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Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads
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DOI：10.1016/j.addr.2024.115346.png)
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Abstract
En 中文
Gene editing technologies have the potential to correct genetic disorders by modifying, inserting, or deleting specific DNA sequences or genes, paving the way for a new class of genetic therapies. While gene editing tools continue to be improved to increase their precision and efficiency, the limited efficacy of in vivo delivery remains a major hurdle for clinical use. An ideal delivery vehicle should be able to target a sufficient number of diseased cells in a transient time window to maximize on-target editing and mitigate off-target events and immunogenicity. Here, we review major advances in novel delivery platforms based on cell-derived vesicles- extracellular vesicles and virus-like particles- for transient delivery of gene editing payloads. We discuss major findings regarding packaging, in vivo biodistribution, therapeutic efficacy, and safety concerns of cell-derived vesicles delivery of gene editing cargos and their potential for clinical translation.
Keywords:
Biovesicles
Extracellular vesicles
Virus -like particles
CRISPR-Cas9
Cre recombinase
Delivery vehicles
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