Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1

…, LM Luu, AM Baranger, SC Zimmerman

文献索引：Jahromi, Amin Haghighat; Fu, Yuan; Miller, Kali A.; Nguyen, Lien; Luu, Long M.; Baranger, Anne M.; Zimmerman, Steven C. Journal of Medicinal Chemistry, 2013 , vol. 56, # 23 p. 9471 - 9481

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被引用次数: 19

摘要

An expanded CUG repeat transcript (CUGexp) is the causative agent of myotonic dystrophy type 1 (DM1) by sequestering muscleblind-like 1 protein (MBNL1), a regulator of alternative splicing. On the basis of a ligand (1) that was previously reported to be active in an in vitro assay, we present the synthesis of a small library containing 10 dimeric ligands (4–13) that differ in length, composition, and attachment point of the linking chain. The oligoamino ...