Anemia Gene Therapy

Clinical Trial for Erythropoietin-Responsive Anemia One Step Closer

Published in Gene Therapy Weekly, November 17th, 1997

Long-term erythropoietin (Epo) expression mediated via a replication-defective adenoviral vector was possible in both an immunocompetent murine model and non-human primate model.

Epo is a cytokine that functions as the major regulator of red blood cell (RBC) production by mammalian bone marrow. In human adults, Epo is produced in the kidney and under hypoxic or anemic conditions, is secreted into the systemic circulation.

When it reaches the bone marrow, Epo stimulates RBC production by enhancing the proliferation of, and preventing the apoptosis of, RBC precursors, including both erythroid burst-forming units (BFU-E) and erythroid colony-forming...

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Source: Gene Therapy Weekly (1997-11-17)

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