Optimization of the Linker Domain in a Dimeric Compound that Degrades an r(CUG) Repeat Expansion in Cells.
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DOI: 10.1021/acs.jmedchem.0c00558
Abstract: RNA repeat expansions are responsible for more than 30 incurable diseases. Among them is myotonic dystrophy type 1 (DM1), the most common form of adult on-set muscular dystrophy. DM1 is caused by an r(CUG) repeat… read more here.
Keywords: expansion; optimization linker; compound; repeat expansion ... See more keywords