Regulatory and Exhausted T Cell Responses to AAV Capsid

著者: Gernoux, Gwladys, Wilson, James M., Mueller, Christian
出版年 Hum Gene Ther (2017)

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

著者: Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
出版年 Mol Ther (2020)

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

著者: Attanasio, Sergio, Ferriero, Rosa, Gernoux, Gwladys, De Cegli, Rossella, Carissimo, Annamaria, Nusco, Edoardo, Campione, Severo, Teckman, Jeffrey, Mueller, Christian, Piccolo, Pasquale, Brunetti-Pierri, Nicola
出版年 J Biol Chem (2020)

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

著者: 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
出版年 Hum Gene Ther (2019)

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

著者: 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
出版年 Hum Gene Ther (2016)

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

著者: 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
出版年 Mol Ther (2017)

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

著者: 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
出版年 Mol Ther Methods Clin Dev (2021)

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

著者: 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.
出版年 Mol Ther Methods Clin Dev (2019)

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

著者: 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.
出版年 Mol Ther (2017)

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

著者: 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
出版年 Mol Ther (2014)

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

著者: 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
出版年 Hum Gene Ther (2018)

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

著者: 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
出版年 Nat Biotechnol (2019)