{"id":"doi:10.1038/s41587-023-02078-y","kind":"article","title":"Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo","authors":["An M","Raguram A","Du SW","Banskota S","Davis JR","Newby GA","Chen PZ","Palczewski K","Liu DR"],"author_count":9,"year":2024,"published":"2024-01-08","venue":"Nature biotechnology","cited_by":242,"doi":"10.1038/s41587-023-02078-y","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41587-023-02078-y","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41587-023-02078-y","updated":null,"pmid":"38191664","pmcid":"PMC11228131","openalex_id":null,"pdf_url":"https://europepmc.org/articles/PMC11228131/pdf","abstract":"Prime editing enables precise installation of genomic substitutions, insertions and deletions in living systems. Efficient in vitro and in vivo delivery of prime editing components, however, remains a challenge. Here we report prime editor engineered virus-like particles (PE-eVLPs) that deliver prime editor proteins, prime editing guide RNAs and nicking single guide RNAs as transient ribonucleoprotein complexes. We systematically engineered v3 and v3b PE-eVLPs with 65- to 170-fold higher editing efficiency in human cells compared to a PE-eVLP construct based on our previously reported base editor eVLP architecture. In two mouse models of genetic blindness, single injections of v3 PE-eVLPs resulted in therapeutically relevant levels of prime editing in the retina, protein expression restoration and partial visual function rescue. Optimized PE-eVLPs support transient in vivo delivery of prime editor ribonucleoproteins, enhancing the potential safety of prime editing by reducing off-target editing and obviating the possibility of oncogenic transgene integration.","abstract_license":"CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)","license":"CC-BY","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":[],"details":null,"provenance":"europepmc","attribution":"Abstract of \"Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo\" by An M, Raguram A, Du SW et al., https://doi.org/10.1038/s41587-023-02078-y, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","domain_slug":"medicine"}],"cited_in_dossiers":[]}