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Article

Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2002

Suwanmanee Thipparat, Sierakowska Halina, Fucharoen Suthat, Kole Ryszard

Abstract excerpt

A HeLa cell line stably expressing the human beta-globin gene carrying thalassemic mutations beta(E)/IVS1-6 served as a thalassemia model for repair of aberrant splicing of beta(E)-globin pre-mRNA with antisense oligonucleotides. Treatment of beta(E)/IVS1-6 HeLa cells with a morpholino oligonucleotide targeted immediately upstream of the aberrant 5' splice site activated by the mutations resulted in an increase...

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Source

Identifiers

PMID: 12498768
DOI: 10.1006/mthe.2002.0805

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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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Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder.PMID 12498768Read ↗

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Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.Semantic proximity Read ↗

[Repair of the splicing defect of thalassemic allele beta IVS-2-654 C-->T in cultured human erythroid cells by antisense RNA].Semantic proximity Read ↗

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Double-Target Antisense U7 snRNAs Promote Efficient Skipping of an Aberrant Exon in Three Human -Thalassemic MutationsSemantic proximity Read ↗

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