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 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.
Highlights
Selective gene silencing by catalytic nucleic acids is a field that has been used with great success for studying natural processes in muscle and brain
Hammerhead ribozymes were capable of cleaving c-myb RNA and as a result inhibit smooth muscle cell proliferation [26]. This finding indicated that hammerhead ribozymes have the potential to inhibit the hyperproliferation of smooth muscle cells that occurs in many patients after coronary angioplasty
Several different therapeutic attempts have been described as potential treatment approaches for many muscle and brain diseases
Summary
Selective gene silencing by catalytic nucleic acids is a field that has been used with great success for studying natural processes in muscle and brain. The most extensively studied of the catalytic nucleic acids, exist in a range of distinct categories of naturally occurring catalytic RNA. These include a series of small ribozymes important for the rolling circle replication of viroid genomes, such as hammerhead and hairpin ribozymes [1,2,3], group I introns [4,5,6], the RNA component of RNase P [7], and hepatitis delta virus ribozyme [8]. This review will focus on the use of ribozymes and DNAzymes towards the study and potential therapy of muscle and brain diseases
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