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Efficient prime editing in vivo and in vitro using lipid nanoparticles
Allen Y Jiang1,2,3, Ana Cristian1,2,3,4, Dominique L Brooks5,6,7
1Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Nature Nanotechnology
|June 15, 2026
Summary
Researchers optimized lipid nanoparticle (LNP) delivery for prime editing, a precise genome editing technology. This enhanced non-viral delivery achieved high in vivo editing efficiency in mouse livers, offering a promising alternative to viral methods.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- Prime editing enables precise genomic modifications in living systems.
- Non-viral lipid nanoparticle (LNP) delivery is preferred for in vivo gene editing.
- Current LNP delivery of prime editing systems shows low efficiency.
Purpose of the Study:
- To develop an optimized platform for lipid nanoparticle (LNP) delivery of prime editing systems.
- To overcome bottlenecks limiting prime editing efficiency with LNPs.
- To establish a non-viral delivery method for efficient in vivo prime editing.
Main Methods:
- Developed a systematic prime editing LNP (PE-LNP) optimization platform.
- Focused on optimizing cargo design to enhance editing efficiency.
- Evaluated PE-LNP performance in mouse liver models.
Main Results:
- Achieved 49% average in vivo prime editing in mouse liver with a single PE-LNP dose.
- Corrected the PAH R408W mutation, a cause of phenylketonuria, in a mouse model.
- Demonstrated minimized off-target editing and transient liver enzyme elevation compared to DNA delivery.
Conclusions:
- PE-LNPs offer a generalizable and efficient non-viral delivery method for in vivo prime editing.
- This approach provides a potentially curative treatment for genetic disorders like phenylketonuria.
- PE-LNPs represent a safe and effective alternative to viral delivery systems for liver gene editing.
