Surface engineering of cell-derived particles for targeted delivery of genome editors
Summary
Genome editing technologies hold significant promise for the treatment of genetic disorders, but safe and efficient delivery remains a major barrier to their clinical translation. Cell-derived particles, including extracellular vesicles (EVs) and virus-like particles (VLPs), have emerged as promising non-viral delivery platforms, offering biological compatibility and the capacity to package and deliver genome editors as ribonucleoprotein (RNP) complexes in a transient manner that reduces the risk of off-target editing.
Genome editing technologies hold significant promise for the treatment of genetic disorders, but safe and efficient delivery remains a major barrier to their clinical translation. Cell-derived particles, including extracellular vesicles (EVs) and virus-like particles (VLPs), have emerged as promising non-viral delivery platforms, offering biological compatibility and the capacity to package and deliver genome editors as ribonucleoprotein (RNP) complexes in a transient manner that reduces the risk of off-target editing. The surface composition of these vehicles is shaped by their biogenesis pathway and their cell of origin, which in turn govern their cellular tropism. Through various engineering strategies, including producer cell engineering, viral-glycoprotein pseudotyping, and post-isolation surface modifications, this tropism can be expanded or redirected toward cell types and tissues beyond the liver. Here, we review the biology underlying cell-derived particle surface composition and the targeting strategies employed to engineer cell-type specificity.
Abstract from PubMed Central (PMID 42981775, PMC13739235). This entry was added automatically by our daily literature monitor because it matches the topics we follow; read the full paper at the original source.
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