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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Hlavní autoři: Lacerra, Giuseppina, Sierakowska, Halina, Carestia, Clementina, Fucharoen, Suthat, Summerton, James, Weller, Dwight, Kole, Ryszard
Médium: Artigo
Jazyk:Inglês
Vydáno: The National Academy of Sciences 2000
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC16909/
https://ncbi.nlm.nih.gov/pubmed/10944225
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