RIBOZYME AND OMICS
Specificity by specific RNA-sensing riboswitch
Efficacy by dual function of RNA-cleavage & transgene induction
Specific targeting of disease-associated/related RNA
Targeting of non-druggable targetInhibition of target RAN expression & expression of disease-specific therapeutic gene by one molecule
Regulation of therapeutic gene expression in proportion to target RNA level
Endogenous cellular RNA expression levelMaintenance of intracellular therapeutic gene expression via DNA vector as tool
High specificity and efficacy, High safety acting w/ its & fidelity, No off-target
Modular Engineering Diverse indication, imaging / theranostics / cell therapy
Gene editing at RNA level
Minimization of genome toxicity & eternal genome changeRetaining advantages / Overcoming disadvantages of existing DNA/RNA technologies