Article

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

2026-07-17

Abstract excerpt

<h4>Purpose</h4> Autosomal dominant retinitis pigmentosa caused by mutations in the rhodopsin gene ( RHO -adRP) is among the most prevalent inherited retinal dystrophies. With nearly 100 distinct pathogenic variants identified to date, the mutational heterogeneity of RHO -adRP severely limits the clinical utility of mutation-specific therapeutic strategies. We aimed to develop a mutation-agnostic gene insertion...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 104ce947-af72-559d-8fcd-a5b2d16689eb
DOI: 10.64898/2026.07.13.738123

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Mutation-agnostic gene insertion therapy for RHO-associated autosomal dominant retinitis pigmentosa using zinc finger nucleasesDOI 10.64898/2026.07.13.738123Read

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

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

Retinal organoids mirror CRISPR/Cas9 gene editing efficiency observed in vivoSemantic proximity Read

Gene therapy for inherited retinal diseases: progress and possibilitiesSemantic proximity Read

Efficient workflow for validating homology-independent targeted integration-mediated gene insertion in rod photoreceptor cells to treat dominant-negative mutations causing retinitis pigmentosaSemantic proximity Read

Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa.Semantic proximity Read

Gene-agnostic approaches to treating inherited retinal degenerationsSemantic proximity Read

Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and BeyondSemantic proximity Read

CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.Semantic proximity Read

Rescue of the disease-associated phenotype in CRISPR-corrected hiPSCs as a therapeutic approach for inherited retinal dystrophiesSemantic proximity Read