Publication: Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients
Issued Date
2000-08-15
Resource Type
ISSN
00278424
Other identifier(s)
2-s2.0-0034662881
Rights
Mahidol University
Rights Holder(s)
SCOPUS
Bibliographic Citation
Proceedings of the National Academy of Sciences of the United States of America. Vol.97, No.17 (2000), 9591-9596
Suggested Citation
Giuseppina Lacerra, Halina Sierakowska, Clementina Carestia, Suthat Fucharoen, James Summerton, Dwight Weller, Ryszard Kole 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. Vol.97, No.17 (2000), 9591-9596. doi:10.1073/pnas.97.17.9591 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/26371
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Title
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients
Abstract
Mononuclear cells from peripheral blood of thalassemic patients were treated with morpholino oligonucleotides antisense to aberrant splice sites in mutant β-globin precursor mRNAs (pre- mRNAs). The oligonucleotides restored correct splicing and translation of β-globin mRNA, increasing the hemoglobin (Hb) A synthesis in erythroid cells from patients with IVS2-654/β(E), IVS2-745/IVS2-745, and IVS2-745/IVS2-1 genotypes. The maximal Hb A level for repaired IVS2-745 mutation was ≃30% of normal; Hb A was still detectable 9 days after a single treatment with oligonucleotide. Thus, expression of defective β-globin genes was repaired and significant level of Hb A was restored in a cell population that would be targeted in clinical applications of this approach.