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Knockdown and replacement therapy mediated by artificial mirtrons in spinocerebellar ataxia 7
We evaluate a knockdown-replacement strategy mediated by mirtrons as an alternative to allele-specific silencing using spinocerebellar ataxia 7 (SCA7) as a model. Mirtrons are introns that form pre-microRNA hairpins after splicing, producing RNAi effectors not processed by Drosha. Mirtron mimics may...
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| Gepubliceerd in: | Nucleic Acids Res |
|---|---|
| Hoofdauteurs: | , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
Oxford University Press
2017
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5569705/ https://ncbi.nlm.nih.gov/pubmed/28575281 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkx483 |
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