| Autor: |
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 |
| Jazyk: |
angličtina |
| Rok vydání: |
2021 |
| Předmět: |
|
| Zdroj: |
Nature Communications, Vol 12, Iss 1, Pp 1-15 (2021) |
| Druh dokumentu: |
article |
| ISSN: |
2041-1723 |
| DOI: |
10.1038/s41467-021-21112-8 |
| Popis: |
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. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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