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Florian Malard, Antje C Wolter, Julien Marquevielle, Estelle Morvan, Agathe Ecoutin, Simon Rüdisser, Frédéric H.‐T. Allain, Sébastien Campagne
Pharmacological modulation of RNA splicing by small molecules is an emerging facet of drug discovery. In this context, the SMN2 splicing modifier SMN-C5 was used as a prototype to understand the mode of action of small molecule splicing modifiers and propose the concept of 5'-splice site bulge repair. In this study, we combined in vitro binding assays and structure determination by NMR spectroscopy to identify the binding modes of four other small molecule splicing modifiers that switch the splicing of either the SMN2 or the HTT gene. Here, we determined the solution structures of risdiplam, branaplam, SMN-CX and SMN-CY bound to the intermolecular RNA helix epitope containing an unpaired adenine within the G-2A-1G+1U+2 motif of the 5'-splice site. Despite notable differences in their scaffolds, risdiplam, SMN-CX, SMN-CY and branaplam contact the RNA epitope similarly to SMN-C5, suggesting that the 5'-splice site bulge repair mechanism can be generalised. These findings not only deepen our understanding of the chemical diversity of splicing modifiers that target A-1 bulged 5'-splice sites, but also identify common pharmacophores required for modulating 5'-splice site selection with small molecules.
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Malard, F., Wolter, A. C., Marquevielle, J., Morvan, E., Ecoutin, A., Rüdisser, S., Allain, F. H., & Campagne, S. (2024). The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design. Nucleic Acids Research, 52(8), 4124–4136. https://doi.org/10.1093/nar/gkae201
Malard, Florian, et al. "The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design." Nucleic Acids Research, vol. 52, no. 8, 2024, pp. 4124–4136, https://doi.org/10.1093/nar/gkae201.
Malard, Florian, Antje C Wolter, Julien Marquevielle, Estelle Morvan, Agathe Ecoutin, Simon Rüdisser, Frédéric H.‐T. Allain, and Sébastien Campagne. "The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design." Nucleic Acids Research 52, no. 8 (2024): 4124–4136. https://doi.org/10.1093/nar/gkae201.
Malard F., Wolter A. C., Marquevielle J. [и др.] The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design // Nucleic Acids Research. — 2024. — Vol. 52, № 8. — P. 4124–4136. — URL: https://doi.org/10.1093/nar/gkae201
@article{malard2024diversity,
title = {{The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design}},
author = {Florian Malard and Antje C Wolter and Julien Marquevielle and Estelle Morvan and Agathe Ecoutin and Simon Rüdisser and Frédéric H.‐T. Allain and Sébastien Campagne},
journal = {Nucleic Acids Research},
year = {2024},
volume = {52},
number = {8},
pages = {4124--4136},
doi = {10.1093/nar/gkae201},
url = {https://doi.org/10.1093/nar/gkae201}
}TY - JOUR AU - Malard, Florian AU - Wolter, Antje C AU - Marquevielle, Julien AU - Morvan, Estelle AU - Ecoutin, Agathe AU - Rüdisser, Simon AU - Allain, Frédéric H.‐T. AU - Campagne, Sébastien TI - The diversity of splicing modifiers acting on A-1 bulged 5′-splice sites reveals rules for rational drug design JO - Nucleic Acids Research VL - 52 IS - 8 SP - 4124 EP - 4136 PY - 2024 DA - 2024/03/30 DO - 10.1093/nar/gkae201 UR - https://doi.org/10.1093/nar/gkae201 ER -
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