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

AAV-mediated gene therapy for sialidosis

AAV介导的唾液酸基因治疗

van de Vlekkert, Diantha; Hu, Huimin; Weesner, Jason A; Fremuth, Leigh E; Brown, Scott A; Lu, Meifen; Gomero, Elida; Campos, Yvan; Sheppard, Heather; d'Azzo, Alessandra

Altered GM1 catabolism affects NMDAR-mediated Ca(2+) signaling at ER-PM junctions and increases synaptic spine formation in a GM1-gangliosidosis model.

GM1 分解代谢的改变会影响 ER-PM 连接处的 NMDAR 介导的 Ca(2+) 信号传导,并在 GM1 神经节苷脂沉积症模型中增加突触棘的形成

Weesner Jason A, Annunziata Ida, van de Vlekkert Diantha, Robinson Camenzind G, Campos Yvan, Mishra Ashutosh, Fremuth Leigh E, Gomero Elida, Hu Huimin, d'Azzo Alessandra

AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

AAV介导的基因疗法治疗半乳糖唾液酸缺乏症:一项长期安全性和有效性研究

Hu, Huimin; Mosca, Rosario; Gomero, Elida; van de Vlekkert, Diantha; Campos, Yvan; Fremuth, Leigh E; Brown, Scott A; Weesner, Jason A; Annunziata, Ida; d'Azzo, Alessandra