Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G<sub>4</sub>C<sub>2</sub>) repeat expansion in vitro and in vivo ALS models.

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作者： Jessica A,Bush ^[1] ; Haruo,Aikawa ^[1] ; Rita,Fuerst ^[1] ; Yue,Li ^[1] ; Andrei,Ursu ^[1] ; Samantha M,Meyer ^[1] ; Raphael I,Benhamou ^[1] ; Jonathan L,Chen ^[1] ; Tanya,Khan ^[1] ; Sarah,Wagner-Griffin ^[1] ; Montina J,Van Meter ^[1] ; Yuquan,Tong ^[1] ; Hailey,Olafson ^[2] ; Kendra K,McKee ^[2] ; Jessica L,Childs-Disney ^[1] ; Tania F,Gendron ^[3] ; Yongjie,Zhang ^[3] ; Alyssa N,Coyne ^[4] ; Eric T,Wang ^[2] ; Ilyas,Yildirim ^[5] ; Kye Won,Wang ^[5] ; Leonard,Petrucelli ^[3] ; Jeffrey D,Rothstein ^[4] ; Matthew D,Disney ^[1]

作者单位： Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA. ^[1] Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA. ^[2] Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA. ^[3] Robert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA. ^[4] Department of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA. ^[5]

主题词肌萎缩侧索硬化(Amyotrophic Lateral Sclerosis);DNA重复序列扩增(DNA Repeat Expansion);额颞叶痴呆(Frontotemporal Dementia);人类(Humans);核糖核酸酶类(Ribonucleases)

DOI 10.1126/scitranslmed.abd5991

PMID 34705518

发布时间 2022-10-28

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