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Possibilities and challenges of small molecule organic compounds for the treatment of repeat diseases

10 Jan 2022

Abstract excerpt

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 DNA structures. Synthetic organic chemists have focused on the development of small organic molecules targeting...

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PMID: 35013029
DOI: 10.2183/pjab.98.003

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