The potential application of ribozymes for the treatment of hematological disorders

Abstract

With the identification and increasing understanding of the genes involved in neoplastic transformation has come the realization that abrogation of these genes' products may lead to cell death or a return to normalcy. The use of ribozymes and their nucleic acid cousins, antisense oligodeoxynucleotides (ODNs), are two such ways of perturbing the disease-related gene expression. This review will look at the development and application of ribozymes to abrogate gene expression, with particular relevance to hematological settings. Some examples of antisense ODNs will also be mentioned where appropriate.