Nuclear-import receptors (NIRs) engage nuclear-localization signals (NLSs) of polypeptides
in the cytoplasm and transport these cargo across the size-selective barrier of the nuclear …

Abstract A G4C2 hexanucleotide repeat expansion in the C9orf72 gene is the most common
genetic cause of ALS and FTLD (C9-ALS/FTLD) with cytoplasmic TDP-43 inclusions …

A repeat expansion in C9orf72 is the major cause of both frontotemporal dementia and
amyotrophic lateral sclerosis, accounting for approximately 1 in 12 cases of either disease …

Disruption of the nuclear pore complex (NPC) and nucleocytoplasmic transport (NCT) have
been implicated in the pathogenesis of neurodegenerative diseases. A GGGGCC …

The devastating effects of the brain losing its ability to control voluntary body movement are
illustrated by diseases such as amyotrophic lateral sclerosis (ALS)-where the nerve cells …

A single guide RNA (sgRNA) directs Cas9 nuclease for gene-specific scission of double-
stranded DNA. High Cas9 activity is essential for efficient gene editing to generate gene …

Hexanucleotide repeat expansions of variable size in C9orf72 are the most prevalent
genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) …

Nucleocytoplasmic transport is the active process of nuclear transport receptors bringing
molecules across the nuclear envelope through nuclear pores. In this work I measured the …

In patients with familial amyotrophic lateral sclerosis (ALS) who carry a mutation in the
C9ORF72 gene, dipeptide-repeat proteins accumulate in nervous system tissue. Of these …

A growing wave of evidence has placed the concept of protein homeostasis at the center of
the pathogenesis of amyotrophic lateral sclerosis (ALS). This is due primarily to the …