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

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...

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Bibliographic Details
Main Authors: Giuseppina Lacerra, Halina Sierakowska, Clementina Carestia, Suthat Fucharoen, James Summerton, Dwight Weller, Ryszard Kole
Other Authors: The University of North Carolina at Chapel Hill
Format: Article
Published: 2018
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Online Access:https://repository.li.mahidol.ac.th/handle/123456789/26371
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Institution: Mahidol University