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

Monitoring biological effects of somatic cell genome editing

监测体细胞基因组编辑的生物学效应

Freedman, Benjamin S; Bulte, Jeff W M; Conklin, Bruce R; Judge, Luke M; Dwinell, Melinda R; Geurts, Aron M; Sitton, Madeleine J; Mahajan, Vineet; Kiani, Samira; Gersbach, Charles A; Ebrahimkhani, Mo R; Kelly, John J; Ronald, John A; Morizane, Ryuji; Gupta, Navin; Shakeri-Zadeh, Ali; Vo, Nicole; Saha, Krishanu; Saxena, Shivani; Gamm, David M; Sinha, Divya; Tarantal, Alice F; Vandsburger, Moriel; Matsubara, Azusa; Fu, Hongxia; Tsai, Shengdar Q

Haplotype editing with CRISPR-Cas9 as a therapeutic approach for dominant-negative missense mutations in NEFL

利用 CRISPR-Cas9 进行单倍型编辑作为治疗 NEFL 中显性负性错义突变的治疗方法

Dua, Poorvi H; Simon, Bazilco M J; Marley, Chiara B E; Feliciano, Carissa M; Watry, Hannah L; Cowan, Quinn T; Steury, Dylan; Abraham, Abin; Gilbertson, Erin N; Ramey, Grace D; Capra, John A; Conklin, Bruce R; Judge, Luke M

Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles

利用人类遗传变异,以治疗为目标,靶向数百个具有显性和非显性致病等位基因的基因。

Ramey, Grace D; Cowan, Quinn T; Saxena, Akshita G; Macklin, Bria L; Watry, Hannah L; Mei, Xiaoyue; Dierks, Philip; Judge, Luke M; Conklin, Bruce R; Capra, John A

Functional analysis of a common BAG3 allele associated with protection from heart failure.

对与预防心力衰竭相关的常见 BAG3 等位基因进行功能分析

Perez-Bermejo Juan A, Judge Luke M, Jensen Christina L, Wu Kenneth, Watry Hannah L, Truong Annie, Ho Jaclyn J, Carter Matthew, Runyon Wendy V, Kaake Robyn M, Pulido Ernst H, Mandegar Mohammad A, Swaney Danielle L, So Po-Lin, Krogan Nevan J, Conklin Bruce R

Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq

利用 DISCOVER-Seq 技术在体内进行无偏 CRISPR 脱靶效应检测

Wienert, Beeke; Wyman, Stacia K; Richardson, Christopher D; Yeh, Charles D; Akcakaya, Pinar; Porritt, Michelle J; Morlock, Michaela; Vu, Jonathan T; Kazane, Katelynn R; Watry, Hannah L; Judge, Luke M; Conklin, Bruce R; Maresca, Marcello; Corn, Jacob E

Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins

在表达不同微型化肌营养不良蛋白的 mdx 肌肉中,微管和生理表型的恢复情况各不相同。

Nelson, D'anna M; Lindsay, Angus; Judge, Luke M; Duan, Dongsheng; Chamberlain, Jeffrey S; Lowe, Dawn A; Ervasti, James M

Expression of the dystrophin isoform Dp116 preserves functional muscle mass and extends lifespan without preventing dystrophy in severely dystrophic mice

在重度肌营养不良小鼠中,肌营养不良蛋白亚型Dp116的表达能够维持功能性肌肉质量并延长寿命,但并不能阻止肌营养不良的发生。

Judge, Luke M; Arnett, Andrea L H; Banks, Glen B; Chamberlain, Jeffrey S