The Application of Ribozymes and DNAzymes in Muscle and Brain

Nikolaos P Mastroyiannopoulos, James B Uney, Leonidas A Phylactou

Research output: Contribution to journalArticle (Academic Journal)peer-review

10 Citations (Scopus)
188 Downloads (Pure)


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.

Original languageEnglish
Pages (from-to)5460-5472
Number of pages13
Issue number8
Early online date9 Aug 2010
Publication statusPublished - Aug 2010


  • Animals
  • Brain
  • DNA, Catalytic
  • Humans
  • Muscles
  • RNA, Catalytic

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