Ribozymes and the anti-gene therapy: how a catalytic RNA can be used to inhibit gene function.
Ribozymes are RNA molecules that possess the dual properties of RNA sequence-specific recognition and site-specific cleavage of other RNA molecules. These properties provide powerful tools for studies requiring gene inhibition, when the DNA sequence is known. The use of these molecules goes beyond basic research, with a potential impact in therapeutical practice in medicine in the near future. In this review, we briefly describe the progress towards developing this class of molecules and its applications for the control of gene expression.
Muotri
A R
AR
Departamento de Microbiologia, Instituto de Ciências Biomédicas, Universidade de São Paulo, São Paulo, Brazil. armuotri@usp.br
da Veiga Pereira
L
L
dos Reis Vasques
L
L
Menck
C F
CF
D000818:Animals; D001483:Base Sequence; D005786:Gene Expression Regulation / Q000187:drug effects*; D006801:Humans; D016337:RNA, Catalytic / Q000627:therapeutic use*
D016337:RNA, Catalytic