Solution structure of the RNA duplex formed by the 5'-end of U1snRNA and the 5'-splice site of SMN2 exon7 in complex with the SMN-C5 splicing modifier

RNA (5'-R(*AP*UP*AP*CP*(PSU)P*(PSU)P*AP*CP*CP*UP*G)-3'), RNA (5'-R(*GP*GP*AP*GP*UP*AP*AP*GP*UP*CP*U)-3')

[(2R,3S,4R,5S)-5-(2,4-dioxo-1H-pyrimidin-5-yl)-3,4-dihydroxy-oxolan-2-yl]methyl dihydrogen phosphate

Structural basis of a small molecule targeting RNA for a specific splicing correction.

10 mM deuterated MES, 50 mM unlabelled sodium chloride, 6 % deuterated glycerol, 100% D2O

10 mM deuterated MES, 50 mM unlabled sodium chloride, 6 % deuterated glycerol, 100% D2O

Case, Darden, Cheatham, III, Simmerling, Wang, Duke, Luo, ... and Kollman