Ribozyme therapy

Citation
Ja. Sandberg et al., Ribozyme therapy, PROG R RES, 31, 2001, pp. 370-373
Citations number
18
Categorie Soggetti
Current Book Contents
ISSN journal
14222140
Volume
31
Year of publication
2001
Pages
370 - 373
Database
ISI
SICI code
1422-2140(2001)31:<370:RT>2.0.ZU;2-1
Abstract
Ribozymes are catalytic RNA molecules that can cleave targeted RNA in a seq uence-specific manner. The ability to target these compounds to a specific molecular target opens up exciting opportunities for rational drug design f or the treatment of asthma and COPD. Strategic cleavage of RNA encoding any of the many cytokines and/or their receptors now believed to play a role i n the etiology of these diseases could result in effective therapeutics wit h minimal toxicity. Ribozymes have been demonstrated to be efficacious in v ivo, result in minimal toxicity, and are currently in clinical trials. Syst emic or pulmonary delivery could be used to deliver ribozymes to their site of action.