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

Modified Polycyclic Compounds Rescue Mis-splicing in Myotonic Dystrophy Type 1 Disease Models.

修饰的多环化合物可挽救 1 型强直性肌营养不良症疾病模型中的错误剪接。

Frias Jesus A, Hicks Sawyer M, Mazdiyasni Hormoz, Mishra Subodh K, Sarkar Kahini, Yeboah Clara, LeFever Noah M, Scotti Marina M, Zeghal Hana, Brandt Naomi, Vangaveti Sweta, Chakma Pramita, Wang Ting, Reid Tammy S, McMichael Omari, Crumbaugh Christopher, Provenzano Marina, Hale Melissa A, Cleary John D, Johnson Nicholas E, Wang Eric T, Reddy Kaalak, Berglund J Andrew

Metformin improves RAN protein pathology, alternative splicing, and behavioral phenotypes in SCA8 mice.

二甲双胍可改善 SCA8 小鼠的 RAN 蛋白病理、选择性剪接和行为表型。

Romano Lisa El, Tsukagoshi Setsuki, Davey-Osuch Emily E, Ajredini Ramadan, Manasi Kamat, Ortiz Tala Vr, Rijos Eduardo, Bourgon Nathan J, Ames S Elaine, Garrett Timothy J, Cleary John D, Wang Eric T, Ranum Laura Pw

Identification of enzymatically modified isoquercitrin as a therapeutic lead for myotonic dystrophy type 1

酶修饰异槲皮苷作为1型强直性肌营养不良症治疗先导化合物的鉴定

Mishra, Subodh K; Hicks, Sawyer M; Frias, Jesus A; Zhang, Jing; Handley, Mark T; Vangaveti, Sweta; Reid, Tammy S; Pandey, Aradhana; McMichael, Omari; Crumbaugh, Christopher; Provenzano, Marina; Hale, Melissa A; Johnson, Nicholas E; Nakamori, Masayuki; Cleary, John D; Reddy, Kaalak; Berglund, J Andrew

Dysregulation of alternative splicing is a transcriptomic feature of patient-derived fibroblasts from CAG repeat expansion spinocerebellar ataxias

CAG重复序列扩增脊髓小脑性共济失调患者来源成纤维细胞的转录组特征是选择性剪接失调。

Aliyeva, Asmer; Lennon, Claudia D; Cleary, John D; Shorrock, Hannah K; Berglund, J Andrew

Widespread alternative splicing dysregulation occurs presymptomatically in CAG expansion spinocerebellar ataxias

CAG重复序列扩增引起的脊髓小脑性共济失调在症状出现前就已发生广泛的替代剪接失调。

Shorrock, Hannah K; Lennon, Claudia D; Aliyeva, Asmer; Davey, Emily E; DeMeo, Cristina C; Pritchard, Caroline E; Planco, Lori; Velez, Jose M; Mascorro-Huamancaja, Alexandra; Shin, Damian S; Cleary, John D; Berglund, J Andrew

Counting the Cost of Daptomycin Versus Vancomycin in Hospitalized Patients: A Cost Minimization Analysis

达托霉素与万古霉素在住院患者中的成本比较:一项成本最小化分析

Wagner, Jamie L; Jones, Bruce M; Stover, Kayla R; Cleary, John D; Bland, Christopher M; Schipper, Katie E; Chastain, Daniel B; Barber, Katie E

Therapeutic strategies for C9orf72 amyotrophic lateral sclerosis and frontotemporal dementia

C9orf72 肌萎缩侧索硬化症和额颞叶痴呆的治疗策略

Hautbergue, Guillaume M; Cleary, John D; Guo, Shu; Ranum, Laura P W

The potential of engineered eukaryotic RNA-binding proteins as molecular tools and therapeutics

工程化真核RNA结合蛋白作为分子工具和治疗药物的潜力

Shotwell, Carl R; Cleary, John D; Berglund, J Andrew

Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules

利用小分子减轻强直性肌营养不良症中的RNA毒性

Reddy, Kaalak; Jenquin, Jana R; Cleary, John D; Berglund, J Andrew

All in the Family: Repeats and ALS/FTD

家族中的重症:ALS/FTD 的重复发病

Pattamatta, Amrutha; Cleary, John D; Ranum, Laura P W