Endogenous ADAR-Mediated RNA Editing

FREST's RNA editing technology uses guide RNA-based oligonucleotides to recruit endogenous ADAR enzymes to target mRNA. ADAR converts a specific adenosine (A) to inosine (I), which is read as guanosine (G) during translation. This enables RNA-level correction or modulation without permanent genome modification or exogenous editing enzymes.

Key Differences Between DNA Editing and RNA Editing

DNA editing introduces changes at the genome level, while RNA editing modifies genetic information at the RNA level. RNA editing enables transient and potentially reversible correction or modulation without permanent genome modification. 

In Vivo RNA Editing Demonstrated with LNP Delivery 

RNA-editing oligonucleotides formulated in LNP were administered intravenously to normal mice at 2 mg/kg. RNA editing activity was measured on Day 2 across multiple tissues, with editing efficiency reaching up to 76% in the liver.

From Platform Validation to Therapeutic Applications 

FREST is translating endogenous ADAR-mediated RNA editing into therapeutic programs by integrating guide RNA design, delivery optimization, in vivo proof-of-concept, and disease-focused target selection.