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

AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.

AAV介导的新型微型肌营养不良蛋白基因转移可改善DMD小鼠模型的病理并增强肌肉功能。

期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2026.102901
Owusu Lawrence, Kim SunJung, Patel Hiren, Foltz Steven, Chan Gary N Y, Kim Kwi Hye, Kopen Aaron, Yang Lin, Lawlor Michael W, Buss Nicholas, Cunningham Justine J, Liu Ye, Danos Olivier, Fiscella Michele
Mouse
DMD
发表日期：2026
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Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip.

在hiPSC衍生的视网膜类器官和芯片视网膜中重现CLN2疾病的病理生理，并证明TPP1基因治疗可逆转该疾病

期刊:Cell Reports Medicine
影响因子:10.6
doi:10.1016/j.xcrm.2025.102244
Corti Serena, Kim Kwi Hye, Chen Ting, Botezatu Adelina, Cora Virginia, Ma Ke, Pashkovskaia Natalia, Bernal Vergara Anamaria, Sperlich Denise, Dave Kaushambee, Tolone Arianna, Reddinger Ryan M, Tully Christopher B, Higgins Mikayla, Kleger Alexander, Breunig Markus, Lopatta Paul, Wingerter Svenja, Cipriano Madalena, Bolz Sylvia, Ueffing Marius, Buss Nicholas, Loskill Peter, Liebau Stefan, Achberger Kevin
其它
3D/类器官
发表日期：2025
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Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I.

鞘内或静脉注射最小有效剂量的 AAV9-IDUA/RGX-111 可预防小鼠 MPS I 的心脏、骨骼和神经系统症状

期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101369
Belur Lalitha R, Huber Avery K, Mantone Hillary, Robertson Mason, Smith Miles C, Karlen Andrea D, Kitto Kelley F, Ou Li, Whitley Chester B, Braunlin Elizabeth, Furcich Justin, Lund Troy C, Seelig Davis, Fairbanks Carolyn A, Buss Nicholas, Kim Kwi Hye, McIvor R Scott
神经科学
发表日期：2024
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Genetic modulation of apoptotic pathways fails to alter disease course in tripeptidyl-peptidase 1 deficient mice

对凋亡通路进行基因调控未能改变三肽基肽酶1缺陷小鼠的疾病进程。

期刊:Neuroscience Letters
影响因子:2
doi:10.1016/j.neulet.2009.01.072
Kim, Kwi-Hye; Sleat, David E; Bernard, Ora; Lobel, Peter
信号转导
凋亡
表观遗传
发表日期：2009
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Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis

二肽基肽酶 I 不能在神经退行性疾病——晚期婴儿神经元蜡样脂褐质沉积症中功能性地补偿三肽基肽酶 I 的缺失。

期刊:Biochemical Journal
影响因子:4.3
doi:10.1042/BJ20080411
Kim, Kwi-Hye; Pham, Christine T; Sleat, David E; Lobel, Peter
神经科学
发表日期：2008
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A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

基于CLN2基因靶向破坏的经典晚期婴儿神经元蜡样脂褐质沉积症小鼠模型，会导致三肽基肽酶I活性丧失和进行性神经退行性变。

期刊:Journal of Neuroscience
影响因子:4
doi:10.1523/JNEUROSCI.2729-04.2004
Sleat, David E; Wiseman, Jennifer A; El-Banna, Mukarram; Kim, Kwi-Hye; Mao, Qinwen; Price, Sandy; Macauley, Shannon L; Sidman, Richard L; Shen, Michael M; Zhao, Qi; Passini, Marco A; Davidson, Beverly L; Stewart, Gregory R; Lobel, Peter
神经科学
发表日期：2004
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