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

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

AAVrh10载体可纠正GM1神经节苷脂沉积症动物模型中的病理,并在非人灵长类动物的中枢神经系统中广泛分布。

Hocquemiller, Michaël; Giersch, Laura; Mei, Xin; Gross, Amanda L; Randle, Ashley N; Gray-Edwards, Heather L; Hudson, Judith A; Todeasa, Sophia; Stoica, Lorelei; Martin, Douglas R; Sena-Esteves, Miguel; Aiach, Karen; Laufer, Ralph

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

利用β-半乳糖苷酶反应性磁共振探针,在GM1神经节苷脂病小鼠模型中实时磁共振追踪AAV基因治疗。

Taghian, Toloo; Batista, Ana Rita; Kamper, Sarah; Caldwell, Michael; Lilley, Laura; Li, Hao; Rodriguez, Paola; Mesa, Katerina; Zheng, Shaokuan; King, Robert M; Gounis, Matthew J; Todeasa, Sophia; Maguire, Anne; Martin, Douglas R; Sena-Esteves, Miguel; Meade, Thomas J; Gray-Edwards, Heather L

Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease

系统性输送 AAVB1-GAA 可清除糖原并延长庞贝病小鼠模型的生存期

Allison M Keeler, Marina Zieger, Sophia H Todeasa, Angela L McCall, Jennifer C Gifford, Samantha Birsak, Sourav R Choudhury, Barry J Byrne, Miguel Sena-Esteves, Mai K ElMallah

Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

腺相关病毒传递的人工 microRNA 可延长肌萎缩侧索硬化症小鼠模型的生存期并延缓瘫痪

Lorelei Stoica, Sophia H Todeasa, Gabriela Toro Cabrera, Johnny S Salameh, Mai K ElMallah, Christian Mueller, Robert H Brown Jr, Miguel Sena-Esteves