CRISPR/Cas-guided single-guide RNAs are presented as part of the RNA therapeutic landscape covered by the review. The abstract associates RNA-based strategies with editing applications.
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CRISPR/Cas-guided single-guide RNAs
Candidate: toolkit item1 source documents4 linked claims
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RNA-based strategies are applied to gene silencing, editing, protein replacement, immune activation, and targeted drug delivery.
RNA-targeted therapy is shifting molecular medicine from a protein-centric view toward an RNA-regulatory network paradigm.
Diverse RNA therapeutics include ASOs, siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.
Common challenges for RNA therapeutics include in vivo stability, delivery efficiency, and immune activation.