Gene therapy for chronic myelogenous leukemia

Chronic myelogenous leukemia (CML) is characterized by a balanced transloca tion that leads to the formation of the the BCR-ABL fusion gene. Although a utografts can prolong the life of CML patients, patients relapse owing to m alignant cells that persist in the graft and the host. This review discusse s various experimental strategies that target the BCR-ABL gene or gene prod ucts that are downstream of it. Various strategies have been adopted to blo ck BCR-ABL at the gene, mRNA and protein level, One promising strategy invo lves the cotransduction of a patient's hematopoietic stem cells (HSCs) with anti-BCR-ABL antisense sequences and a drug resistance gene. This might al low for the elimination of any residual disease in the graft or host by che motherapy while rendering any drug-resistant, malignant CML HSCs functional ly normal.