The Sense of Targeting Nonsense-Mediated Decay in C9-ALS/FTD

Hana M Odeh; James Shorter

doi:10.1016/j.neuron.2020.03.017

The Sense of Targeting Nonsense-Mediated Decay in C9-ALS/FTD

Neuron. 2020 Apr 8;106(1):6-9. doi: 10.1016/j.neuron.2020.03.017.

Authors

Hana M Odeh¹, James Shorter²

Affiliations

¹ Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
² Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: jshorter@pennmedicine.upenn.edu.

PMID: 32272067
DOI: 10.1016/j.neuron.2020.03.017

Abstract

Defective nucleocytoplasmic transport contributes to C9-ALS/FTD, but an inventory of proteins that become redistributed has remained elusive. In this issue of Neuron, Ortega et al. (2020) catalog these redistributed proteins and pinpoint nonsense-mediated decay as a therapeutic target for C9-ALS/FTD.

Publication types

Comment

MeSH terms

Amyotrophic Lateral Sclerosis*
C9orf72 Protein
Frontotemporal Dementia*
Humans
Proteomics

Substances

C9orf72 Protein
C9orf72 protein, human

Supplementary concepts

Amyotrophic Lateral Sclerosis 2, Juvenile