Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

Single-agent, single-target therapeutic approaches are often limited by a complex disease pathobiology. We report rationally designed, multi-target agents for myotonic dystrophy type 1 (DM1). DM1 originates in an abnormal expansion of CTG repeats (CTGexp) in the DMPK gene. The resultant expanded CUG...

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Veröffentlicht in:	Journal of the American Chemical Society 2015-11, Vol.137 (44), p.14180-14189
Hauptverfasser:	Nguyen, Lien, Luu, Long M, Peng, Shaohong, Serrano, Julio F, Chan, H. Y. Edwin, Zimmerman, Steven C
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Disease Models, Animal DNA - genetics DNA - metabolism Drosophila Drug Design HeLa Cells Humans Myotonic Dystrophy - drug therapy Myotonic Dystrophy - genetics Myotonic Dystrophy - pathology RNA - biosynthesis RNA - genetics RNA - metabolism Small Molecule Libraries - chemical synthesis Small Molecule Libraries - chemistry Small Molecule Libraries - pharmacology Trinucleotide Repeat Expansion - drug effects Trinucleotide Repeat Expansion - genetics
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