Hybrid nucleic acid molecules and vectors including .beta.-globin regulatory elements
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United States of America Patent
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Feb 25, 2003
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N/A
app pub date -
Oct 1, 1999
filing date -
Oct 1, 1998
priority date (Note) -
Expired
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Abstract
The invention relates to hybrid nucleic acid molecules for gene therapy in cells of the erythroid lineage, and in particular .alpha.-, .beta.-, .delta.-, .epsilon., .gamma.-, or .zeta.-globin nucleotide sequences operably linked to .beta.-globin regulatory elements. The hybrid nucleic acid molecules, at single copy, are capable of producing a polypeptide. The hybrid nucleic acid molecules are useful for treatment of hemoglobinopathies such as sickle cell anemia or .beta.-thalassemia. The hybrid nucleic acid molecules are useful at single copy for erythroid expression at single copy of RNA and polypeptides in transgenic animals.
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