日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

在非人灵长类动物中脑脊液内注射 AAV9 和 AAVrh10:哪些神经系统疾病有前景的途径和载体?

Bey, Karim; Deniaud, Johan; Dubreil, Laurence; Joussemet, Béatrice; Cristini, Joseph; Ciron, Carine; Hordeaux, Juliette; Le Boulc'h, Morwenn; Marche, Kevin; Maquigneau, Maud; Guilbaud, Michaël; Moreau, Rosalie; Larcher, Thibaut; Deschamps, Jack-Yves; Fusellier, Marion; Blouin, Véronique; Sevin, Caroline; Cartier, Nathalie; Adjali, Oumeya; Aubourg, Patrick; Moullier, Philippe; Colle, Marie-Anne

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

衣壳特异性清除循环中针对腺相关病毒载体的抗体

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

Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis

AAV4基因疗法治疗RPE65 Leber先天性黑蒙患者的安全性和长期疗效

Le Meur, Guylène; Lebranchu, Pierre; Billaud, Fanny; Adjali, Oumeya; Schmitt, Sébastien; Bézieau, Stéphane; Péréon, Yann; Valabregue, Romain; Ivan, Catherine; Darmon, Christophe; Moullier, Philippe; Rolling, Fabienne; Weber, Michel

Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR

重组AAV载体参考标准的实际应用:重点关注通过游离ITR qPCR进行载体基因组滴定

D'Costa, Susan; Blouin, Veronique; Broucque, Frederic; Penaud-Budloo, Magalie; François, Achille; Perez, Irene C; Le Bec, Christine; Moullier, Philippe; Snyder, Richard O; Ayuso, Eduard

Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype

理论上可通过外显子53跳跃治疗的非行走型杜氏肌营养不良症患者表现出严重的表型

Servais, Laurent; Montus, Marie; Guiner, Caroline Le; Ben Yaou, Rabah; Annoussamy, Mélanie; Moraux, Amélie; Hogrel, Jean-Yves; Seferian, Andreea M; Zehrouni, Karima; Le Moing, Anne-Gaëlle; Gidaro, Teresa; Vanhulle, Catherine; Laugel, Vincent; Butoianu, Nina; Cuisset, Jean-Marie; Sabouraud, Pascal; Cances, Claude; Klein, Andrea; Leturcq, France; Moullier, Philippe; Voit, Thomas

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates

通过全基因组计算机分析鉴定的肝脏特异性转录模块,可实现小鼠和非人灵长类动物的高效基因治疗。

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

Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer

在非人灵长类动物AAV基因转移模型中,IL10处理的树突状细胞的生成和体内评估

Moreau, Aurélie; Vandamme, Céline; Segovia, Mercedes; Devaux, Marie; Guilbaud, Mickaël; Tilly, Gaëlle; Jaulin, Nicolas; Le Duff, Johanne; Cherel, Yan; Deschamps, Jack-Yves; Anegon, Ignacio; Moullier, Philippe; Cuturi, Maria Cristina; Adjali, Oumeya

Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material

重组腺相关病毒8型参考标准物质的制备和表征

Ayuso, Eduard; Blouin, Véronique; Lock, Martin; McGorray, Susan; Leon, Xavier; Alvira, Mauricio R; Auricchio, Alberto; Bucher, Stephanie; Chtarto, Abdelwahed; Clark, K Reed; Darmon, Christophe; Doria, Monica; Fountain, Will; Gao, Guangping; Gao, Kai; Giacca, Mauro; Kleinschmidt, Juergen; Leuchs, Barbara; Melas, Catherine; Mizukami, Hiroaki; Müller, Marcus; Noordman, Yvet; Bockstael, Olivier; Ozawa, Keiya; Pythoud, Catherine; Sumaroka, Marina; Surosky, Richard; Tenenbaum, Liliane; van der Linden, Inge; Weins, Brigitte; Wright, J Fraser; Zhang, Xinhua; Zentilin, Lorena; Bosch, Fatima; Snyder, Richard O; Moullier, Philippe

Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver

rAAV载体在非人灵长类动物骨骼肌和肝脏中的整合频率和分子间重组

Nowrouzi, Ali; Penaud-Budloo, Magalie; Kaeppel, Christine; Appelt, Uwe; Le Guiner, Caroline; Moullier, Philippe; von Kalle, Christof; Snyder, Richard O; Schmidt, Manfred

Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection.

开发和应用内部阈值控制(ITC)实时PCR检测外源DNA的方法

Ni Weiyi, Le Guiner Caroline, Moullier Philippe, Snyder Richard O