DEVELOPMENT OF RIBOZYMES FOR GENE-THERAPY

Ribozymes are a class of RNA molecules that can perform catalytically in the absence of protein. Specifically, they can hybridize to and cle ave target RNA molecules independent of cellular proteins. The cleaved target RNA can not be translated thereby preventing synthesis of a sp ecific protein. The therapeutic application is to target the ribozyme to the mRNA of a key protein or proteins involved in maintaining a dis ease state resulting in a cure. The ribozymes cain be chemically synth esized and delivered to cells can be expressed from an expression vect or following either permanent or transient transfection. Therapeutic a pplications of ribozymes have been in the area of AIDS and cancer. The following article describes the ribozymes in more detail with respect to optimizing the design to obtain the maximal cleavage rate, identif ying cleavage sites within the target RNA and delivering the ribozymes to cell of interest both in vitro and in vivo.