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

Generation of an induced pluripotent stem cell line (NCHi023-A) from a 41-year-old male with nemaline myopathy carrying autosomal dominant ACTA1 c.809-10C>A mutation.

从一名患有线状肌病的 41 岁男性(携带常染色体显性 ACTA1 c.809-10C>A 突变)中生成诱导多能干细胞系 (NCHi023-A)

Hanley Meghan, Ye Shiqiao, Zhu Jingting, Yu Yang, Lin Hui, Flanigan Kevin, Rashnonejad Afrooz, Zhao Ming-Tao

Navigating the Landscape of CMT1B: Understanding Genetic Pathways, Disease Models, and Potential Therapeutic Approaches

探索 CMT1B 的全貌:了解遗传通路、疾病模型和潜在治疗方法

McCulloch, Mary Kate; Mehryab, Fatemeh; Rashnonejad, Afrooz

Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b

CRISPR-Cas13b基因疗法治疗FSHD的持续疗效受到Cas13b免疫反应的挑战。

Rashnonejad, Afrooz; Farea, Manal; Amini-Chermahini, Gholamhossein; Coulis, Gerald; Taylor, Noah; Fowler, Allison; Villalta, Armando; King, Oliver D; Harper, Scott Q

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

Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

设计的 U7 snRNA 可抑制 DUX4 表达并改善 DUX4 过表达细胞和 FSHD 患者肌管中的 FSHD 相关结果

Afrooz Rashnonejad, Gholamhossein Amini-Chermahini, Noah K Taylor, Nicolas Wein, Scott Q Harper

Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice

使用重组 AAV9 单次注射进行胎儿基因治疗挽救了小鼠的 SMA 表型

Afrooz Rashnonejad, Gholamhossein Amini Chermahini, Cumhur Gündüz, Hüseyin Onay, Ayça Aykut, Burak Durmaz, Meral Baka, Qin Su, Guangping Gao, Ferda Özkınay

RNAscope in situ hybridization-based method for detecting DUX4 RNA expression in vitro

RNAscope原位杂交法体外检测DUX4 RNA表达

Amini Chermahini, Gholamhossein; Rashnonejad, Afrooz; Harper, Scott Q

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD

AAV 介导的卵泡抑素基因治疗可改善 TIC-DUX4 小鼠 FSHD 模型的功能结果

Carlee R Giesige, Lindsay M Wallace, Kristin N Heller, Jocelyn O Eidahl, Nizar Y Saad, Allison M Fowler, Nettie K Pyne, Mustafa Al-Kharsan, Afrooz Rashnonejad, Gholamhossein Amini Chermahini, Jacqueline S Domire, Diana Mukweyi, Sara E Garwick-Coppens, Susan M Guckes, K John McLaughlin, Kathrin Meyer

Large-Scale Production of Adeno-Associated Viral Vector Serotype-9 Carrying the Human Survival Motor Neuron Gene.

大规模生产携带人类运动神经元存活基因的腺相关病毒载体9型

Rashnonejad Afrooz, Chermahini Gholamhossein Amini, Li Shaoyong, Ozkinay Ferda, Gao Guangping