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Possibilities and challenges of small molecule organic compounds for the treatment of repeat diseases
The instability of repeat sequences in the human genome results in the onset of many neurological diseases if the repeats expand above a certain threshold. The transcripts containing long repeats sequester RNA binding proteins. The mechanism of repeat instability involves metastable slip-out hairpin...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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The Japan Academy
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8795530/ https://www.ncbi.nlm.nih.gov/pubmed/35013029 http://dx.doi.org/10.2183/pjab.98.003 |