Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G 4 C 2 ) repeat expansion in vitro and in vivo ALS models-Reference-Cited by-同舟云学术

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

Published:2021-10-27 Issue:617 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Bush Jessica A.¹^ORCID,Aikawa Haruo¹^ORCID,Fuerst Rita¹,Li Yue¹,Ursu Andrei¹,Meyer Samantha M.¹^ORCID,Benhamou Raphael I.¹^ORCID,Chen Jonathan L.¹^ORCID,Khan Tanya¹^ORCID,Wagner-Griffin Sarah¹^ORCID,Van Meter Montina J.¹^ORCID,Tong Yuquan¹^ORCID,Olafson Hailey²,McKee Kendra K.²^ORCID,Childs-Disney Jessica L.¹,Gendron Tania F.³^ORCID,Zhang Yongjie³^ORCID,Coyne Alyssa N.⁴^ORCID,Wang Eric T.²^ORCID,Yildirim Ilyas⁵,Wang Kye Won⁵^ORCID,Petrucelli Leonard³^ORCID,Rothstein Jeffrey D.⁴^ORCID,Disney Matthew D.¹^ORCID

Affiliation:

1. Department of Chemistry, Scripps Research Institute, 130 Scripps Way, Jupiter, FL 33458, USA.

2. Center for NeuroGenetics, College of Medicine, University of Florida, Gainesville, FL 32610, USA.

3. Department of Neuroscience, Mayo Clinic, 4500 San Pablo Road, Jacksonville, FL 32224, USA.

4. Robert Packard Center for ALS Research, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA.

5. Department of Chemistry and Biochemistry, Florida Atlantic University, Jupiter, FL 33458, USA.

Abstract

Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference81 articles.

1. Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

2. A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

3. Large C9orf72 Hexanucleotide Repeat Expansions Are Seen in Multiple Neurodegenerative Syndromes and Are More Frequent Than Expected in the UK Population

4. Association between repeat sizes and clinical and pathological characteristics in carriers of C9ORF72 repeat expansions (Xpansize-72): a cross-sectional cohort study

5. Unconventional Translation of C9ORF72 GGGGCC Expansion Generates Insoluble Polypeptides Specific to c9FTD/ALS

Cited by 47 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Targeting RNA with small molecules, from RNA structures to precision medicines: IUPHAR review: 40;British Journal of Pharmacology;2024-09-03

2. RNAs in the diagnosis and treatment of major diseases;Chinese Science Bulletin;2024-08-01

3. Transcriptome-Wide, Unbiased Profiling of Ribonuclease Targeting Chimeras;Journal of the American Chemical Society;2024-07-24

4. Small Molecules That Degrade RNA;Methods and Principles in Medicinal Chemistry;2024-07-05

5. Screening and Lead Generation Techniques for RNA Binders;Methods and Principles in Medicinal Chemistry;2024-07-05