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。

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

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

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

Nonclinical strategies and considerations to enable the redosing of gene therapies

实现基因治疗重复给药的非临床策略和考量

Moffit, Jeffrey S; Alatsis, Kathila R; Blanset, Diann L; Buss, Nicholas; Rana, Payal

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

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

Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

基因疗法可改善Cln2R207X小鼠模型中与皮质神经元丢失相关的自发性癫痫发作

Takahashi, Keigo; Eultgen, Elizabeth M; Wang, Sophie H; Rensing, Nicholas R; Nelvagal, Hemanth R; Dearborn, Joshua T; Danos, Olivier; Buss, Nicholas; Sands, Mark S; Wong, Michael; Cooper, Jonathan D

MEDI6012: Recombinant Human Lecithin Cholesterol Acyltransferase, High-Density Lipoprotein, and Low-Density Lipoprotein Receptor-Mediated Reverse Cholesterol Transport

MEDI6012:重组人卵磷脂胆固醇酰基转移酶、高密度脂蛋白和低密度脂蛋白受体介导的逆向胆固醇转运

George, Richard T; Abuhatzira, Liron; Stoughton, Susan M; Karathanasis, Sotirios K; She, Dewei; Jin, ChaoYu; Buss, Nicholas A P S; Bakker-Arkema, Rebecca; Ongstad, Emily L; Koren, Michael; Hirshberg, Boaz

Amphibian breeding phenology influences offspring size and response to a common wetland contaminant

两栖动物繁殖物候影响后代大小和对常见湿地污染物的反应

Buss, Nicholas; Swierk, Lindsey; Hua, Jessica

Mistargeting of SH3TC2 away from the recycling endosome causes Charcot-Marie-Tooth disease type 4C

SH3TC2 错误地远离循环内体,导致 4C 型腓骨肌萎缩症

Rhys C Roberts, Andrew A Peden, Folma Buss, Nicholas A Bright, Morwena Latouche, Mary M Reilly, John Kendrick-Jones, J Paul Luzio