Therapeutic applications of ribozymes.

Abstract:

:The demonstration that RNA can be cleaved by cis or trans ribozymes (catalytic RNAs, RNA enzymes) has potentially important therapeutic implications. Since their discovery in the 1980s, the biochemistry and conserved sequences of ribozymes have been well characterized. Ribozymes are effective modulators of gene expression because of their simple structure, sitespecific cleavage activity, and catalytic potential. The targets of ribozyme-mediated gene modulation have ranged from cancer cells to foreign genes that cause infectious diseases. Additional target sites for ribozymes are in initial phases of development and design. Ribozymes have been targeted against a myriad of genes, including oncogenes (ras, BCR-ABL, c-fos) and drug resistance genes, as well as the human immunodeficiency virus-type I genome. These ribozymes have cleaved the target RNAs in vitro and altered the cellular pathology. Currently, the therapeutic application of ribozymes to human diseases is limited by gene transfer systems. It is anticipated that ribozymes ultimately will play an important role in human gene therapy.

journal_name

Pharmacol Ther

authors

Kijima H,Ishida H,Ohkawa T,Kashani-Sabet M,Scanlon KJ

doi

10.1016/0163-7258(95)02008-x

subject

Has Abstract

pub_date

1995-01-01 00:00:00

pages

247-67

issue

2

eissn

0163-7258

issn

1879-016X

pii

0163-7258(95)02008-X

journal_volume

68

pub_type

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