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Article

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jul 2007

Zayed Hatem, Xia Lily, Yerich Anton, Yant Stephen R, Kay Mark A, Puttaraju M, McGarrity Gerard J, Wiest David L, McIvor R Scott, Tolar Jakub, Blazar Bruce R

Abstract excerpt

Spliceosome-mediated RNA trans-splicing (SMaRT) is an emerging technology for the repair of defective pre-messenger RNA (pre-mRNA) molecules. It is especially useful in the treatment of genetic disorders involving large genes. Although viral vectors have been used for achieving long-lasting expression of trans-splicing molecules, the immunogenicity and suboptimal safety profiles associated with viral-based...

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Identifiers

PMID: 17457319
DOI: 10.1038/sj.mt.6300178

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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