Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
Small molecules correcting the splicing deficit of the survival of motor neuron 2 (SMN2) gene have been identified as having therapeutic potential. Here, the authors provide evidence that SMN2 mRNA forms a ribonucleoprotein complex that can be specifically targeted by these small molecules.
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Nature Portfolio
2017
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oai:doaj.org-article:86ed63c63e664f938352b802e813bfcc2021-12-02T15:39:02ZBinding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers10.1038/s41467-017-01559-42041-1723https://doaj.org/article/86ed63c63e664f938352b802e813bfcc2017-11-01T00:00:00Zhttps://doi.org/10.1038/s41467-017-01559-4https://doaj.org/toc/2041-1723Small molecules correcting the splicing deficit of the survival of motor neuron 2 (SMN2) gene have been identified as having therapeutic potential. Here, the authors provide evidence that SMN2 mRNA forms a ribonucleoprotein complex that can be specifically targeted by these small molecules.Manaswini SivaramakrishnanKathleen D. McCarthySébastien CampagneSylwia HuberSonja MeierAngélique AugustinTobias HeckelHélène MeistermannMelanie N. HugPascale BirrerAhmed MoursySarah KhawajaRoland SchmuckiNikos BerntenisNicolas GiroudSabrina GollingManuel TzourosBalazs BanfaiGonzalo Duran-PachecoJens LamerzYing Hsiu LiuThomas LuebbersHasane RatniMartin EbelingAntoine ClérySergey PaushkinAdrian R. KrainerFrédéric H.-T. AllainFriedrich MetzgerNature PortfolioarticleScienceQENNature Communications, Vol 8, Iss 1, Pp 1-13 (2017) |
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DOAJ |
| collection |
DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q Manaswini Sivaramakrishnan Kathleen D. McCarthy Sébastien Campagne Sylwia Huber Sonja Meier Angélique Augustin Tobias Heckel Hélène Meistermann Melanie N. Hug Pascale Birrer Ahmed Moursy Sarah Khawaja Roland Schmucki Nikos Berntenis Nicolas Giroud Sabrina Golling Manuel Tzouros Balazs Banfai Gonzalo Duran-Pacheco Jens Lamerz Ying Hsiu Liu Thomas Luebbers Hasane Ratni Martin Ebeling Antoine Cléry Sergey Paushkin Adrian R. Krainer Frédéric H.-T. Allain Friedrich Metzger Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| description |
Small molecules correcting the splicing deficit of the survival of motor neuron 2 (SMN2) gene have been identified as having therapeutic potential. Here, the authors provide evidence that SMN2 mRNA forms a ribonucleoprotein complex that can be specifically targeted by these small molecules. |
| format |
article |
| author |
Manaswini Sivaramakrishnan Kathleen D. McCarthy Sébastien Campagne Sylwia Huber Sonja Meier Angélique Augustin Tobias Heckel Hélène Meistermann Melanie N. Hug Pascale Birrer Ahmed Moursy Sarah Khawaja Roland Schmucki Nikos Berntenis Nicolas Giroud Sabrina Golling Manuel Tzouros Balazs Banfai Gonzalo Duran-Pacheco Jens Lamerz Ying Hsiu Liu Thomas Luebbers Hasane Ratni Martin Ebeling Antoine Cléry Sergey Paushkin Adrian R. Krainer Frédéric H.-T. Allain Friedrich Metzger |
| author_facet |
Manaswini Sivaramakrishnan Kathleen D. McCarthy Sébastien Campagne Sylwia Huber Sonja Meier Angélique Augustin Tobias Heckel Hélène Meistermann Melanie N. Hug Pascale Birrer Ahmed Moursy Sarah Khawaja Roland Schmucki Nikos Berntenis Nicolas Giroud Sabrina Golling Manuel Tzouros Balazs Banfai Gonzalo Duran-Pacheco Jens Lamerz Ying Hsiu Liu Thomas Luebbers Hasane Ratni Martin Ebeling Antoine Cléry Sergey Paushkin Adrian R. Krainer Frédéric H.-T. Allain Friedrich Metzger |
| author_sort |
Manaswini Sivaramakrishnan |
| title |
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| title_short |
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| title_full |
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| title_fullStr |
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| title_full_unstemmed |
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers |
| title_sort |
binding to smn2 pre-mrna-protein complex elicits specificity for small molecule splicing modifiers |
| publisher |
Nature Portfolio |
| publishDate |
2017 |
| url |
https://doaj.org/article/86ed63c63e664f938352b802e813bfcc |
| work_keys_str_mv |
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