Gene therapy for the hemoglobin disorders: past, present, and future.

  title={Gene therapy for the hemoglobin disorders: past, present, and future.},
  author={Derek A. Persons and A. W. Nienhuis},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  volume={97 10},
W ith the development in the early 1980s of technology to transfer genes to murine hematopoietic stem cells by using recombinant murine oncoretroviral vectors (1), the possibility of genetic therapy for a number of human disorders of the lympho-hematopoietic system seemed an attainable goal. Hemoglobin disorders were among the first diseases to be considered for gene therapy. Both sickle cell disease and the thalassemias are common monogenic disorders worldwide that cause serious morbidity and… CONTINUE READING


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