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

High value of (64)Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease

高 (64)Cu 值可作为评估威尔逊病基因治疗后生理性铜排泄恢复情况的工具

Murillo, Oihana; Collantes, Maria; Gazquez, Cristina; Moreno, Daniel; Hernandez-Alcoceba, Ruben; Barberia, Miren; Ecay, Margarita; Tamarit, Blanche; Douar, Anne; Ferrer, Veronica; Combal, Jean Philippe; Peñuelas, Ivan; Bénichou, Bernard; Gonzalez-Aseguinolaza, Gloria

Optimising the IgG-degrading enzyme treatment regimen for enhanced adeno-associated virus transduction in the presence of neutralising antibodies

优化 IgG 降解酶治疗方案以在存在中和抗体的情况下增强腺相关病毒的转导

Irene Ros-Gañán, Mirja Hommel, Laia Trigueros-Motos, Blanche Tamarit, Estefanía Rodríguez-García, David Salas, Guiomar Pérez, Anne Douar, Jean Philippe Combal, Bernard Benichou, Veronica Ferrer, Gloria González-Aseguinolaza

Optogenetic therapy: high spatiotemporal resolution and pattern discrimination compatible with vision restoration in non-human primates

光遗传疗法:高时空分辨率和模式辨别能力,与非人灵长类动物的视觉恢复相兼容

Gauvain, Gregory; Akolkar, Himanshu; Chaffiol, Antoine; Arcizet, Fabrice; Khoei, Mina A; Desrosiers, Mélissa; Jaillard, Céline; Caplette, Romain; Marre, Olivier; Bertin, Stéphane; Fovet, Claire-Maelle; Demilly, Joanna; Forster, Valérie; Brazhnikova, Elena; Hantraye, Philippe; Pouget, Pierre; Douar, Anne; Pruneau, Didier; Chavas, Joël; Sahel, José-Alain; Dalkara, Deniz; Duebel, Jens; Benosman, Ryad; Picaud, Serge

Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

使用腺相关病毒血清型 Anc80 在小鼠模型中提供苯丙酮尿症的持久治愈

Robert A Kaiser, Nicholas D Weber, Laia Trigueros-Motos, Kari L Allen, Michael Martinez, William Cao, Caitlin J VanLith, Lori G Hillin, Anne Douar, Gloria González-Aseguinolaza, Rafael Aldabe, Joseph B Lillegard

Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

临床相关小鼠模型中对进行性家族性肝内胆汁淤积症 3 型进行基因治疗

Nicholas D Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou

Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material

重组腺相关病毒2型参考标准物质的特性分析

Lock, Martin; McGorray, Susan; Auricchio, Alberto; Ayuso, Eduard; Beecham, E Jeffrey; Blouin-Tavel, Véronique; Bosch, Fatima; Bose, Mahuya; Byrne, Barry J; Caton, Tina; Chiorini, John A; Chtarto, Abdelwahed; Clark, K Reed; Conlon, Thomas; Darmon, Christophe; Doria, Monica; Douar, Anne; Flotte, Terence R; Francis, Joyce D; Francois, Achille; Giacca, Mauro; Korn, Michael T; Korytov, Irina; Leon, Xavier; Leuchs, Barbara; Lux, Gabriele; Melas, Catherine; Mizukami, Hiroaki; Moullier, Philippe; Müller, Marcus; Ozawa, Keiya; Philipsberg, Tina; Poulard, Karine; Raupp, Christina; Rivière, Christel; Roosendaal, Sigrid D; Samulski, R Jude; Soltys, Steven M; Surosky, Richard; Tenenbaum, Liliane; Thomas, Darby L; van Montfort, Bart; Veres, Gabor; Wright, J Fraser; Xu, Yili; Zelenaia, Olga; Zentilin, Lorena; Snyder, Richard O

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

静脉注射自互补的 AAV9 可将转基因递送至成人运动神经元

Sandra Duque, Béatrice Joussemet, Christel Riviere, Thibaut Marais, Laurence Dubreil, Anne-Marie Douar, John Fyfe, Philippe Moullier, Marie-Anne Colle, Martine Barkats