LNP-RNA prime editors

LNP-RNA prime editors refer to a drug delivery platform in which prime editor mRNA and chemically modified prime editing guide RNA (pegRNA) are encapsulated within lipid nanoparticles (LNPs) for in vivo prime genome editing. Prime editing enables search-and-replace genome alterations using a Cas9 nickase fused to a reverse transcriptase and a pegRNA, allowing precise nucleotide changes without double-strand breaks. Delivery via LNPs—rather than adeno-associated virus (AAV)—addresses issues of vector size, re-dosing, and chronic expression. This formulation enables in vivo gene editing but current studies report low efficiency, partly due to challenges in encapsulating large mRNAs and guide RNAs. LNP-RNA prime editors are being investigated primarily in preclinical models, such as mouse liver editing and phenylketonuria correction models. This technology is under active development by researchers at organizations such as University of Massachusetts Chan Medical School, with commercial interest from companies developing synthetic RNAs (e.g., TriLink Biotechnologies) and LNP components.

Other names

lipid nanoparticle prime editorLNP prime editor