Ataxin-2 mRNA (ATXN2 mRNA)

ATXN2 mRNA encodes **ataxin-2**, a highly conserved RNA-binding protein that regulates mRNA translation, stability, and processing in diverse tissues[1][2][3][4][6]. The mRNA harbors a CAG repeat that—when abnormally expanded—causes diseases such as **spinocerebellar ataxia type 2 (SCA2)** and increases risk for **amyotrophic lateral sclerosis (ALS)**[1][2][6]. Mechanistically, ATXN2 mRNA is translated into ataxin-2, which interacts with poly(A)-binding protein and other splicing factors, modulates mRNA turnover via direct RNA interactions, and participates in cellular processes including stress granule formation and circadian rhythm regulation[1][2][3][4][6]. Therapeutic interest focuses on reducing toxic effects from mutant or expanded ATXN2 mRNA via RNA-targeting strategies, such as antisense oligonucleotide therapy, with ongoing research into safety and disease-modifying efficacy[2][4].

Other names

ataxin-2 transcriptATXN2 transcriptATXN2 gene RNA

Inhibition of mRNA translation or enhancement of mRNA decay (antisense/siRNA approaches) Reduction of toxic ataxin-2 protein production via targeting expanded CAG repeat mRNA