未登录Capsid-specific removal of circulating antibodies to adeno-associated virus vectors
Bertin, Berangere; Veron, Philippe; Leborgne, Christian; Deschamps, Jack-Yves; Moullec, Sophie; Fromes, Yves; Collaud, Fanny; Boutin, Sylvie; Latournerie, Virginie; van Wittenberghe, Laetitia; Delache, Benoit; Le Grand, Roger; Dereuddre-Bosquet, Nathalie; Benveniste, Olivier; Moullier, Philippe; Masurier, Carole; Merten, Otto; Mingozzi, Federico .png)
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收藏Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8+ T Cells in Human Donors Allows the Detection of a TEMRA SubpopulationVandamme, Celine; Xicluna, Rebecca; Hesnard, Leslie; Devaux, Marie; Jaulin, Nicolas; Guilbaud, Mickael; Le Duff, Johanne; Couzinie, Celia; Moullier, Philippe; Saulquin, Xavier; Adjali, Oumeya 分享 收藏 Chemical modification of the adeno-associated virus capsid to improve gene deliveryMevel, Mathieu; Bouzelha, Mohammed; Leray, Aurelien; Pacouret, Simon; Guilbaud, Mickael; Penaud-Budloo, Magalie; Alvarez-Dorta, Dimitri; Dubreil, Laurence; Gouin, Sebastien G.; Combal, Jean Philippe; Hommel, Mirja; Gonzalez-Aseguinolaza, Gloria; Blouin, Veronique; Moullier, Philippe; Adjali, Oumeya; Deniaud, David; Ayuso, Eduard 分享 收藏 RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target EffectDomenger, Claire; Allais, Marine; Francois, Virginie; Leger, Adrien; Lecomte, Emilie; Montus, Marie; Servais, Laurent; Voit, Thomas; Moullier, Philippe; Audic, Yann; Le Guiner, Caroline 分享 收藏 Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with &ITRPE65 &ITLeber Congenital AmaurosisLe Meur, Guylene; Lebranchu, Pierre; Billaud, Fanny; Adjali, Oumeya; Schmitt, Sebastien; Bezieau, Stephan; Pereon, Yann; Valabregue, Romain; Ivan, Catherine; Darmon, Christophe; Moullier, Philippe; Rolling, Fabienne; Weber, Michel 分享 收藏 Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophyLe Guiner, Caroline; Servais, Laurent; Montus, Marie; Larcher, Thibaut; Fraysse, Bodvael; Moullec, Sophie; Allais, Marine; Francois, Virginie; Dutilleul, Maeva; Malerba, Alberto; Koo, Taeyoung; Thibaut, Jean-Laurent; Matot, Beatrice; Devaux, Marie; Le Duff, Johanne; Deschamps, Jack-Yves; Barthelemy, Ines; Blot, Stephane; Testault, Isabelle; Wahbi, Karim; Ederhy, Stephane; Martin, Samia; Veron, Philippe; Georger, Christophe; Athanasopoulos, Takis; Masurier, Carole; Mingozzi, Federico; Carlier, Pierre; Gjata, Bernard; Hogrel, Jean-Yves; Adjali, Oumeya; Mavilio, Fulvio; Voit, Thomas; Moullier, Philippe; Dickson, George 分享 收藏 AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV PreparationsPacouret, Simon; Bouzelha, Mohammed; Shelke, Rajani; Andres-Mateos, Eva; Xiao, Ru; Maurer, Anna; Mevel, Mathieu; Turunen, Heikki; Barungi, Trisha; Penaud-Budloo, Magalie; Broucque, Frederic; Blouin, Veronique; Moullier, Philippe; Ayuso, Eduard; Vandenberghe, Luk H. 分享 收藏 Impact of a Treatment with Antioxidant on Gene Transfer Efficiency After Recombinant Adeno-Associated Vector Injection in a Mouse Model of Duchenne Muscular DystrophyDupont, Jean-Baptiste; Tournaire, Benoit; Durand, Romain; Marolleau, Beatrice; Bertil, Emilie; Georger, Christophe; Lecomte, Emilie; Cogne, Benjamin; Gjata, Bernard; van Wittenberghe, Laetitia; Vignaud, Alban; Snyder, Richard O.; Moullier, Philippe; Leger, Adrien 分享 收藏 Phenotypic and Functional Characterisation of Human Anti-AAV CD8+T Cells Using MHC Class I Tetramer-Associated Magnetic EnrichmentVandamme, Celine; Devaux, Marie; Jaulin, Nicolas; Guilbaud, Mickael; Le Duff, Johanne; Hesnard, Leslie; Djamai, Hanane; Bonneville, Marc; Moullier, Philippe; Saulquin, Xavier; Adjali, Oumeya 分享 收藏 AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient DogsPichard, Virginie; Provost, Nathalie; Mendes-Madeira, Alexandra; Libeau, Lyse; Hulin, Philippe; Tshilenge, Kizito-Tshitoko; Biget, Marine; Ameline, Baptiste; Deschamps, Jack-Yves; Weber, Michel; Le Meur, Guylene; Colle, Marie-Anne; Moullier, Philippe; Rolling, Fabienne 分享 收藏 Development and Validation of Identity and Homogeneity Assays for AAV PreparationsPacouret, Simon; Mevel, Mathieu; Bouzelha, Mohammed; Maurer, Anna; Shelke, Rajani; Xiao, Ru; Penaud-Budloo, Magalie; Turunen, Heikki; Broucque, Frederic; Blouin, Veronique; Moullier, Philippe; Ayuso, Eduard; Vandenberghe, Luk H. 分享 收藏 分享 收藏 Minimally Effective Dose of Systemic AAV8-MTM1 Needed To Prolong Survival and Correct Severe Muscle Pathology in a Canine Model of X-Linked Myotubular MyopathyMack, David L.; Poulard, Karine; Goddard, Melissa; Snyder, Jessica M.; Grange, Robert W.; Doering, Jon; Czerniecki, Stefan; Strande, Jennifer L.; Latournerie, Virginie; Veron, Philippe; Meng, Hui; Yang, Lin; Liu, Fujun; Le Bec, Christine; Buscara, Laurine; Martin, Samia; Moullier, Philippe; O'Callaghan, Michael; Mingozzi, Federico; Beggs, Alan H.; Lawlor, Michael W.; Buj-Bello, Anna; Mavilio, Fulvio; Childers, Martin K. 分享 收藏 Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation SequencingLecomte, Emilie; Tournaire, Benoit; Cogne, Benjamin; Dupont, Jean-Baptiste; Lindenbaum, Pierre; Martin-Fontaine, Melanie; Broucque, Frederic; Robin, Cecile; Hebben, Matthias; Merten, Otto-Wilhelm; Blouin, Veronique; Francois, Achille; Redon, Richard; Moullier, Philippe; Leger, Adrien 分享 收藏 Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne PatientsLe Guiner, Caroline; Montus, Marie; Servais, Laurent; Cherel, Yan; Francois, Virginie; Thibaud, Jean-Laurent; Wary, Claire; Matot, Beatrice; Larcher, Thibaut; Guigand, Lydie; Dutilleul, Maeva; Domenger, Claire; Allais, Marine; Beuvin, Maud; Moraux, Amelie; Le Duff, Johanne; Devaux, Marie; Jaulin, Nicolas; Guilbaud, Mickael; Latournerie, Virginie; Veron, Philippe; Boutin, Sylvie; Leborgne, Christian; Desgue, Diana; Deschamps, Jack-Yves; Moullec, Sophie; Fromes, Yves; Vulin, Adeline; Smith, Richard H.; Laroudie, Nicolas; Barnay-Toutain, Frederic; Riviere, Christel; Bucher, Stephanie; Thanh-Hoa Le; Delaunay, Nicolas; Gasmi, Mehdi; Kotin, Robert M.; Bonne, Gisele; Adjali, Oumeya; Masurier, Carole; Hogrel, Jean-Yves; Carlier, Pierre; Moullier, Philippe; Voit, Thomas 分享 收藏 Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human PrimatesChuah, 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, Veronique; Christophe, Olivier; Moullier, Philippe; Mingozzi, Federico; VandenDriessche, Thierry 分享 收藏 分享 收藏 Successful Gene Therapy in the RPGRIP1-deficient Dog: a Large Model of Cone-Rod DystrophyLheriteau, Elsa; Petit, Lolita; Weber, Michel; Le Meur, Guylene; Deschamps, Jack-Yves; Libeau, Lyse; Mendes-Madeira, Alexandra; Guihal, Caroline; Francois, Achille; Guyon, Richard; Provost, Nathalie; Lemoine, Francoise; Papal, Samantha; El-Amraoui, Aziz; Colle, Marie-Anne; Moullier, Philippe; Rolling, Fabienne 分享 收藏 Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular MyopathyChilders, Martin K.; Joubert, Romain; Poulard, Karine; Moal, Christelle; Grange, Robert W.; Doering, Jonathan A.; Lawlor, Michael W.; Rider, Branden E.; Jamet, Thibaud; Daniele, Nathalie; Martin, Samia; Riviere, Christel; Soker, Thomas; Hammer, Caroline; Van Wittenberghe, Laetitia; Lockard, Mandy; Guan, Xuan; Goddard, Melissa; Mitchell, Erin; Barber, Jane; Williams, J. Koudy; Mack, David L.; Furth, Mark E.; Vignaud, Alban; Masurier, Carole; Mavilio, Fulvio; Moullier, Philippe; Beggs, Alan H.; Buj-Bello, Anna 分享 收藏 分享 收藏 
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