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

Myonuclear Domain-Associated and Central Nucleation-Dependent Spatial Restriction of Dystrophin Protein Expression

肌核结构域相关和中心核依赖的肌营养不良蛋白表达的空间限制

Chwalenia, Katarzyna; Feng, Vivi-Yun; Hemmer, Nicole; Hildyard, John C W; Roskrow, Liberty E; Piercy, Richard J; Wang, Eric T; Aartsma-Rus, Annemieke; van Putten, Maaike; Wood, Matthew J A; Roberts, Thomas C

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

Muscleblind-like proteins dimerize by forming disulfide bonds to regulate alternative splicing and pathogenic RNA foci formation

肌盲样蛋白通过形成二硫键而二聚化,从而调控选择性剪接和致病性RNA聚集体的形成。

Knudson, Luke A; Kosti, Adam; Moss, Kathryn R; Shi, Liang; Nguyen, GiaLinh N; Janusz-Kaminska, Aleksandra; Zhou, Eric X; Hildebrandt, Ryan P; Wang, Eric T; Bassell, Gary J

CASP8 intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer's disease risk

通过聚甘氨酸-精氨酸病理学鉴定的CASP8内含子扩增会增加阿尔茨海默病风险

Nguyen, Lien; Ajredini, Ramadan; Guo, Shu; Romano, Lisa E L; Tomas, Rodrigo F; Bell, Logan R; Ranum, Paul T; Zu, Tao; Bañez Coronel, Monica; Kelley, Chase P; Redding-Ochoa, Javier; Nizamis, Evangelos; Melloni, Alexandra; Connors, Theresa R; Gaona, Angelica; Thangaraju, Kiruphagaran; Pletnikova, Olga; Clark, H Brent; Davidson, Beverly L; Yachnis, Anthony T; Golde, Todd E; Lou, XiangYang; Wang, Eric T; Renton, Alan E; Goate, Alison; Valdmanis, Paul N; Prokop, Stefan; Troncoso, Juan C; Hyman, Bradley T; Ranum, Laura P W

Interventionally targeting somatic CAG expansions can be a rapid disease-modifying therapeutic avenue: Preclinical evidence.

干预性靶向体细胞 CAG 扩增可能是一种快速改变疾病的治疗途径:临床前证据

Gall-Duncan Terence, Ko Sangyoon Y, Quick Isabelle K, Khan Mahreen, Feng Kristie, Kelley Chase P, Coleman Annabelle, Touze Alexiane, Tang Shuqian, Mehkary Mustafa, Yokoi Katsuyuki, Herrington Casey R, You Justin, Lambie Scott C, Prasolava Tanya K, Panigrahi Gagan B, Park Jeehye, Nakatani Kazuhiko, Byrne Lauren M, Wang Peixiang, Schneekloth John S Jr, Nakamori Masayuki, Frankland Paul W, Wang Eric T, Pearson Christopher E

Choroid plexus mis-splicing and altered cerebrospinal fluid composition in myotonic dystrophy type 1

肌强直性营养不良1型患者的脉络丛剪接异常和脑脊液成分改变

Nutter, Curtis A; Kidd, Benjamin M; Carter, Helmut A; Hamel, Johanna I; Mackie, Philip M; Kumbkarni, Nayha; Davenport, Mackenzie L; Tuyn, Dana M; Gopinath, Adithya; Creigh, Peter D; Sznajder, Łukasz J; Wang, Eric T; Ranum, Laura P W; Khoshbouei, Habibeh; Day, John W; Sampson, Jacinda B; Prokop, Stefan; Swanson, Maurice S

Transcriptome-Wide Studies of RNA-Targeted Small Molecules Provide a Simple and Selective r(CUG)(exp) Degrader in Myotonic Dystrophy

转录组范围内的RNA靶向小分子研究为强直性肌营养不良症提供了一种简单且选择性的r(CUG)(exp)降解剂

Gibaut, Quentin M R; Bush, Jessica A; Tong, Yuquan; Baisden, Jared T; Taghavi, Amirhossein; Olafson, Hailey; Yao, Xiyuan; Childs-Disney, Jessica L; Wang, Eric T; Disney, Matthew D

The myonuclear domain in adult skeletal muscle fibres: past, present and future

成人骨骼肌纤维中的肌核域:过去、现在和未来

Bagley, James R; Denes, Lance T; McCarthy, John J; Wang, Eric T; Murach, Kevin A

A comprehensive atlas of fetal splicing patterns in the brain of adult myotonic dystrophy type 1 patients

成人1型强直性肌营养不良症患者大脑中胎儿剪接模式的综合图谱

Degener, Max J F; van Cruchten, Remco T P; Otero, Brittney A; Wang, Eric T; Wansink, Derick G; 't Hoen, Peter A C