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Limits of Using Oligonucleotides for Allele-Selective Inhibition at Trinucleotide Repeat Sequences –Targeting the CAG Repeat within Ataxin-1
Trinucleotide repeats are responsible for many genetic diseases. Previous studies have shown that duplex RNAs (dsRNAs) can be used to target expression of a mutant repeat allele while leaving expression of the wild-type allele untouched, creating opportunities for allele-selective inhibition and bet...
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| Publicado no: | Nucleosides Nucleotides Nucleic Acids |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7174099/ https://ncbi.nlm.nih.gov/pubmed/31645175 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/15257770.2019.1671592 |
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