What is the role of gene therapy in the treatment of sickle cell anemia (SCA)?

Updated: May 12, 2021
  • Author: Joseph E Maakaron, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Gene therapy is emerging as a possible cure for severe SCD. Experimental approaches include modification of autologous stem cells with lentiviral vectors to add normal globin genes, gene editing to correct the sickle cell disease mutation, and genetic silencing to enhance production of fetal hemoglobin. [66, 67, 68] For example, a pilot study by Esrick et al reported reduction or elimination of clinical manifestations of SCD in six patients who received autologous CD34+ cells transduced with a lentiviral vector that downregulates a gene responsible for repressing fetal hemoglobin production in adult red blood cells. [69]


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