Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.
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Nature Publishing Group
2021-02-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-021-21112-8 |
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doaj-c67a2cf526554819ac5be15eaecb07de2021-02-14T12:12:48ZengNature Publishing GroupNature Communications2041-17232021-02-0112111510.1038/s41467-021-21112-8Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical modelsYuanjing Liu0Jean-Cosme Dodart1Helene Tran2Shaunna Berkovitch3Maurine Braun4Michael Byrne5Ann F. Durbin6Xiao Shelley Hu7Naoki Iwamoto8Hyun Gyung Jang9Pachamuthu Kandasamy10Fangjun Liu11Kenneth Longo12Jörg Ruschel13Juili Shelke14Hailin Yang15Yuan Yin16Amy Donner17Zhong Zhong18Chandra Vargeese19Robert H. Brown20Wave Life Sciences Ltd.Wave Life Sciences Ltd.Department of Neurology, University of MassachusettsWave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Wave Life Sciences Ltd.Department of Neurology, University of MassachusettsC9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.https://doi.org/10.1038/s41467-021-21112-8 |
| collection |
DOAJ |
| language |
English |
| format |
Article |
| sources |
DOAJ |
| author |
Yuanjing Liu Jean-Cosme Dodart Helene Tran Shaunna Berkovitch Maurine Braun Michael Byrne Ann F. Durbin Xiao Shelley Hu Naoki Iwamoto Hyun Gyung Jang Pachamuthu Kandasamy Fangjun Liu Kenneth Longo Jörg Ruschel Juili Shelke Hailin Yang Yuan Yin Amy Donner Zhong Zhong Chandra Vargeese Robert H. Brown |
| spellingShingle |
Yuanjing Liu Jean-Cosme Dodart Helene Tran Shaunna Berkovitch Maurine Braun Michael Byrne Ann F. Durbin Xiao Shelley Hu Naoki Iwamoto Hyun Gyung Jang Pachamuthu Kandasamy Fangjun Liu Kenneth Longo Jörg Ruschel Juili Shelke Hailin Yang Yuan Yin Amy Donner Zhong Zhong Chandra Vargeese Robert H. Brown Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models Nature Communications |
| author_facet |
Yuanjing Liu Jean-Cosme Dodart Helene Tran Shaunna Berkovitch Maurine Braun Michael Byrne Ann F. Durbin Xiao Shelley Hu Naoki Iwamoto Hyun Gyung Jang Pachamuthu Kandasamy Fangjun Liu Kenneth Longo Jörg Ruschel Juili Shelke Hailin Yang Yuan Yin Amy Donner Zhong Zhong Chandra Vargeese Robert H. Brown |
| author_sort |
Yuanjing Liu |
| title |
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models |
| title_short |
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models |
| title_full |
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models |
| title_fullStr |
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models |
| title_full_unstemmed |
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models |
| title_sort |
variant-selective stereopure oligonucleotides protect against pathologies associated with c9orf72-repeat expansion in preclinical models |
| publisher |
Nature Publishing Group |
| series |
Nature Communications |
| issn |
2041-1723 |
| publishDate |
2021-02-01 |
| description |
C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models. |
| url |
https://doi.org/10.1038/s41467-021-21112-8 |
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