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

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model

单次注射靶向 MECP2 的治疗性二价 siRNA 可使 MECP2 重复小鼠模型免于死亡一年。

Hariharan, Vignesh N; Summers, Ashley; Clipperton-Allen, Amy E; Caiazzi, Jillian; Hildebrand, Samuel R; O'Reilly, Daniel; Tang, Qi; Kennedy, Zachary; Echeverria, Dimas; McHugh, Nicholas; Cooper, David; Souza, Jacqueline; Ferguson, Chantal; Bogdanik, Laurent; Coenraads, Monica; Khvorova, Anastasia

Gain-of-function mutations of TRPV4 acting in endothelial cells drive blood-CNS barrier breakdown and motor neuron degeneration in mice

小鼠内皮细胞中TRPV4的功能获得性突变会导致血脑屏障破坏和运动神经元退化。

Sullivan, Jeremy M; Bagnell, Anna M; Alevy, Jonathan; Avila, Elvia Mena; Mihaljević, Ljubica; Saavedra-Rivera, Pamela C; Kong, Lingling; Huh, Jennifer S; McCray, Brett A; Aisenberg, William H; Zuberi, Aamir R; Bogdanik, Laurent; Lutz, Cathleen M; Qiu, Zhaozhu; Quinlan, Katharina A; Searson, Peter C; Sumner, Charlotte J

Clinically relevant mouse models of Charcot-Marie-Tooth type 2S

临床相关的 2S 型 Charcot-Marie-Tooth 小鼠模型

Paige B Martin, Sarah E Holbrook, Amy N Hicks, Timothy J Hines, Laurent P Bogdanik, Robert W Burgess, Gregory A Cox

The integrated stress response contributes to tRNA synthetase-associated peripheral neuropathy

整合应激反应会导致tRNA合成酶相关的周围神经病变。

Spaulding, E L; Hines, T J; Bais, P; Tadenev, A L D; Schneider, R; Jewett, D; Pattavina, B; Pratt, S L; Morelli, K H; Stum, M G; Hill, D P; Gobet, C; Pipis, M; Reilly, M M; Jennings, M J; Horvath, R; Bai, Y; Shy, M E; Alvarez-Castelao, B; Schuman, E M; Bogdanik, L P; Storkebaum, E; Burgess, R W

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

AAV9 介导的 FIG4 递送可延长 Charcot-Marie-Tooth 病 4J 型小鼠模型的寿命

Maximiliano Presa, Rachel M Bailey, Crystal Davis, Tara Murphy, Jenn Cook, Randy Walls, Hannah Wilpan, Laurent Bogdanik, Guy M Lenk, Robert W Burgess, Steven J Gray, Cathleen Lutz

TRPV4 Antagonism Prevents Mechanically Induced Myotonia

TRPV4拮抗剂可预防机械诱发的肌强直

Dupont, Chris; Novak, Kevin; Denman, Kirsten; Myers, Jessica H; Sullivan, Jeremy M; Walker, Phillip V 2nd; Brown, Nicklaus L; Ladle, David R; Bogdanik, Laurent; Lutz, Cathleen M; A Voss, Andrew; Sumner, Charlotte J; Rich, Mark M

C9orf72 is required for proper macrophage and microglial function in mice

C9orf72 是小鼠巨噬细胞和小胶质细胞正常功能所必需的

J G O'Rourke, L Bogdanik, A Yáñez, D Lall, A J Wolf, A K M G Muhammad, R Ho, S Carmona, J P Vit, J Zarrow, K J Kim, S Bell, M B Harms, T M Miller, C A Dangler, D M Underhill, H S Goodridge, C M Lutz, R H Baloh

Effect of genetic background on the dystrophic phenotype in mdx mice

遗传背景对 mdx 小鼠营养不良表型的影响

William D Coley, Laurent Bogdanik, Maria Candida Vila, Qing Yu, Jack H Van Der Meulen, Sree Rayavarapu, James S Novak, Marie Nearing, James L Quinn, Allison Saunders, Connor Dolan, Whitney Andrews, Catherine Lammert, Andrew Austin, Terence A Partridge, Gregory A Cox, Cathleen Lutz, Kanneboyina Nagar

C9orf72 BAC Transgenic Mice Display Typical Pathologic Features of ALS/FTD

C9orf72 BAC 转基因小鼠表现出 ALS/FTD 的典型病理特征

Jacqueline G O'Rourke, Laurent Bogdanik, A K M G Muhammad, Tania F Gendron, Kevin J Kim, Andrew Austin, Janet Cady, Elaine Y Liu, Jonah Zarrow, Sharday Grant, Ritchie Ho, Shaughn Bell, Sharon Carmona, Megan Simpkinson, Deepti Lall, Kathryn Wu, Lillian Daughrity, Dennis W Dickson, Matthew B Harms, Le

Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy

系统性、症状后反义寡核苷酸可挽救 II/III 型脊髓性肌萎缩症小鼠模型中的运动单元成熟延迟

Bogdanik, Laurent P; Osborne, Melissa A; Davis, Crystal; Martin, Whitney P; Austin, Andrew; Rigo, Frank; Bennett, C Frank; Lutz, Cathleen M