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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Auteurs principaux: | , , , , , , , , , , , , , , , , , , , , |
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Format: | article |
Langue: | EN |
Publié: |
Nature Portfolio
2021
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Accès en ligne: | https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de |
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