Article

NMR structures and magnetic force spectroscopy studies of small molecules binding to models of an RNA CAG repeat expansion

2024-08-23

Abstract excerpt

<h4>ABSTRACT</h4> RNA repeat expansions fold into stable structures and cause microsatellite diseases such as Huntington’s disease (HD), myotonic dystrophy type 1 (DM1), and spinocerebellar ataxias (SCAs). The trinucleotide expansion of r(CAG), or r(CAG) exp , causes both HD and SCA3, and the RNA’s toxicity has been traced to its translation into polyglutamine (polyQ; HD) as well as aberrant pre-mRNA alternative...

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Literature Corpus work: a4b45d10-248c-506e-a500-bea9c0c7f3ea
DOI: 10.1101/2024.08.20.608150

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