The Structural Domains And Mechanism Of Action Of Ribozymes

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1) Describe the structural domains and mechanism of action of ribozymes. How can ribozymes be useful as therapeutic agents? (10 points)

RNA molecules that act as enzymes are called ribozymes. They are capable of catalyzing the cleavage of their own RNA or other RNA substrates (Missailidis, 2008). Ribozymes are involved in viroid replication, RNA splicing and protein synthesis. (Clark & Russel, 2005) The structure of ribozymes consist of a catalytic domain and a substrate binding domain. (Ebrahimian, 2015)

The substrate binding domain has a specific sequence antisense to the target mRNA. This sequence recognizes and hybridizes specifically to its substrate. (Missailidis, 2008). Alteration of the substrate binding domain can be done so that the substrate specifically cleaves any mRNA sequence. The RNA catalytic domain cleaves the substrate at a target site recognized by the ribozyme (Glick &Pasternak, 2003). The resultant products are then degraded by ribonucleases and the ribozyme dissociates itself from the RNA products and binds to another mRNA to be cleaved. (Missailidis, 2008)
Therapeutic ribozymes can be designed to target almost any RNA sequence and decrease the amount of a particular protein that is synthesized (Glick & Pasternak, 2003). This can be done by incorporating the catalytic domain of ribozyme into short oligonucleotides antisense to the target mRNA (Missailidis, 2008). Ribozymes can be used to identify specific sites and introduced into the system to

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