Rationally designed small molecules targeting toxic CAG repeat RNA that causes Huntington's disease (HD) and spinocerebellar ataxia (SCAs)

Eshan Khan
Eshan Khan
Subodh Kumar Mishra
Subodh Kumar Mishra
Ribhav Mishra
Ribhav Mishra
Arpita Tawani
Arpita Tawani

Biochimie, Volume 163, 2019, Pages 21-32.

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Drug DesignHuntington’s diseaseRNApoly Q aggregationsmall molecules

摘要

Huntington's diseases (HD) is a very devastating disease caused by r(CAG) expansion in HTT gene, encoding the huntingtin protein. r(CAG) expansion causes disease via multiple pathways including, 1) loss of normal protein function like sequestration of RNA binding protein such as Muscleblind-like (MBNL) and nucleolin, 2) Gain of function f...更多

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