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 Portfolio
2021
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oai:doaj.org-article:c67a2cf526554819ac5be15eaecb07de2021-12-02T14:26:59ZVariant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models10.1038/s41467-021-21112-82041-1723https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de2021-02-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-21112-8https://doaj.org/toc/2041-1723C9orf72 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.Yuanjing LiuJean-Cosme DodartHelene TranShaunna BerkovitchMaurine BraunMichael ByrneAnn F. DurbinXiao Shelley HuNaoki IwamotoHyun Gyung JangPachamuthu KandasamyFangjun LiuKenneth LongoJörg RuschelJuili ShelkeHailin YangYuan YinAmy DonnerZhong ZhongChandra VargeeseRobert H. BrownNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-15 (2021) |
| institution |
DOAJ |
| collection |
DOAJ |
| language |
EN |
| topic |
Science Q |
| spellingShingle |
Science Q 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 |
| 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. |
| format |
article |
| 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 |
| 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 Portfolio |
| publishDate |
2021 |
| url |
https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de |
| work_keys_str_mv |
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