yves, Le Guiner, Caroline, Moullier, Philippe, Cherel, Yan, Adjali, Oumeya
Publicado 2021

TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD

por Creisméas, Anna, Gazaille, Claire, Bourdon, Audrey, Lallemand, Marc-Antoine, François, Virginie, Allais, Marine, Ledevin, Mireille, Larcher, Thibaut, Toumaniantz, Gilles, Lafoux, Aude, Huchet, Corinne, Anegon, Ignacio, Adjali, Oumeya, Le Guiner, Caroline, Fraysse, Bodvaël
Publicado 2021

Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution

por Ducloyer, Jean-Baptiste, Pichard, Virginie, Mevel, Mathieu, Galy, Anne, Lefevre, Gaelle M., Brument, Nicole, Alvarez-Dorta, Dimitri, Deniaud, David, Mendes-Madeira, Alexandra, Libeau, Lyse, Le Guiner, Caroline, Cronin, Therese, Adjali, Oumeya, Weber, Michel, Le Meur, Guylène
Publicado 2023

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

por Le Guiner, Caroline, Xiao, Xiao, Larcher, Thibaut, Lafoux, Aude, Huchet, Corinne, Toumaniantz, Gilles, Adjali, Oumeya, Anegon, Ignacio, Remy, Séverine, Grieger, Josh, Li, Juan, Farrokhi, Vahid, Neubert, Hendrik, Owens, Jane, McIntyre, Maritza, Moullier, Philippe, Samulski, R. Jude
Publicado 2023

AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A

por Gautier, Benoit, Hajjar, Helene, Soares, Sylvia, Berthelot, Jade, Deck, Marie, Abbou, Scarlette, Campbell, Graham, Ceprian, Maria, Gonzalez, Sergio, Fovet, Claire-Maëlle, Schütza, Vlad, Jouvenel, Antoine, Rivat, Cyril, Zerah, Michel, François, Virginie, Le Guiner, Caroline, Aubourg, Patrick, Fledrich, Robert, Tricaud, Nicolas
Publicado 2021

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy

por Larcher, Thibaut, Lafoux, Aude, Tesson, Laurent, Remy, Séverine, Thepenier, Virginie, François, Virginie, Le Guiner, Caroline, Goubin, Helicia, Dutilleul, Maéva, Guigand, Lydie, Toumaniantz, Gilles, De Cian, Anne, Boix, Charlotte, Renaud, Jean-Baptiste, Cherel, Yan, Giovannangeli, Carine, Concordet, Jean-Paul, Anegon, Ignacio, Huchet, Corinne
Publicado 2014

Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA

por Dupont, Jean-Baptiste, Tournaire, Benoit, Georger, Christophe, Marolleau, Béatrice, Jeanson-Leh, Laurence, Ledevin, Mireille, Lindenbaum, Pierre, Lecomte, Emilie, Cogné, Benjamin, Dubreil, Laurence, Larcher, Thibaut, Gjata, Bernard, Van Wittenberghe, Laetitia, Le Guiner, Caroline, Penaud-Budloo, Magalie, Snyder, Richard O, Moullier, Philippe, Léger, Adrien
Publicado 2015

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

por Chuah, Marinee K, Petrus, Inge, De Bleser, Pieter, Le Guiner, Caroline, Gernoux, Gwladys, Adjali, Oumeya, Nair, Nisha, Willems, Jessica, Evens, Hanneke, Rincon, Melvin Y, Matrai, Janka, Di Matteo, Mario, Samara-Kuko, Ermira, Yan, Bing, Acosta-Sanchez, Abel, Meliani, Amine, Cherel, Ghislaine, Blouin, Véronique, Christophe, Olivier, Moullier, Philippe, Mingozzi, Federico, VandenDriessche, Thierry
Publicado 2014