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

A systemically deliverable lipid-conjugated siRNA targeting DUX4 as an facioscapulohumeral muscular dystrophy therapeutic.

一种可系统递送的脂质偶联 siRNA,靶向 DUX4,作为面肩肱型肌营养不良症的治疗方法

Daman Katelyn, Yan Jing, Biscans Annabelle, Echeverria Dimas, Shmushkovich Taisia, Wolfson Alexey, Alterman Julia F, Khvorova Anastasia, Emerson Charles P Jr

Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

通过脂质纳米颗粒递送在骨骼肌中高效进行基于Cas9核酸酶的基因编辑

Iyer, Sukanya; Daman, Katelyn; Sun, Yehui; Tutto, Amanda; Holbrook, Sarah E; Joynt, Anya T; Yan, Jing; Ambegaokar, Prajakta; Guo, Dongsheng; Liu, Pengpeng; Stauffer, Jennifer; Maitland, Stacy A; Lee, Sang M; Gallagher, Thomas L; Cox, Gregory A; Keeler, Allison M; Siegwart, Daniel J; Emerson, Charles P; Wolfe, Scot A

Flavones provide resistance to DUX4-induced toxicity via an mTor-independent mechanism

黄酮类化合物通过一种不依赖于mTOR的机制提供对DUX4诱导毒性的抵抗力。

Cohen, Justin; Huang, Shushu; Koczwara, Katherine E; Woods, Kristen T; Ho, Vincent; Woodman, Keryn G; Arbiser, Jack L; Daman, Katelyn; Lek, Monkol; Emerson, Charles P Jr; DeSimone, Alec M

Post-Translational Modifications of the DUX4 Protein Impact Toxic Function in FSHD Cell Models

DUX4蛋白的翻译后修饰影响其在FSHD细胞模型中的毒性功能

Knox, Renatta N; Eidahl, Jocelyn O; Wallace, Lindsay M; Choudury, Sarah G; Rashnonejad, Afrooz; Daman, Katelyn; Guggenbiller, Matthew J; Saad, Nizar Y; Hoover, Michael E; Zhang, Liwen; Branson, Owen E; Emerson, Charles P Jr; Freitas, Michael A; Harper, Scott Q

A human immune/muscle xenograft model of FSHD muscle pathology

FSHD肌肉病理的人类免疫/肌肉异种移植模型

Daman, Katelyn; Yan, Jing; Burzenski, Lisa M; Kady, Jamie; Shultz, Leonard D; Brehm, Michael A; Emerson, Charles P