A Small Molecule that Binds an RNA Repeat Expansion Stimulates Its Decay via the Exosome Complex

Author:

Angelbello Alicia J.,Benhamou Raphael I.,Rzuczek Suzanne G.,Choudhary Shruti,Tang Zhenzhi,Chen Jonathan L.,Roy Madhuparna,Wang Kye Won,Yildirim Ilyas,Jun Albert S.,Thornton Charles A.,Disney Matthew D.

Funder

Myotonic Dystrophy Foundation

National Institutes of Health

National Ataxia Foundation

Muscular Dystrophy Association

Publisher

Elsevier BV

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology,Molecular Biology,Molecular Medicine,Biochemistry

Reference64 articles.

1. Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model;Angelbello;Proc. Natl. Acad. Sci. U S A,2019

2. Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds;Arandel;Dis. Models Mech.,2017

3. A toxic RNA catalyzes the cellular synthesis of its own inhibitor, shunting it to endogenous decay pathways;Benhamou;Cell Chem. Biol.,2020

4. The protein data bank;Berman;Nucleic Acids Res.,2000

5. Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member;Brook;Cell,1992

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