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Amyotrophic lateral sclerosis (ALS)

Fusion (involved in t(12;16) in malignant liposarcoma) (FUS; TLS)

Cell culture studies suggest oligonucleotides that induce FUS exon seven skipping could help prevent the accumulation of mutant FUS protein in ALS motor neurons. FUS is a DNA- and RNA-binding protein that is frequently mutated in patients with ALS and accumulates in the cytoplasm to become neurotoxic. In vitro and in cultured cells, wild-type FUS autoregulated its own expression by binding to exon seven of its own pre-mRNA transcript and promoting exon skipping and pre-mRNA degradation. In cells expressing mutant FUS variants with defective autoregulation, antisense oligonucleotides targeting a flanking splice site in the FUS pre-mRNA restored exon seven skipping. Next steps include optimizing the oligonucleotides.

SciBX 6(48); doi:10.1038/scibx.2013.1394
Published online Dec. 19, 2013

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Zhou, Y. et al. PLoS Genet.;
published online Oct. 31, 2013;
Contact: Geoffrey G. Hicks, University of Manitoba, Winnipeg, Manitoba, Canada