The Application of Ribozymes and DNAzymes in Muscle and Brain

Molecules

Nikolaos P. Mastroyiannopoulos¹  James B. Uney¹ 

[1] ¹Department of Molecular Genetics, Function & Therapy, The Cyprus Institute of Neurology & Genetics. PO Box 23462, Nicosia, Cyprus ²Henry Welcome Laboratories for Integrative Neurosciences and Endocrinology, University of Bristol, Whilston street, Bristol, BS13NY, UK

关键词: catalytic nucleic acids;    ribozyme;    DNAzyme;    brain;    muscle;   

DOI  :  10.3390/molecules15085460

来源: mdpi

【 摘 要 】

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 DNAzymes in muscle and brain. There are several diseases which affect muscle and brain and catalytic nucleic acids have been used as tools to target specific cellular transcripts involved in these groups of diseases.

【 授权许可】

CC BY   
This is an open access article distributed under the Creative Commons Attribution License (CC BY) which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.