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

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

Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle.

将 rAAV 进行区域静脉注射 (RI) 到非人灵长类动物骨骼肌后未观察到免疫毒性

Toromanoff Alice, Adjali Oumeya, Larcher Thibaut, Hill Marcelo, Guigand Lydie, Chenuaud Pierre, Deschamps Jack-Yves, Gauthier Olivier, Blancho Gilles, Vanhove Bernard, Rolling Fabienne, Chérel Yan, Moullier Philippe, Anegon Ignacio, Le Guiner Caroline

In vivo gene regulation using tetracycline-regulatable systems

利用四环素可调控系统进行体内基因调控

Stieger, Knut; Belbellaa, Brahim; Le Guiner, Caroline; Moullier, Philippe; Rolling, Fabienne