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. 2022 Jul 22;19(1):943–960. doi: 10.1080/15476286.2022.2100971

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© 2022 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.

This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Figure 7. — Small-molecule splicing modifiers can strengthen 5’-splice site (5ʹss) interaction with U1 snRNP through the mechanism of bulge-repair. (a) Chemical structure of Risdiplam, Branaplam, and analogues for the treatment of spinal muscular atrophy (SMA) and Huntington disease. (b) Solution structures of U1 snRNA 5’-end (grey) in complex with the 5ʹss (blue) of SMN exon 7, in absence (left, pdb code: 6HMI) and in presence (right, pdb code: 6HMO) of SMNC5 (yellow) [101]. Within the 5ʹss, the A-1 (red) bulging out in absence of small molecule (left) turns inward when SMN-C5 is present (right). (c) The bulge-repair concept. A weak 5ʹss may be strengthened by small-molecule splicing modifiers acting as a glue between U1 snRNP and the 5ʹss.