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

Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease

全身性AAV9基因疗法可减轻CLN1疾病小鼠模型中的神经肌肉接头退化和肌肉萎缩。

Ziółkowska, Ewa A; Jablonka-Shariff, Albina; Williams, Letitia L; Eultgen, Elizabeth M; Wood, Matthew D; Hunter, Daniel A; Sands, Mark S; Snyder-Warwick, Alison K; Cooper, Jonathan D

GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice

GABA能中间神经元与CLN2疾病小鼠的致命性癫痫表型有关。

Takahashi, Keigo; Rensing, Nicholas R; Eultgen, Elizabeth M; Williams, Letitia L; Wang, Sophie H; Nelvagal, Hemanth R; Le, Steven Q; Roberts, Marie S; Doray, Balraj; Han, Edward B; Dickson, Patricia I; Wong, Michael; Sands, Mark S; Cooper, Jonathan D

Gene therapy ameliorates neuromuscular pathology in CLN3 disease

基因疗法可改善CLN3疾病的神经肌肉病理。

Ziółkowska, Ewa A; Jablonka-Shariff, Albina; Williams, Letitia L; Jansen, Matthew J; Wang, Sophie H; Eultgen, Elizabeth M; Wood, Matthew D; Hunter, Daniel A; Sharma, Jaiprakash; Sardiello, Marco; Reese, Robyn; Pestronk, Alan; Sands, Mark S; Snyder-Warwick, Alison K; Cooper, Jonathan D

Limited therapeutic efficacy of N-acetyl-L-leucine in a mouse model of CLN1 disease

N-乙酰-L-亮氨酸在CLN1疾病小鼠模型中的治疗效果有限

Ziółkowska, Ewa A; Pagán Torres, Nicole A; Chen, Hsintsung; Williams, Letitia L; Eultgen, Elizabeth M; Nowacka, Agnieszka; Dearborn, Joshua T; Nelvagal, Hemanth R; Whiteman, Ineka T; Platt, Frances M; Cooper, Jonathan D

Severe white matter astrocytopathy in CADASIL

CADASIL 中的严重白质星形细胞病

Yoshiki Hase, Aiqing Chen, Letitia L Bates, Lucinda J L Craggs, Yumi Yamamoto, Elizabeth Gemmell, Arthur E Oakley, Viktor I Korolchuk, Raj N Kalaria

Establishing a health information workforce: innovation for low- and middle-income countries

建立卫生信息人才队伍:面向中低收入国家的创新

Ledikwe, Jenny H; Reason, Letitia L; Burnett, Sarah M; Busang, Lesego; Bodika, Stephane; Lebelonyane, Refeletswe; Ludick, Steven; Matshediso, Ellah; Mawandia, Shreshth; Mmelesi, Mpho; Sento, Baraedi; Semo, Bazghina-Werq