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Article

Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2015

Berger Adeline, Lorain Stéphanie, Joséphine Charlène, Desrosiers Melissa, Peccate Cécile, Voit Thomas, Garcia Luis, Sahel José-Alain, Bemelmans Alexis-Pierre

Abstract excerpt

The promising clinical results obtained for ocular gene therapy in recent years have paved the way for gene supplementation to treat recessively inherited forms of retinal degeneration. The situation is more complex for dominant mutations, as the toxic mutant gene product must be removed. We used spliceosome-mediated RNA trans-splicing as a strategy for repairing the transcript of the rhodopsin gene, the gene...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 25619725
DOI: 10.1038/mt.2015.11
PMCID: PMC4427870

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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