Allele-specific silencing of a dominant SETX mutation in familial amyotrophic lateral sclerosis type 4
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Amyotrophic lateral sclerosis 4 (ALS4) is an autosomal dominant motor neuron disease that is molecularly characterized by reduced R-loop levels and caused by pathogenic variants in senataxin (SETX). SETX encodes an RNA/DNA helicase that resolves three-stranded... ...
