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Article

Spliceosome-mediated trans-splicing: the therapeutic cut and paste.

The Journal of investigative dermatology - 1 Aug 2012

Wally Verena, Murauer Eva M, Bauer Johann W

Abstract excerpt

Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of genetic diseases, SMaRT offers several advantages over traditional gene-replacement strategies. SMaRT protocols have recently been used for in vitro phenotypic correction of a variety of genetic disorders, ranging from epidermolysis bullosa to...

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Identifiers

PMID: 22495179
DOI: 10.1038/jid.2012.101

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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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