Beta Thalassemia (Animal Models)

Development of Murine Model Facilitates Gene Therapy Research

Published in Blood Weekly, October 23rd, 1995

A recently developed murine model will facilitate the development of a potential gene therapy cure for human (beta)⁰-thalassemia.

(beta)⁰-thalassemia is a disease characterized by severe anemia that begins during the first month of life. As the level of fetal hemoglobin declines, the affected child is unable to replace it with the major adult hemoglobin.

Individuals become transfusion-dependent, and while transfusion medicine has vastly improved survival over the past few years, significant complications from transfusion associated hepatitis and iron overload remain problematic.

Bone marrow transplantation...

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Source: Blood Weekly (1995-10-23)

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