Target intelligence / Profile preview

Rhodopsin mRNA (mutant) (RHO mRNA (mutant))

Target
RHO mRNA (mutant)
Molecular classification
Other (mRNA)
01

Overview

Mutant RHO mRNA refers to the messenger RNA transcribed from a mutated rhodopsin gene (RHO), which encodes the light-sensitive receptor protein in rod photoreceptor cells of the retina. Mutations in RHO are a major cause of autosomal dominant retinitis pigmentosa, where the pathogenic mRNA leads to production of toxic or dysfunctional rhodopsin. Therapeutic strategies target mutant RHO mRNA to either selectively degrade it or prevent its translation in order to mitigate disease effects[7].

Other names
Mutant rhodopsin mRNAPathogenic RHO mRNA transcriptMutant RHO transcript
02

Mechanism of action

Selective degradation of mutant mRNA (RNA interference, antisense oligonucleotides); RNA editing to correct mutant transcript; Exon skipping (antisense oligonucleotides)

03

Biological functions

Encodes for mutant rhodopsin protein, associated with phototransduction in the retinaPathogenic when containing dominant disease-causing mutations
04

Disease associations

Neurodegenerative disease (primarily inherited retinal degenerations, especially autosomal dominant retinitis pigmentosa)Other (retinal dystrophies)
05

Safety considerations

Off-target effects (unintended suppression of wild-type RHO mRNA could cause further vision loss)Immunogenicity of RNA therapeuticsIncomplete efficacy in eliminating all mutant transcripts
06

Interacting drugs

RNA interference therapeutics (e.g., small interfering RNAs in experimental settings)

2 more in the full profile.

07

Biomarkers

Presence of pathogenic RHO mutations on genetic testingQuantification of mutant RHO mRNA (research/experimental)

Beyond the preview

Go deeper on Rhodopsin mRNA (mutant) (RHO mRNA (mutant)).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Rhodopsin mRNA (mutant) (RHO mRNA (mutant)).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call