The ALS/FTLD-related RNA-binding proteins TDP-43 and FUS have common downstream RNA targets in cortical neurons

FEBS Open Bio. 2013 Nov 20:4:1-10. doi: 10.1016/j.fob.2013.11.001. eCollection 2013.

Abstract

TDP-43 and FUS are linked to amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD), and loss of function of either protein contributes to these neurodegenerative conditions. To elucidate the TDP-43- and FUS-regulated pathophysiological RNA metabolism cascades, we assessed the differential gene expression and alternative splicing profiles related to regulation by either TDP-43 or FUS in primary cortical neurons. These profiles overlapped by >25% with respect to gene expression and >9% with respect to alternative splicing. The shared downstream RNA targets of TDP-43 and FUS may form a common pathway in the neurodegenerative processes of ALS/FTLD.

Keywords: ALS; ALS, amyotrophic lateral sclerosis; Cugbp1, CUG triplet  repeat, RNA-binding protein 1; DAVID, Database for  Annotation, Visualization and Integrated Discovery; FTLD; FTLD, frontotemporal lobar degeneration; FUS; FUS, fused in sarcoma; GFAP, glial fibrillary acidic protein; GO, Gene Ontology; LTP, long-term potentiation; RIN, RNA integrity numbers; RMA, robust multichip average; RRMs, RNA recognition motifs; SBMA, spinal and bulbar muscular atrophy; TDP-43; TDP-43, transactive response (TAR) DNA-binding protein 43; TGF, transforming growth factor; Transcriptome; hnRNAPs, heterogeneous ribonucleoproteins; shCont, shRNA/control; shCugbp1, shRNA/Cugbp1; shFUS, shRNA/FUS; shTDP, shRNA/TDP-43.