Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers

Small molecule splicing modifiers have been previously described that target the general splicing machinery and thus have low specificity for individual genes. Several potent molecules correcting the splicing deficit of the SMN2 (survival of motor neuron 2) gene have been identified and these molecu...

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Published in:Nat Commun
Main Authors: Sivaramakrishnan, Manaswini, McCarthy, Kathleen D., Campagne, Sébastien, Huber, Sylwia, Meier, Sonja, Augustin, Angélique, Heckel, Tobias, Meistermann, Hélène, Hug, Melanie N., Birrer, Pascale, Moursy, Ahmed, Khawaja, Sarah, Schmucki, Roland, Berntenis, Nikos, Giroud, Nicolas, Golling, Sabrina, Tzouros, Manuel, Banfai, Balazs, Duran-Pacheco, Gonzalo, Lamerz, Jens, Hsiu Liu, Ying, Luebbers, Thomas, Ratni, Hasane, Ebeling, Martin, Cléry, Antoine, Paushkin, Sergey, Krainer, Adrian R., Allain, Frédéric H.-T., Metzger, Friedrich
Format: Artigo
Language:Inglês
Published: Nature Publishing Group UK 2017
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5684323/
https://ncbi.nlm.nih.gov/pubmed/29133793
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-017-01559-4
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