Article

A mutation-agnostic and allele-specific ASO strategy demonstrates potent functional rescue and retinal preservation in RHO-linked retinitis pigmentosa

2026-09-01

Abstract excerpt

Autosomal dominant retinitis pigmentosa (adRP) caused by RHO mutations is a leading form of inherited retinal degeneration. Extensive allelic heterogeneity of RHO pathogenic variants limits the translational applicability of mutation-specific gene therapies. To address this, we developed SNARE (SNP-guided Silencing of Aberrant RHO Expression), a mutation-independent, allele-specific antisense oligonucleotide (ASO)...

Identifiers and source

Literature Corpus work: 4acb8002-5392-550c-86a2-d461b99f51e3
DOI: 10.64898/2026.08.25.747013

Open publication

Related research

Semantic proximity does not establish scientific evidence. Graph snapshot: 2026-09-29.

Click a neighbor to travelStep 1 · 12 closest

A mutation-agnostic and allele-specific ASO strategy demonstrates potent functional rescue and retinal preservation in RHO-linked retinitis pigmentosaDOI 10.64898/2026.08.25.747013Read

Allele-specific gene editing approach for vision loss restoration in <i>RHO</i> -associated Retinitis PigmentosaSemantic proximity Read

Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa.Semantic proximity Read

Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa.Semantic proximity Read

Allele-specific antisense oligonucleotides for the treatment of BEST1-related dominantly inherited retinal diseases: An in vitro model.Semantic proximity Read

Mutation-agnostic gene insertion therapy for RHO-associated autosomal dominant retinitis pigmentosa using zinc finger nucleasesSemantic proximity Read

Genetic treatment for autosomal dominant inherited retinal dystrophies: approaches, challenges and targeted genotypes.Semantic proximity Read

Applications of antisense oligonucleotides for the treatment of inherited retinal diseases.Semantic proximity Read

RNA therapeutics for retinal diseasesSemantic proximity Read

Allele-specific CRISPR/Cas9 genome editing of the single-base P23H mutation for rhodopsin associated dominant retinitis pigmentosaSemantic proximity Read

Disease modeling and pharmacological rescue of autosomal dominant retinitis pigmentosa associated with RHO copy number variationSemantic proximity Read

Disease modeling and pharmacological rescue of autosomal dominant Retinitis Pigmentosa associated with <i>RHO</i> copy number variationSemantic proximity Read

Alternative Splicing Dysregulation in Retinitis Pigmentosa: Pathogenic Mechanisms and Therapeutic Opportunities.Semantic proximity Read