Abstract

<h3>Objective:</h3> To determine the effects of WVE-004 on <i>C9orf72</i> G<sub>4</sub>C<sub>2</sub> repeat-containing transcripts, DPR proteins, and C9orf72 protein levels. <h3>Background:</h3> A large <i>G</i><i><sub>4</sub></i><i>C</i><i><sub>2</sub></i><i> repeat expansion</i> in the <i>C9orf72</i> gene is the...

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