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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Main Authors: | , , , , , , |
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Other Authors: | |
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 |