Regulatory and Exhausted T Cell Responses to AAV Capsid

Por Gernoux, Gwladys, Wilson, James M., Mueller, Christian
Publicado no Hum Gene Ther (2017)

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

Por Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
Publicado no Mol Ther (2020)

CHOP and c-JUN up-regulate the mutant Z α(1)-antitrypsin, exacerbating its aggregation and liver proteotoxicity

Por Attanasio, Sergio, Ferriero, Rosa, Gernoux, Gwladys, De Cegli, Rossella, Carissimo, Annamaria, Nusco, Edoardo, Campione, Severo, Teckman, Jeffrey, Mueller, Christian, Piccolo, Pasquale, Brunetti-Pierri, Nicola
Publicado no J Biol Chem (2020)

Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity

Por Guilbaud, Mickaël, Devaux, Marie, Couzinié, Celia, Le Duff, Johanne, Toromanoff, Alice, Vandamme, Céline, Jaulin, Nicolas, Gernoux, Gwladys, Larcher, Thibaut, Moullier, Philippe, Le Guiner, Caroline, Adjali, Oumeya
Publicado no Hum Gene Ther (2019)

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates

Por Borel, Florie, Gernoux, Gwladys, Cardozo, Brynn, Metterville, Jake P., Toro Cabreja, Gabriela C., Song, Lina, Su, Qin, Gao, Guang Ping, Elmallah, Mai K., Brown, Robert H., Mueller, Christian
Publicado no Hum Gene Ther (2016)

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

Por Borel, Florie, Tang, Qiushi, Gernoux, Gwladys, Greer, Cynthia, Wang, Ziqiong, Barzel, Adi, Kay, Mark A., Shultz, Leonard D., Greiner, Dale L., Flotte, Terence R., Brehm, Michael A., Mueller, Christian
Publicado no Mol Ther (2017)

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 no Mol Ther Methods Clin Dev (2021)

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

Por Gruntman, Alisha M., Gernoux, Gwladys, Tang, Qiushi, Ye, Guo-Jie, Knop, Dave R., Wang, Gensheng, Benson, Janet, Coleman, Kristen E., Keeler, Allison M., Mueller, Christian, Chicoine, Louis G., Chulay, Jeffrey D., Flotte, Terence R.
Publicado no Mol Ther Methods Clin Dev (2019)

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

Por Mueller, Christian, Gernoux, Gwladys, Gruntman, Alisha M., Borel, Florie, Reeves, Emer P., Calcedo, Roberto, Rouhani, Farshid N., Yachnis, Anthony, Humphries, Margaret, Campbell-Thompson, Martha, Messina, Louis, Chulay, Jeffrey D., Trapnell, Bruce, Wilson, James M., McElvaney, Noel G., Flotte, Terence R.
Publicado no Mol Ther (2017)

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 no Mol Ther (2014)

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

Por Pfister, Edith L., DiNardo, Natalie, Mondo, Erica, Borel, Florie, Conroy, Faith, Fraser, Cara, Gernoux, Gwladys, Han, Xin, Hu, Danjing, Johnson, Emily, Kennington, Lori, Liu, PengPeng, Reid, Suzanne J., Sapp, Ellen, Vodicka, Petr, Kuchel, Tim, Morton, A. Jennifer, Howland, David, Moser, Richard, Sena-Esteves, Miguel, Gao, Guangping, Mueller, Christian, DiFiglia, Marian, Aronin, Neil
Publicado no Hum Gene Ther (2018)

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

Por Alterman, Julia F., Godinho, Bruno M.D.C., Hassler, Matthew R., Ferguson, Chantal M., Echeverria, Dimas, Sapp, Ellen, Haraszti, Reka A., Coles, Andrew H., Conroy, Faith, Miller, Rachael, Roux, Loic, Yan, Paul, Knox, Emily G., Turanov, Anton A., King, Robert M., Gernoux, Gwladys, Mueller, Christian, Gray-Edwards, Heather L., Moser, Richard P., Bishop, Nina C., Jaber, Samer M., Gounis, Matthew J., Sena-Esteves, Miguel, Pai, Athma A., DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Publicado no Nat Biotechnol (2019)