Repurposing Oseltamivir Against C<b>A</b>G Repeat Mediated Toxicity in Huntington's Disease and Spinocerebellar Ataxia Using Cellular and <i>Drosophila</i> Model.

ABSTRACT: Huntington's disease (HD) and Spinocerebellar Ataxia (SCA) are debilitating neurological disorders triggered by the expansion of CAG sequences within the specific genes (HTT and ATXN, respectively). These are characterized as poly glutamine (polyQ) disorders, which are marked by widespread neurodegeneration and metabolic irregularities across systemic, cellular, and intracellular levels. This study aimed to identify small molecules that specifically interact with and target the toxic CAG repeat RNA. Here, we investigated the neuroprotective effects of Oseltamivir, an antiviral drug, against the HD and SCA-causing CAG repeats, through biophysical, cellular, and Drosophila model-based studies. Using a multidimensional approach encompassing biophysical techniques,

SUBMITTER: Singh K 

PROVIDER: S-EPMC12019426 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature