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

Liver-directed AAV gene therapy normalizes disease symptoms and provides cross-correction in a model of lysosomal acid lipase deficiency.

肝脏靶向 AAV 基因疗法可使疾病症状正常化,并在溶酶体酸性脂肪酶缺乏症模型中提供交叉纠正

Lam Patricia, Zygmunt Deborah A, Ashbrook Anna, Yan Cong, Du Hong, Martin Paul T

Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice.

双重 FKRP/FST 基因疗法可使 LGMDR9 小鼠恢复正常行走能力,增强力量,减少病理变化,并增强基因表达

Lam Patricia, Zygmunt Deborah A, Ashbrook Anna, Bennett Macey, Vetter Tatyana A, Martin Paul T

rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice

rAAVrh74.MCK.GALGT2 在 C57BL/6J 小鼠静脉注射后显示出安全性和广泛的肌肉糖基化作用

Zygmunt, Deborah A; Xu, Rui; Jia, Ying; Ashbrook, Anna; Menke, Chelsea; Shao, Guohong; Yoon, Jung Hae; Hamilton, Sonia; Pisharath, Harshan; Bolon, Brad; Martin, Paul T