Expert: Antisense oligonucleotide (ASO) targeting FUS (Fused in Sarcoma) pre-mRNA. Reduces expression of toxic gain-of-function mutant FUS protein in motor neurons. Designed for ALS patients with pathogenic FUS mutations (~3% of familial ALS cases).
Everyday: A genetic medicine that targets the FUS gene — which, when mutated, causes a specific type of ALS (motor neuron disease). It tells the cell to produce less of the toxic mutant FUS protein that kills nerve cells.
Targets: []