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Article

RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture.

Human mutation - 1 Mar 2006

Palfi Arpad, Ader Marius, Kiang Anna-Sophia, Millington-Ward Sophia, Clark Gerry, O'Reilly Mary, McMahon Helena P, Kenna Paul F, Humphries Peter, Farrar G Jane

Abstract excerpt

Extensive mutational heterogeneity presents a significant barrier to the development of therapeutics for RDS-peripherin-linked autosomal-dominant retinitis pigmentosa (RP), for which more than 50 disease-related mutations have been identified to date. Mutation-independent suppression, using RNA interference (RNAi), together with simultaneous expression of a replacement rds gene (r-rds, which has been altered to...

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PMID: 16419083
DOI: 10.1002/humu.20287

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

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