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  • Title: Gene therapy: repairing haemoglobin disorders with ribozymes.
    Author: Weatherall DJ.
    Journal: Curr Biol; 1998 Sep 24; 8(19):R696-8. PubMed ID: 9768357.
    Abstract:
    A ribozyme-mediated approach has made it possible to replace the region in beta globin mRNA containing the sickle-cell-anaemia mutation with a gamma-globin-encoding sequence. This is an interesting new way of correcting monogenic disease, but there are major problems to overcome before it could be applied in the clinic.
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