Next-generation biological vector platforms for in vivo delivery of genome editing agents

被引：5

作者：

Leclerc, Delphine ^{[1
]}

Siroky, Michael ^{[1
]}

Miller, Shannon M. ^{[1
]}

机构：

[1] Scripps Res Inst, Dept Chem, La Jolla, CA 92037 USA

来源：

CURRENT OPINION IN BIOTECHNOLOGY | 2024年 / 85卷

关键词：

molecule therapeutics; gene augmentation therapies; GENE-TRANSFER; LENTIVIRAL VECTORS; SKELETAL-MUSCLE; ADENOASSOCIATED VIRUS; DIRECTED EVOLUTION; EFFICIENT; CRISPR/CAS9; PROTEIN; RNA; EXPRESSION;

D O I：

10.1016/j.copbio.2023.103040

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

CRISPR-based genome editing holds promise for addressing genetic disease, infectious disease, and cancer and has rapidly advanced from primary research to clinical trials in recent years. However, the lack of safe and potent in vivo delivery methods for CRISPR components has limited most ongoing clinical trials to ex vivo gene therapy. Effective CRISPR in vivo genome editing necessitates an effective vehicle ensuring target cell transduction while minimizing off-target effects, toxicity, and immune reactions. In this review, we examine promising biological-derived platforms to deliver DNA editing agents in vivo and the engineering thereof, encompassing potent viral-based vehicles, flexible protein nanocages, and mammalian-derived particles.

引用

页数：11

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