Antisense strategies in the treatment of hematological malignancies

Antisense nucleic acids and ribozymes recognize their target in a highly sequence-specific manner and are thought to be useful inhibitors of aberrant gene expression and pathogenic viral functions. Ribozymes combine the properties of antisense RNA and the ability to cleave the target RNA in an almost irreversible manner. Hematopoietic diseases such as infection of CD4+ human cells with the human immunodeficiency virus type 1 (HIV-1) or those forms of leukemia that occur as a result of chromosomal aberrations have challenged and strongly enhanced antisense research. Meanwhile, a number of clinical studies is conducted that involve the use of antisense nucleic acids and ribozymes. The aim of this review is to give a brief summary on the current state and the prospects of antisense nucleic acids and ribozymes, with particular view on antisense-mediated inhibition of HIV-1 replication and the expression of the ber-abl fusion gene that is linked with chronic myelogenous leukemia.