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Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models.
Bush, Jessica A; Aikawa, Haruo; Fuerst, Rita; Li, Yue; Ursu, Andrei; Meyer, Samantha M; Benhamou, Raphael I; Chen, Jonathan L; Khan, Tanya; Wagner-Griffin, Sarah; Van Meter, Montina J; Tong, Yuquan; Olafson, Hailey; McKee, Kendra K; Childs-Disney, Jessica L; Gendron, Tania F; Zhang, Yongjie; Coyne, Alyssa N; Wang, Eric T; Yildirim, Ilyas; Wang, Kye Won; Petrucelli, Leonard; Rothstein, Jeffrey D; Disney, Matthew D.
Affiliation
  • Bush JA; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Aikawa H; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Fuerst R; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Li Y; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Ursu A; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Meyer SM; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Benhamou RI; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Chen JL; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Khan T; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Wagner-Griffin S; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Van Meter MJ; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Tong Y; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Olafson H; Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • McKee KK; Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Childs-Disney JL; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
  • Gendron TF; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.
  • Zhang Y; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.
  • Coyne AN; Robert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.
  • Wang ET; Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.
  • Yildirim I; Department of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA.
  • Wang KW; Department of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA.
  • Petrucelli L; Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.
  • Rothstein JD; Robert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.
  • Disney MD; Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.
Sci Transl Med ; 13(617): eabd5991, 2021 Oct 27.
Article in En | MEDLINE | ID: mdl-34705518
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Frontotemporal Dementia / Amyotrophic Lateral Sclerosis Type of study: Prognostic_studies Limits: Humans Language: En Journal: Sci Transl Med Year: 2021 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Frontotemporal Dementia / Amyotrophic Lateral Sclerosis Type of study: Prognostic_studies Limits: Humans Language: En Journal: Sci Transl Med Year: 2021 Document type: Article