Fused in sarcoma mislocalization is a widespread characteristic of amyotrophic lateral sclerosis

Takeaway

  • Nuclear-to-cytoplasmic mislocalization of unaggregated fused in sarcoma (FUS) RNA binding protein occurs through direct interaction with aberrantly retained intron 9 within the SFPQ transcript and is a widespread feature in amyotrophic lateral sclerosis (ALS).

Why this matters

  • Targeting the nuclear-to-cytoplasmic mislocalization of FUS may prove to be a useful therapeutic strategy in multiple forms of ALS.