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

Enteric nervous system degeneration in human and murine CLN3 disease, is ameliorated by gene therapy in mice

人类和小鼠CLN3疾病中的肠神经系统退化,可通过小鼠基因治疗得到缓解。

Ziółkowska, Ewa A; Williams, Letitia L; Jansen, Matthew J; Wang, Sophie H; Eultgen, Elizabeth M; Sharma, Jaiprakash; Sardiello, Marco; Bradley, Rebecca P; Whiteman, Ineka T; Sands, Mark S; Heuckeroth, Robert O; 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

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases

溶酶体贮积症中溶酶体-血小板激活因子的继发性积累

Kell, Pamela; Mishra, Sonali; Gray-Edwards, Heather L; Martin, Douglas R; Gaudioso, Angel; Ledesma, María Dolores; Bongarzone, Ernesto R; Sands, Mark S; Sidransky, Ellen; Ory, Daniel S; Jiang, Xuntian

644. Impact of COVID-19 on Tuberculosis Control and Surveillance: An 11-Year Retrospective Analysis in Belo Horizonte, Brazil

644. COVID-19 对结核病控制和监测的影响:巴西贝洛奥里藏特市 11 年回顾性分析

Ziółkowska, Ewa A; Williams, Letitia L; Jansen, Matthew J; Wang, Sophie H; Eultgen, Elizabeth M; Sharma, Jaiprakash; Sardiello, Marco; Bradley, Rebecca P; Whiteman, Ineka T; Sands, Mark S; Heuckeroth, Robert O; Cooper, Jonathan D; De Vito, Andrea; Ceccarelli, Manuela; Colpani, Agnese; Venanzi Rullo, Emmanuele; Spampinato, Serena; Sofia, Sonia Agata; Pantò, Grazia; Albanese, Antonio; Frasca, maria Chiara; Paternò Raddusa, Michele Salvatore; Campanella, Antonio Edoardo; Santoro, Laura; Russotto, Ylenia; todaro, laura; Coco, Mariagiovanna; Micali, Cristina; Pulvirenti, Sarah; calì, claudia; Di Rosolini, Maria Antonietta; moi, Giulia; conti, giuseppe; Marino, Andrea; Pellicanò, Giovanni Francesco; Iacobello, Carmelo; Montineri, Arturo; Santi Cacopardo, Bruno; angioni, goffredo; Madeddu, Giordano; Nunnari, Giuseppe; Aurélio Angelo, Marco; De Vasconcelos, Débora; Ferreira, Giovana; Bassas, Meritxell; Almeida, Laiz; Ferreira Carvalho, Walisson; Couto, Bráulio R G M

Haploinsufficiency of lysosomal enzyme genes in Alzheimer's disease.

阿尔茨海默病中溶酶体酶基因的单倍体不足

Benitez Bruno A, Wallace Clare E, Patel Maulikkumar, Nykanen Niko-Petteri, Yuede Carla M, Eaton Samantha L, Pottier Cyril, Cetin Arda, Johnson Matthew, Bevan Mia T, Gardiner Woodrow D, Edwards Hannah M, Doherty Brookelyn M, Harrigan Ryan T, Kurian Dominic, Wishart Thomas M, Smith Colin, Cirrito John R, Sands Mark S

Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

基因疗法可改善Cln2R207X小鼠模型中与皮质神经元丢失相关的自发性癫痫发作

Takahashi, Keigo; Eultgen, Elizabeth M; Wang, Sophie H; Rensing, Nicholas R; Nelvagal, Hemanth R; Dearborn, Joshua T; Danos, Olivier; Buss, Nicholas; Sands, Mark S; Wong, Michael; Cooper, Jonathan D

Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases

克拉伯病临床前研究:溶酶体贮积症新型联合疗法的研究模型

Heller, Gregory; Bradbury, Allison M; Sands, Mark S; Bongarzone, Ernesto R

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

酶替代疗法治疗小鼠和绵羊CLN1疾病的跨物种疗效

Nelvagal, Hemanth R; Eaton, Samantha L; Wang, Sophie H; Eultgen, Elizabeth M; Takahashi, Keigo; Le, Steven Q; Nesbitt, Rachel; Dearborn, Joshua T; Siano, Nicholas; Puhl, Ana C; Dickson, Patricia I; Thompson, Gerard; Murdoch, Fraser; Brennan, Paul M; Gray, Mark; Greenhalgh, Stephen N; Tennant, Peter; Gregson, Rachael; Clutton, Eddie; Nixon, James; Proudfoot, Chris; Guido, Stefano; Lillico, Simon G; Whitelaw, C Bruce A; Lu, Jui-Yun; Hofmann, Sandra L; Ekins, Sean; Sands, Mark S; Wishart, Thomas M; Cooper, Jonathan D