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
Structural basis of a small molecule targeting RNA for a specific splicing correction.
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')
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
1 mM RNA duplex (13C on adenine riboses of the 5'SS) + 1 mM SMN-C5
Case, Darden, Cheatham III, Simmerling, Wang, Duke, Luo, ... and Kollman
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')
5'-splice site bulge repair, U1 snRNA, Spinal Muscular Atrophy, bulge nucleotide, splicing modifier, RNA
binding site for residues PSU A 5 and PSU A 6