Gene therapy: Repairing haemoglobin disorders with ribozymes
نویسنده
چکیده
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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عنوان ژورنال:
- Current Biology
دوره 8 شماره
صفحات -
تاریخ انتشار 1998