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Article

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing.

Nature medicine - 1 Aug 2003

Chao Hengjun, Mansfield S Gary, Bartel Robert C, Hiriyanna Suja, Mitchell Lloyd G, Garcia-Blanco Mariano A, Walsh Christopher E

Abstract excerpt

Conventional gene therapy of hemophilia A relies on the transfer of factor VIII (FVIII; encoded by the F8 gene) cDNA. We carried out spliceosome-mediated RNA trans-splicing (SMaRT) to repair mutant FVIII mRNA. A pre-trans-splicing molecule (PTM) corrected endogenous FVIII mRNA in F8 knockout mice with the hemophilia A phenotype, producing sufficient functional FVIII to correct the hemophilia A phenotype. This is...

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Identifiers

PMID: 12847523
DOI: 10.1038/nm900

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

Source receipt: pubmed, retrieved 7 Sept 2026.

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