Correction of genetic blood defects by gene transfer

Abstract

Gene therapy has been proposed as an appealing tool for introducing a normal gene into affected hematopoietic stem cells to correct their inherited defect. Theoretically, in the absence of a related human leukocyte antigen identical donor, gene therapy could be an alternative given the accessibility and the information available on the hematopoietic stem cell biology. This chapter describes the progress and limits of the gene therapy approach applied to some genetic blood defects that appear to be good targets for this strategy. © 2006 Humana Press Inc.