Dystrophin pre-mRNA exon 23

Dystrophin pre-mRNA exon 23 refers to a specific RNA sequence element and is not considered a therapeutic target in the conventional sense, as it is not a molecular receptor, enzyme, transporter, or other drug-binding protein. Instead, it is the subject of therapeutic intervention, particularly in the context of Duchenne muscular dystrophy (DMD) research. In the *mdx* mouse model, this exon contains a nonsense mutation that causes premature translation termination. Therapeutic strategies, such as antisense oligonucleotide (AO) and CRISPR-based genome editing, aim to induce 'exon skipping' – the removal of this mutated exon during RNA processing. This process maintains the reading frame, allowing the production of a shorter, but partially functional, Becker-dystrophin-like protein. The actual therapeutic targets in these studies are the splicing machinery, regulatory elements that process the RNA, or the genomic DNA encoding exon 23, which are modulated by various therapeutic agents.

Other names

exon 23 of the dystrophin pre-mRNA

Not applicable, as Dystrophin pre-mRNA exon 23 is not a direct therapeutic target. It is the subject of therapeutic interventions aimed at modulating its processing (e.g., exon skipping) rather than being directly bound or acted upon by drugs in a conventional target-drug interaction.