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Article

Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.

15 Sept 1993

Abstract excerpt

Antisense 2'-O-methylribooligonucleotides were targeted against specific sequence elements in mutated human beta-globin pre-mRNAs to restore correct splicing of these RNAs in vitro. The following mutations of the beta-globin gene, A-->G at nt 110 of the first intron (beta 110), T-->G at nt 705 and C-->T at nt 654 of the second intron (IVS2(705) and IVS2(654), respectively), which led to aberrant splicing of the...

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Identifiers

PMID: 8378346
DOI: 10.1073/pnas.90.18.8673

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Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.PMID 8378346Read ↗

Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotidesSemantic proximity Read ↗

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

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

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

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

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

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

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

Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids.Semantic proximity Read ↗

Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.Semantic proximity Read ↗

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