Exon-Skipping ASO Reading-Frame Repair
Interactive 3D simulator of antisense-oligonucleotide (ASO) exon-skipping therapy for Duchenne muscular dystrophy: pick a patient's dystrophin deletion genotype, dose an exon-51-skipping ASO, and watch the reading-frame arithmetic decide whether the ribosome builds a truncated functional protein or hits a premature stop.
Duchenne muscular dystrophy is usually caused by a dystrophin gene deletion that throws the ribosome's reading frame out of register, producing a premature stop codon and no protein. This simulator models the real arithmetic behind splice-switching antisense-oligonucleotide therapy (the eteplirsen/exon-51 class of drugs): pick a patient's exon deletion genotype, dose an ASO that forces the spliceosome to additionally skip exon 51, and watch whether the combined length of everything removed lands back on a multiple of three. A 3D ribosome translates the resulting mRNA codon-by-codon, building a polypeptide chain that either runs to completion (frame restored, Becker-like functional protein) or halts at a premature stop (frame still broken, transcript flagged for nonsense-mediated decay) — with a live population strip showing what fraction of transcripts across many molecules are rescued at the current ASO dose.
Pick a Duchenne muscular dystrophy patient's dystrophin deletion genotype, dose an exon-51-skipping antisense oligonucleotide, and watch a 3D ribosome translate the resulting mRNA to see whether the reading-frame arithmetic restores a functional truncated protein or hits a premature stop.
3D · Three.js / WebGL renderer · 60 FPS target · runs fully client-side, no install