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

Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy

大鼠接受微型肌营养不良蛋白基因治疗后出现剂量和基因型依赖性心律失常和猝死

Le Guiner, Caroline; Toumaniantz, Gilles; Larcher, Thibaut; Marchand, Sylvie; Buscara, Laurine; Cedrone, Grégory; Moreira, Cladice Varela; Lancelot, Amandine; Georger, Christophe; Lafoux, Aude; Couzinié, Célia; Augereau, David; Jounier, Corinne; Hivonnait, Agnès; Creoff, Estelle; Blaie, Stéphanie; Huchet, Corinne; Adjali, Oumeya; Daniele, Nathalie; Perret, Gérald; Braun, Serge

Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival

带有纳米刀片的配体修饰的rAAV6载体可在不影响细胞存活率的情况下,实现造血干细胞(HSPC)中的高水平基因敲入。

Gutierrez-Guerrero, Alejandra; Périan, Séverine; Leray, Aurélien; Martinello, Chiara; Abrey Recalde, Maria Jimena; Costa, Caroline; Herrero, Cecilia Iglesias; Bouzelha, Mohammed; Alvarez-Dorta, Dimitri; Gouin, Sébastien G; Ayuso, Eduard; Adjali, Oumeya; Büning, Hildegard; Deniaud, David; Mével, Mathieu; Verhoeyen, Els

AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear.

AAV介导的内耳基因递送可引发哺乳动物内耳中轻微的宿主免疫反应

Ishibashi Yasuko, Zhu Jianliang, Gernoux Gwladys, Yu Yunkai, Suh Michelle J, Isgrig Kevin, Grati Mhamed, Olszewski Rafal, Hoa Michael, Liang Cao, Friedman Thomas B, Adjali Oumeya, Chien Wade W

Immune-driven gene expression loss following intramuscular AAV delivery to non-human primates is only transient.

对非人灵长类动物进行肌肉注射 AAV 后,免疫驱动的基因表达丧失只是暂时的

Journou Malo, Devaux Marie, Jaulin Nicolas, Pichard Virginie, Segovia Mercedes, Moreau Aurélie, Le Duff Johanne, Cuturi Maria Cristina, Guilbaud Mickaël, Adjali Oumeya

Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer

AAV介导的基因转移后,非人灵长类动物肝脏中病毒基因组清除不完全与慢性炎症有关。

Pichard, Virginie; Guilbaud, Mickaël; Devaux, Marie; Jaulin, Nicolas; Journou, Malo; Cospolite, Magalie; Garcia, Alexandra; Ferry, Nicolas; Michalak-Provost, Sophie; Gernoux, Gwladys; Adjali, Oumeya

Generation of a compound heterozygous ABCA4 rat model with pathological features of STGD1.

构建具有 STGD1 病理特征的复合杂合子 ABCA4 大鼠模型

Morival Clément, Croyal Mikaël, Remy Séverine, Mortier Elodie, Libeau Lyse, Veziers Joëlle, Provost Nathalie, Demilly Joanna, Mendes-Madeira Alexandra, Isiegas Carolina, Tesson Laurent, Anegon Ignacio, Adjali Oumeya, Cronin Therese

Characterization of residual microRNAs in AAV vector batches produced in HEK293 mammalian cells and Sf9 insect cells

对在HEK293哺乳动物细胞和Sf9昆虫细胞中产生的AAV载体批次中残留的microRNA进行表征

Penaud-Budloo, Magalie; Lecomte, Emilie; Lecomte, Quentin; Pacouret, Simon; Broucque, Frédéric; Guy-Duché, Aurélien; Dupont, Jean-Baptiste; Jeanson-Leh, Laurence; Robin, Cécile; Blouin, Véronique; Ayuso, Eduard; Adjali, Oumeya

Dystrophin deficiency impairs cell junction formation during embryonic myogenesis from pluripotent stem cells

肌营养不良蛋白缺乏会损害多能干细胞胚胎肌发生过程中的细胞连接形成

Elise Mozin, Emmanuelle Massouridès, Virginie Mournetas, Clémence Lièvre, Audrey Bourdon, Dana L Jackson, Jonathan S Packer, Juyoung Seong, Cole Trapnell, Caroline Le Guiner, Oumeya Adjali, Christian Pinset, David L Mack, Jean-Baptiste Dupont

Dystrophin deficiency impairs cell junction formation during embryonic myogenesis

肌营养不良蛋白缺乏会损害胚胎肌肉发生过程中的细胞连接形成

Elise Mozin, Emmanuelle Massouridès, Virginie Mournetas, Clémence Lièvre, Audrey Bourdon, Dana L Jackson, Jonathan S Packer, Juyoung Seong, Cole Trapnell, Caroline Le Guiner, Oumeya Adjali, Christian Pinset, David L Mack, Jean-Baptiste Dupont

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice

胸腺内注射AAV可使治疗靶点特异性整合到小鼠的TCR基因位点。

Calabria, Andrea; Cipriani, Carlo; Spinozzi, Giulio; Rudilosso, Laura; Esposito, Simona; Benedicenti, Fabrizio; Albertini, Alessandra; Pouzolles, Marie; Luoni, Mirko; Giannelli, Serena; Broccoli, Vania; Guilbaud, Mickael; Adjali, Oumeya; Taylor, Naomi; Zimmermann, Valérie S; Montini, Eugenio; Cesana, Daniela