Ataxin-1 messenger RNA (ATXN1 mRNA)

Ataxin-1 mRNA is the messenger RNA produced from the *ATXN1* gene, which encodes the ataxin-1 protein. The ataxin-1 protein is a polyglutamine-containing nuclear protein involved in transcriptional repression, chromatin binding, and RNA processing, and is particularly implicated in neuronal function. Expanded CAG repeats in the mRNA lead to a longer polyglutamine tract in the translated protein and cause spinocerebellar ataxia type 1 (SCA1), a progressive neurodegenerative disorder characterized by loss of cerebellar Purkinje neurons[7][9]. Ataxin-1 is also implicated in immune function and cancer. The mRNA itself is of therapeutic interest primarily for gene-silencing approaches aiming to lower mutant protein expression in SCA1 and related disorders[7][9].

Other names

ATXN1 mRNASpinocerebellar ataxia type 1 mRNASCA1 mRNAAtx1 mRNA

Antisense oligonucleotide binding followed by RNase H-mediated degradation of ataxin-1 mRNA or translation inhibition. RNA interference (siRNA or shRNA targeting ataxin-1 mRNA). Modulation of splicing to skip mutant exons (hypothetical/mechanistic).