Gene therapy: repairing haemoglobin disorders with ribozymes.
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.
MRC Molecular Haematology Unit, University of Oxford, John Radcliffe Hospital, UK.
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Weatherall
D000755:Anemia, Sickle Cell / Q000235:genetics / Q000628:therapy; D000818:Animals; D001809:Blood Viscosity; D002478:Cells, Cultured; D015672:Erythroid Precursor Cells / Q000187:drug effects / Q000378:metabolism; D005319:Fetal Hemoglobin / Q000096:biosynthesis / Q000235:genetics; D015316:Genetic Therapy / Q000009:adverse effects; D005914:Globins / Q000235:genetics*; D006453:Hemoglobinopathies / Q000235:genetics / Q000628:therapy*; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D016337:RNA, Catalytic / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000187:drug effects* / Q000235:genetics / Q000378:metabolism
D016337:RNA, Catalytic; D012333:RNA, Messenger; D005914:Globins; D005319:Fetal Hemoglobin