Huntingtin pre-mRNA (HTT pre-mRNA)

Huntingtin pre-mRNA is the precursor transcript produced from the HTT gene, which encodes the huntingtin protein. This pre-mRNA undergoes extensive processing, including alternative splicing and polyadenylation, to generate mature mRNAs that are translated into either wild-type or mutant huntingtin proteins. Mutant huntingtin pre-mRNA contributes to disease via the production of toxic truncated proteins through aberrant splicing and the formation of nuclear clusters or foci containing mutant mRNAs within neurons. Therapeutic strategies often target mutant HTT pre-mRNA using antisense oligonucleotides or other approaches designed to reduce production or alter processing before translation occurs.

Other names

HTT precursor mRNAHuntingtin primary transcript

Reduction of mutant HTT mRNA levels; modulation of HTT splicing