Blood Disorders

Gene Therapy for Sickle Cell Disease and (beta)-Thalassemia One Step Closer

Published in Gene Therapy Weekly, April 29th, 1996

A stable, high-titer retroviral vector has been developed that gives rise to high levels of expression of transduced globin genes and offers hope for gene therapy of sickle cell disease and (beta)-thalassemia.

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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Source: Gene Therapy Weekly (1996-04-29)

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