A Model for Gene Therapy: Gene Replacement in the Treatment of Sickle Cell Anemia and Thalassemia

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This research paper was written in 1978 by Ward Merkeley, M.D. when he was a first year medical student attending the University Of Utah School of Medicine. It is one of the first original papers suggesting and exploring the theoretical potentials and practical limitations of Gene Therapy. The paper discusses in technical detail the means of isolating and inserting a normal hemogloblin gene into the erythoid stem cells of people with Sickle Cell Anemia and B Thalassemia. The difficulties and limitation of Gene Therapy are discussed in detail, as well as, some ethical considerations.