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October 27, 2021Science Translational MedicineOpen Access

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

Jessica A. Bush Haruo Aikawa Rita Fuerst Yue Li Andrei Ursu Samantha M. Meyer Raphael I. Benhamou Jonathan L. Chen Tanya Khan Sarah Wagner-Griffin Montina J. Van Meter Yuquan Tong Hailey Olafson Kendra McKee Jessica L. Childs‐Disney Tania F. Gendron Yong‐Jie Zhang Alyssa N. Coyne Eric T. Wang Ilyas Yildirim Kye Won Wang Leonard Petrucelli Jeffrey D. Rothstein Matthew D. Disney

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Abstract

is a strategy for mitigating c9ALS/FTD-associated pathologies and studying disease-associated pathways in preclinical models.

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Bush et al. (2021) studied this question.

synapsesocial.com/papers/6a9917ab1c6cd5ba4818f8ac https://doi.org/10.1126/scitranslmed.abd5991

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