title: Drosophila melanogasterAs a Model Organism to Study RNA Toxicity of Repeat Expansion-Associated Neurodegenerative and Neuromuscular Diseases.
abstract: For nearly a century, the fruit fly,Drosophila melanogaster, 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,Drosophilahas emerged as a model organism for human neurodegenerative and neuromuscular disorders. In this review, we highlight a number of recent studies that utilized theDrosophilamodel 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 demonstrateDrosophilaas an excellentin vivomodel system that can reveal novel mechanistic insights into human disorders, providing the foundation for translational research and therapeutic development.
authors.affiliation: Laboratory of Drosophila ResearchHong Kong, Hong Kong; Biochemistry ProgramHong Kong, Hong Kong.
authors.forename: Alex C
authors.initials: AC
authors.lastname: Koon
authors.affiliation: Laboratory of Drosophila ResearchHong Kong, Hong Kong; Biochemistry ProgramHong Kong, Hong Kong; Cell and Molecular Biology ProgramHong Kong, Hong Kong; Molecular Biotechnology Program, Faculty of Science, School of Life SciencesHong Kong, Hong Kong; School of Life Sciences, Gerald Choa Neuroscience Centre, The Chinese University of Hong KongHong Kong, Hong Kong.
authors.forename: Ho Yin Edwin
authors.initials: HY
authors.lastname: Chan
keywords: C9orf72; Huntington's disease; amyotrophic lateral sclerosis (ALS); fragile X; frontotemporal dementia (FTD); myotonic dystrophy; polyglutamine disease; spinocerebellar ataxia