Drosophila Models for Human Diseases

Yumiko Azuma, Ikuko Mizuta, Takahiko Tokuda, Toshiki Mizuno, Azuma, Yumiko, Mizuta, Ikuko, Tokuda, Takahiko, Mizuno, Toshiki

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that affects upper and lower motor neurons in the brain and the spinal cord. Due to the progressive neurodegeneration, ALS leads to paralysis and death caused by respiratory failure 2-5 years after the onset of symptoms. There is no effective cure available. Most ALS cases are sporadic, without family history, whereas 10% of the cases are familial. Identification of variants in more than 30 different loci has provided insight into the pathogenic molecular mechanisms mediating disease pathogenesis. Studies of a Drosophila melanogaster model for each of the ALS genes can contribute to uncovering pathophysiological mechanism of ALS and finding targets of the disease-modifying therapy. In this review, we focus on three ALS-causing genes: TAR DNA-binding protein (TDP-43), fused in sarcoma/translocated in liposarcoma (FUS/TLS), and chromosome 9 open reading frame 72 (C9orf72).