AUTHOR=Butti Zoe , Patten Shunmoogum A. 

TITLE=RNA Dysregulation in Amyotrophic Lateral Sclerosis

JOURNAL=Frontiers in Genetics

VOLUME=9

YEAR=2019

URL=https://www.frontiersin.org/journals/genetics/articles/10.3389/fgene.2018.00712

DOI=10.3389/fgene.2018.00712

ISSN=1664-8021

ABSTRACT=<p>Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease and is characterized by the degeneration of upper and lower motor neurons. It has become increasingly clear that RNA dysregulation is a key contributor to ALS pathogenesis. The major ALS genes <italic>SOD1, TARDBP, FUS</italic>, and <italic>C9orf72</italic> are involved in aspects of RNA metabolism processes such as mRNA transcription, alternative splicing, RNA transport, mRNA stabilization, and miRNA biogenesis. In this review, we highlight the current understanding of RNA dysregulation in ALS pathogenesis involving these major ALS genes and discuss the potential of therapeutic strategies targeting disease RNAs for treating ALS.</p>