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Article

Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.

Proceedings of the National Academy of Sciences of the United States of America - 15 Aug 2000

Lacerra G, Sierakowska H, Carestia C, Fucharoen S, Summerton J, Weller D, Kole R

Abstract excerpt

Mononuclear cells from peripheral blood of thalassemic patients were treated with morpholino oligonucleotides antisense to aberrant splice sites in mutant beta-globin precursor mRNAs (pre-mRNAs). The oligonucleotides restored correct splicing and translation of beta-globin mRNA, increasing the hemoglobin (Hb) A synthesis in erythroid cells from patients with IVS2-654/beta(E), IVS2-745/IVS2-745, and...

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Share to beta-Thalassemia
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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Erythroid Precursor Cells

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Source

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Identifiers

PMID: 10944225
DOI: 10.1073/pnas.97.17.9591
PMCID: PMC16909

Provenance

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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Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients.PMID 10944225Read ↗

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