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

Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease

新型启动子驱动治疗性转基因表达，并在庞贝病小鼠模型中逃避转基因特异性免疫反应

期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2025.109717
Sun, Baodong; Yi, Haiqing; Han, Sang-Oh; Li, Songtao; Eisner, William; Brooks, Elizabeth D; Bacon, Rebecca; Koeberl, Dwight D
免疫/内分泌
发表日期：2026
文献求助 收藏

High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa

高活性MyoAAV衣壳增强了GSD IIIa小鼠模型的骨骼肌矫正效果

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101567
Liao, Kuo-An; Han, Sang-Oh; Barzi, Mercedes; Yi, Haiqing; Eisner, William; Bissig-Choisat, Beatrice; Bissig, Karl-Dimiter; Sun, Baodong
发表日期：2025
文献求助 收藏

Phase I study of liver depot gene therapy in late-onset Pompe disease.

针对晚发型庞贝氏症的肝脏基因治疗的I期研究

期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2023.02.014
Smith Edward C, Hopkins Sam, Case Laura E, Xu Ming, Walters Crista, Dearmey Stephanie, Han Sang-Oh, Spears Tracy G, Chichester Jessica A, Bossen Edward H, Hornik Christoph P, Cohen Jennifer L, Bali Deeksha, Kishnani Priya S, Koeberl Dwight D
基因治疗
发表日期：2023
文献求助 收藏

Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease

在患有庞贝氏病的雌性小鼠中，有效的雄激素激素给药联合腺相关病毒载体介导的基因治疗

期刊:Human Gene Therapy
影响因子:4
doi:10.1089/hum.2021.218
Han, Sang-Oh; Gheorghiu, Dorothy; Chang, Alex; Mapatano, Sweet Hope; Li, Songtao; Brooks, Elizabeth; Koeberl, Dwight
基因治疗
微生物学
免疫/内分泌
发表日期：2022
文献求助 收藏

Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

幼鼠和成年小鼠的比较揭示了庞贝氏症肝脏沉积基因治疗的年龄效应

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2019.11.020
Han, Sang-Oh; Li, Songtao; McCall, Angela; Arnson, Benjamin; Everitt, Jeffrey I; Zhang, Haoyue; Young, Sarah P; ElMallah, Mai K; Koeberl, Dwight D
基因治疗
发表日期：2020
文献求助 收藏

Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease

在患有庞贝病的小鼠中评估抗高血压药物联合酶替代疗法的疗效

期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2019.10.005
Han, Sang-Oh; Haynes, Alexina C; Li, Songtao; Abraham, Dennis M; Kishnani, Priya S; Steet, Richard; Koeberl, Dwight D
心血管
高血压
发表日期：2020
文献求助 收藏

Erratum: Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

更正：低剂量肝脏靶向基因疗法通过诱导免疫耐受增强庞贝病酶替代疗法的疗效

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2019.04.005
Han, Sang-Oh; Ronzitti, Giuseppe; Arnson, Benjamin; Leborgne, Christian; Li, Songtao; Mingozzi, Federico; Koeberl, Dwight
免疫/内分泌
发表日期：2019
文献求助 收藏

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

低剂量肝脏靶向基因疗法通过诱导免疫耐受增强庞贝病酶替代疗法的疗效

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2016.12.010
Han, Sang-Oh; Ronzitti, Giuseppe; Arnson, Benjamin; Leborgne, Christian; Li, Songtao; Mingozzi, Federico; Koeberl, Dwight
免疫/内分泌
发表日期：2017
文献求助 收藏

A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease

β受体阻滞剂普萘洛尔会降低庞贝氏症酶替代疗法的疗效。

期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2015.09.012
Han, Sang-Oh; Pope, Rand; Li, Songtao; Kishnani, Priya S; Steet, Richard; Koeberl, Dwight D
信号转导
发表日期：2016
文献求助 收藏

Salmeterol enhances the cardiac response to gene therapy in Pompe disease

沙美特罗可增强庞贝氏症基因治疗的心脏反应

期刊:Molecular Genetics and Metabolism
影响因子:3.5
doi:10.1016/j.ymgme.2016.03.006
Han, Sang-Oh; Li, Songtao; Koeberl, Dwight D
基因治疗
发表日期：2016
文献求助 收藏