the application of ribozymes and dnazymes in muscle and brain核酶的应用和dnazymes肌肉和大脑.pdfVIP

Molecules 2010, 15, 5460-5472; doi:10.3390/molecule OPEN ACCESS molecules ISSN 1420-3049 /journal/molecules Review The Application of Ribozymes and DNAzymes in Muscle and Brain Nikolaos P. Mastroyiannopoulos 1, James B. Uney 2 and Leonidas A. Phylactou 1,* 1 Department of Molecular Genetics, Function Therapy, The Cyprus Institute of Neurology Genetics. PO Box 23462, Nicosia, Cyprus 2 Henry Welcome Laboratories for Integrative Neurosciences and Endocrinology, University of Bristol, Whilston street, Bristol, BS13NY, UK * Author to whom correspondence should be addressed; E-Mail: laphylac@cing.ac.cy; Tel.:+357 223582600; Fax: +357 Received: 18 June 2010; in revised form: 3 August 2010 / Accepted: 5 August 2010 / Published: 9 August 2010 Abstract: The discovery of catalytic nucleic acids (CNAs) has provided scientists with valuable tools for the identification of new therapies for several untreated diseases through down regulation or modulation of endogenous gene expression involved in these ailments. These CNAs aim either towards the elimination or repair of pathological gene expression. Ribozymes, a class of CNAs, can be mostly used to down-regulate (by RNA cleavage) or repair (by RNA trans-splicing) unwanted gene expression involved in disease. DNAzymes, derived by in vitro selection processes are also able to bind and cleave RNA targets and therefore down-regulate gene expression. The purpose of this review is to present and discuss several applications of ribozymes and