ABSTRACT
Chronic lymphocytic leukemia (CLL) is a malignancy of well-differentiated B
lymphocytes that express surface immunoglobulin and a constellation of surface
differentiation antigens. CLL cells generally are resistant to apoptosis but maintain
some of the physiologic functions of B cells such as the ability to present antigen when
appropriately stimulated. Although there are a number of chromosome abnormalities
associated with CLL such as trisomy 12 and deletions at 13q, 11q, or 17p, there have not
been any genes found responsible for development or progression of the disease.
Therefore, gene replacement or knockout strategies have not been considered. Gene
therapy approaches for patients with CLL have primarily been directed at modifying
autologous leukemia cells to produce antileukemia vaccines. Harnessing and utilizing the
immune system as a therapeutic modality could provide a unique and powerful approach,
distinct from traditional chemotherapy. Strategies will be reviewed in this chapter.
