Targeting leukemic fusion proteins with small interfering RNAs:recent advances and therapeutic potentials

RNA interference has become an indispensable research tool to study gene func-tions in a wide variety of organisms.Because of their high efficacy and specificity,RNA interference-based approaches may also translate into new therapeutic stra-tegies to treat human diseases.In particular,oncogenes such as leukemic fusionproteins,which arise from chromosomal translocations,are promising targets forsuch gene silencing approaches,because they are exclusively expressed in pre-cancerous and cancerous tissues,and because they are frequently indispensablefor maintaining the malignant phenotype.This review summarizes recent deve-lopments in targeting leukemia-specific genes and discusses problems andapproaches for possible clinical applications.