Gene therapy using hematopoietic stem cells.

While gene therapy using hematopoietic stem cells was the first area of investigation in the field, success has proven elusive. However, significant progress has been achieved recently in methods for more effective gene transfer and expression. In addition to greatly improved results using retroviral vectors, adeno-associated vectors and lentiviral vectors appear to be promising for stable transduction of hematopoietic stem cells. These advances, documented in animal transplant models, are now being applied to clinical trials.