AUTHOR=Koon Alex C. , Chan Ho Yin Edwin 

TITLE=Drosophila melanogaster As a Model Organism to Study RNA Toxicity of Repeat Expansion-Associated Neurodegenerative and Neuromuscular Diseases

JOURNAL=Frontiers in Cellular Neuroscience

VOLUME=11

YEAR=2017

URL=https://www.frontiersin.org/journals/cellular-neuroscience/articles/10.3389/fncel.2017.00070

DOI=10.3389/fncel.2017.00070

ISSN=1662-5102

ABSTRACT=<p>For nearly a century, the fruit fly, <italic>Drosophila melanogaster</italic>, has proven to be a valuable tool in our understanding of fundamental biological processes, and has empowered our discoveries, particularly in the field of neuroscience. In recent years, <italic>Drosophila</italic> has emerged as a model organism for human neurodegenerative and neuromuscular disorders. In this review, we highlight a number of recent studies that utilized the <italic>Drosophila</italic> model to study repeat-expansion associated diseases (READs), such as polyglutamine diseases, fragile X-associated tremor/ataxia syndrome (FXTAS), myotonic dystrophy type 1 (DM1) and type 2 (DM2), and C9ORF72-associated amyotrophic lateral sclerosis/frontotemporal dementia (C9-ALS/FTD). Discoveries regarding the possible mechanisms of RNA toxicity will be focused here. These studies demonstrate <italic>Drosophila</italic> as an excellent <italic>in vivo</italic> model system that can reveal novel mechanistic insights into human disorders, providing the foundation for translational research and therapeutic development.</p>