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