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Article

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

Proceedings of the National Academy of Sciences of the United States of America - 27 Jan 2009

Svasti Saovaros, Suwanmanee Thipparat, Fucharoen Suthat, Moulton Hong M, Nelson Michelle H, Maeda Nobuyo, Smithies Oliver, Kole Ryszard

Abstract excerpt

Repair of beta-globin pre-mRNA rendered defective by a thalassemia-causing splicing mutation, IVS2-654, in intron 2 of the human beta-globin gene was accomplished in vivo in a mouse model of IVS2-654 thalassemia. This was effected by a systemically delivered splice-switching oligonucleotide (SSO), a morpholino oligomer conjugated to an arginine-rich peptide. The SSO blocked the aberrant splice site in the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 19164558
DOI: 10.1073/pnas.0812436106
PMCID: PMC2633555

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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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