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

Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results

前颗粒蛋白 AAV 基因治疗额颞叶痴呆:转化研究和 1/2 期试验中期结果

Jeffrey Sevigny, Olga Uspenskaya, Laura Dean Heckman, Li Chin Wong, Daniel A Hatch, Ambika Tewari, Rik Vandenberghe, David J Irwin, Dario Saracino, Isabelle Le Ber, Rebekah Ahmed, Jonathan D Rohrer, Adam L Boxer, Sebastian Boland, Patricia Sheehan, Alissa Brandes, Suzanne R Burstein, Benjamin M Shyk

Single-cell RNA-seq reveals alterations in peripheral CX3CR1 and nonclassical monocytes in familial tauopathy

单细胞 RNA 测序揭示家族性 tau 蛋白病中外周 CX3CR1 和非经典单核细胞的改变

Daniel W Sirkis #, Caroline Warly Solsberg #, Taylor P Johnson, Luke W Bonham, Virginia E Sturm, Suzee E Lee, Katherine P Rankin, Howard J Rosen, Adam L Boxer, William W Seeley, Bruce L Miller, Ethan G Geier, Jennifer S Yokoyama2

Detection of emerging neurodegeneration using Bayesian linear mixed-effect modeling

利用贝叶斯线性混合效应模型检测早期神经退行性疾病

Yann Cobigo ,Matthew S Goh ,Amy Wolf ,Adam M Staffaroni ,John Kornak ,Bruce L Miller ,Gil D Rabinovici ,William W Seeley ,Salvatore Spina ,Adam L Boxer ,Bradley F Boeve ,Lei Wang ,Ricardo Allegri ,Marty Farlow ,Hiroshi Mori ,Richard J Perrin ,Joel Kramer ,Howard J Rosen

Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic

利用可穿透血脑屏障的生物制剂挽救由 Grn 功能丧失引起的溶酶体贮积症

Todd Logan ,Matthew J Simon ,Anil Rana ,Gerald M Cherf ,Ankita Srivastava ,Sonnet S Davis ,Ray Lieh Yoon Low ,Chi-Lu Chiu ,Meng Fang ,Fen Huang ,Akhil Bhalla ,Ceyda Llapashtica ,Rachel Prorok ,Michelle E Pizzo ,Meredith E K Calvert ,Elizabeth W Sun ,Jennifer Hsiao-Nakamoto ,Yashas Rajendra ,Katrina W Lexa ,Devendra B Srivastava ,Bettina van Lengerich ,Junhua Wang ,Yaneth Robles-Colmenares ,Do Jin Kim ,Joseph Duque ,Melina Lenser ,Timothy K Earr ,Hoang Nguyen ,Roni Chau ,Buyankhishig Tsogtbaatar ,Ritesh Ravi ,Lukas L Skuja ,Hilda Solanoy ,Howard J Rosen ,Bradley F Boeve ,Adam L Boxer ,Hilary W Heuer ,Mark S Dennis ,Mihalis S Kariolis ,Kathryn M Monroe ,Laralynne Przybyla ,Pascal E Sanchez ,Rene Meisner ,Dolores Diaz ,Kirk R Henne ,Ryan J Watts ,Anastasia G Henry ,Kannan Gunasekaran ,Giuseppe Astarita ,Jung H Suh ,Joseph W Lewcock ,Sarah L DeVos ,Gilbert Di Paolo

Promoting tau secretion and propagation by hyperactive p300/CBP via autophagy-lysosomal pathway in tauopathy

在 tau 病中,过度活跃的 p300/CBP 通过自噬-溶酶体途径促进 tau 的分泌和增殖

Xu Chen, Yaqiao Li, Chao Wang, Yinyan Tang, Sue-Ann Mok, Richard M Tsai, Julio C Rojas, Anna Karydas, Bruce L Miller, Adam L Boxer, Jason E Gestwicki, Michelle Arkin, Ana Maria Cuervo, Li Gan2

A152T tau allele causes neurodegeneration that can be ameliorated in a zebrafish model by autophagy induction

A152T tau 等位基因可导致神经退行性病变,在斑马鱼模型中可通过自噬诱导得到改善

Ana Lopez, Suzee E Lee, Kevin Wojta, Eliana Marisa Ramos, Eric Klein, Jason Chen, Adam L Boxer, Maria Luisa Gorno-Tempini, Daniel H Geschwind, Lars Schlotawa, Nikolay V Ogryzko, Eileen H Bigio, Emily Rogalski, Sandra Weintraub, Marsel M Mesulam; Tauopathy Genetics Consortium; Angeleen Fleming, Giova

Network-driven plasma proteomics expose molecular changes in the Alzheimer's brain

网络驱动的血浆蛋白质组学揭示阿尔茨海默病患者大脑中的分子变化

Philipp A Jaeger, Kurt M Lucin, Markus Britschgi, Badri Vardarajan, Ruo-Pan Huang, Elizabeth D Kirby, Rachelle Abbey, Bradley F Boeve, Adam L Boxer, Lindsay A Farrer, NiCole Finch, Neill R Graff-Radford, Elizabeth Head, Matan Hofree, Ruochun Huang, Hudson Johns, Anna Karydas, David S Knopman, Andrey

Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons

利用 iPSC 衍生的人类神经元中的 C9ORF72 重复扩增来模拟额颞叶痴呆症的关键病理特征

Sandra Almeida, Eduardo Gascon, Hélène Tran, Hsin Jung Chou, Tania F Gendron, Steven Degroot, Andrew R Tapper, Chantal Sellier, Nicolas Charlet-Berguerand, Anna Karydas, William W Seeley, Adam L Boxer, Leonard Petrucelli, Bruce L Miller, Fen-Biao Gao