Why some RNA drugs work better than others
MPSAs reveal IUPAC motifs for the 5’ss specificities of risdiplam and branaplam. A, B Structures of (A) risdiplam and (B) branaplam. C MPSA performed in the context of a minigene library spanning exons 6, 7, and 8 of SMN2, In this library, the 5’ss of exon 20 was replaced by 285 variant 5’ss sequences of …