Published in Gene Therapy Weekly, April 29th, 1996
These diseases are single-gene disorders that are the result of a mutation that alters the structure of (beta)-globin chains. Although the molecular bases of these disorders have been thoroughly characterized, and the major regulatory elements of the (beta)-globin gene cluster precisely localized, translation of this knowledge into an effective gene therapy protocol has not been realized.
Previous studies have shown the ability of retroviral...
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