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Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides

In one form of β-thalassemia, a genetic blood disorder, a mutation in intron 2 of the β-globin gene (IVS2-654) causes aberrant splicing of β-globin pre-mRNA and, consequently, β-globin deficiency. Treatment of mammalian cells stably expressing the IVS2-654 human β-globin gene with antisense oligonuc...

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Bibliografske podrobnosti
Main Authors: Sierakowska, Halina, Sambade, Maria J., Agrawal, Sudhir, Kole, Ryszard
Format: Artigo
Jezik:Inglês
Izdano: The National Academy of Sciences of the USA 1996
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC24007/
https://ncbi.nlm.nih.gov/pubmed/8917506
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