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

Anticodon-edited transfer RNAs (ACE-tRNAs) encoded as therapeutic nonviral minimal DNA vectors

反密码子编辑的转移RNA(ACE-tRNA)编码为治疗性非病毒最小DNA载体

Porter, Joseph J; Ko, Wooree; Sorensen, Emily G; Cheung, Zachary; Thrasher, Kari; Couch, Tyler; Gabell, Jeffrey T; Shwe, Victoria; Hyatt, Julia; Licata, Jennasea B; Peterson, Luke K; Dean, David A; Lueck, John D

Therapeutic approaches for nonsense mutations in CFTR

CFTR基因无义突变的治疗方法

Kelly-Aubert, Mairead; Thrasher, Kari; Korostelev, Andrei A; Lueck, John D; Sermet-Gaudelus, Isabelle

G(z)ESTY as an optimized cell-based assay for initial steps in GPCR deorphanization.

G(z)ESTY 是一种优化的基于细胞的检测方法,用于 GPCR 去孤儿化的初始步骤

Franchini Luca, Porter Joseph J, Lueck John D, Orlandi Cesare

Mechanism-based approach in designing patient-specific combination therapies for nonsense mutation diseases

基于机制的方法设计针对无义突变疾病的患者特异性联合疗法

Bhat, Saleem Y; Bhattacharya, Arpan; Li, Hong; Cui, Xiaonan; Lueck, John D; Goldman, Yale E; Cooperman, Barry S

ACE-tRNAs are a platform technology for suppressing nonsense mutations that cause cystic fibrosis.

ACE-tRNA 是一种用于抑制导致囊性纤维化的无义突变的平台技术

Ko Wooree, Porter Joseph J, Spelier Sacha, Sorensen Emily G, Bhatt Priyanka, Gabell Jeffrey T, van der Windt Isabelle, Couch Tyler, Coote Kevin, Mense Martin, Beekman Jeffrey M, Lueck John D

Subnuclear organization and mislocalization of plasmids reduce transgene expression

质粒的亚核组织结构异常和定位错误会降低转基因表达

Gu, Ningyang; Baliga, Uday K; Porter, Joseph J; Lueck, John D; Dean, David A

Elimination of myotonia improves myopathy in a muscleblind knockout model of myotonic dystrophy

在肌强直性肌营养不良症的肌肉盲敲除模型中,消除肌强直可改善肌病。

Sipple, Matthew T; Hamazaki, Sakura A; Todorow, Vanessa; Cisco, Lily A; Lupia, Katherine M; Heil, Christina S; Meinke, Peter; Thornton, Charles A; Lueck, John D

Optimization of ACE-tRNAs function in translation for suppression of nonsense mutations

优化ACE-tRNA在翻译中的功能以抑制无义突变

Porter, Joseph J; Ko, Wooree; Sorensen, Emily G; Lueck, John D

A cystic fibrosis gene editing approach that is on target

一种靶向囊性纤维化的基因编辑方法

Porter, Joseph J; Lueck, John D

Engineered tRNAs efficiently suppress CDKL5 premature termination codons

工程改造的tRNA能有效抑制CDKL5提前终止密码子

Pezzini, Stefano; Mustaccia, Aurora; Aboa, Pierre; Faustini, Giorgia; Branchini, Alessio; Pinotti, Mirko; Frasca, Angelisa; Porter, Joseph J; Lueck, John D; Landsberger, Nicoletta