Rhodopsin messenger RNA (RHO mRNA) (RHO mRNA)

Rhodopsin (RHO) mRNA is the messenger RNA transcript of the RHO gene, which encodes the rhodopsin protein, a specialized G protein-coupled receptor (GPCR) located in the rod cells of the retina (UniProt P08100). Rhodopsin is critical for visual phototransduction, particularly in low-light conditions, by converting light into electrical signals (PubMed: 29055357). Mutations in RHO mRNA, most notably the P23H point mutation, lead to the synthesis of misfolded proteins that accumulate in the endoplasmic reticulum, causing rod cell apoptosis and resulting in autosomal dominant retinitis pigmentosa (adRP) (NIH: GeneReviews - Retinitis Pigmentosa). Therapeutic strategies targeting RHO mRNA include antisense oligonucleotides (ASOs) like QR-1123, which selectively bind and promote the degradation of mutant transcripts to reduce proteotoxicity (ClinicalTrials.gov: NCT04123626). Other approaches utilize a "knockdown and replace" method, where siRNA or CRISPR/Cas9 targets the endogenous mRNA/DNA while a mutation-proof cDNA is delivered to restore functional protein levels (PubMed: 32673560). These interventions aim to preserve retinal structure and slow the progressive vision loss characteristic of RHO-associated dystrophies.

Other names

Opsin-2 mRNARHO transcriptRhodopsin transcriptRP4 mRNA

Antisense-mediated degradation of mutant mRNA transcripts or gene augmentation to restore wild-type protein levels.