Rational Design of Ligands Targeting Triplet Repeating Transcripts That Cause RNA Dominant Disease: Application to Myotonic Muscular Dystrophy Type 1 and Spinocerebellar Ataxia Type 3.

Herein, we describe the design of high affinity ligands that bind expanded rCUG and rCAG repeat RNAs expressed in myotonic dystrophy type 1 (DM1) and spinocerebellar ataxia type 3. These ligands also inhibit, with nanomolar IC5o values, the formation of RNA-protein complexes that are implicated in b...

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Publicado en:	Journal of the American Chemical Society Vol. 131; no. 28; pp. 9767 - 9780
Autores principales:	Pushechnikov, Alexei, Lee, Melissa M., Childs-Disney, Jessica L., Sobczak, Krzysztof, French, Jonathan M., Thornton, Charles A., Disney, Matthew D.
Formato:	Artículo
Publicado:	American Chemical Society 7/22/2009
Materias:	Ligands (Chemistry) Coordination compounds Biochemistry Immobilized ligands (Biochemistry) RNA Ribose
Acceso en línea:	Ver este registro en EBSCOhost