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Article

Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides

12 Nov 1996

Abstract excerpt

In one form of beta-thalassemia, a genetic blood disorder, a mutation in intron 2 of the beta-globin gene (IVS2-654) causes aberrant splicing of beta-globin pre-mRNA and, consequently, beta-globin deficiency. Treatment of mammalian cells stably expressing the IVS2-654 human beta-globin gene with antisense oligonucleotides targeted at the aberrant splice sites restored correct splicing in a dose-dependent fashion,...

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PMID: 8917506
DOI: 10.1073/pnas.93.23.12840

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Related research

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Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotidesPMID 8917506Read ↗

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

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.Semantic proximity Read ↗

Reversal of aberrant splicing of beta-thalassemia allele by antisense RNA in vitro and in vivo.Semantic proximity Read ↗

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

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

Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.Semantic proximity Read ↗

Restoration of correct splicing of thalassemic beta-globin pre-mRNA by modified U1 snRNAs.Semantic proximity Read ↗

Correction of aberrant pre-mRNA splicing by antisense oligonucleotides in beta-thalassemia Egyptian patients with IVSI-110 mutation.Semantic proximity Read ↗

High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA.Semantic proximity Read ↗

Amelioration of beta654-thalassemia in mouse model with the knockdown of aberrantly spliced beta-globin mRNA.Semantic proximity Read ↗

Efficient and specific repair of sickle beta-globin RNA by trans-splicing ribozymes.Semantic proximity Read ↗

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