A Selective Alkylating Agent for CTG Repeats in Myotonic Dystrophy Type 1.
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DOI: 10.1021/acschembio.1c00949
Abstract: Disease intervention at the DNA level generally has been avoided because of off-target effects. Recent advances in genome editing technologies using CRISPR-Cas9 have opened a new era in DNA-targeted therapeutic approaches. However, delivery of such… read more here.
Keywords: ctg repeats; dna; dystrophy type; ctg ... See more keywords