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.
Weatherall
D J
DJ
MRC Molecular Haematology Unit, University of Oxford, John Radcliffe Hospital, UK.
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