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.
MeSH terms
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Anemia, Sickle Cell / genetics
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Anemia, Sickle Cell / therapy
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Animals
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Blood Viscosity
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Cells, Cultured
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Erythroid Precursor Cells / drug effects
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Erythroid Precursor Cells / metabolism
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Fetal Hemoglobin / biosynthesis
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Fetal Hemoglobin / genetics
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Genetic Therapy* / adverse effects
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Globins / genetics*
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Hemoglobinopathies / genetics
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Hemoglobinopathies / therapy*
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Humans
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Mice
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Mice, Transgenic
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RNA, Catalytic / therapeutic use*
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RNA, Messenger / drug effects*
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RNA, Messenger / genetics
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RNA, Messenger / metabolism
Substances
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RNA, Catalytic
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RNA, Messenger
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Globins
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Fetal Hemoglobin