Hematopoietic stem cell gene therapy: selecting only the best.

  • Arthur Bank
  • Published 2003 in The Journal of clinical investigation
Hematopoietic stem cell (HSC) gene therapy can potentially cure a variety of human hematopoietic diseases, such as sickle cell disease. Selection and expansion of gene-corrected HSCs has now been accomplished for the first time using HSC from large animals - dogs and humans - with a novel drug-resistance gene, MGMT, which is not expressed in normal HSCs… (More)