在过去的几十年里，基因编辑证明了有可能减弱遗传性疾病、传染性疾病、免疫性疾病、癌症和退行性疾病的各个根源。最近，事实证明，成簇规则间隔短回文重复序列-CRISPR 相关蛋白 9 (CRISPR-Cas9) 编辑可有效编辑基因组、癌性或微生物 DNA，从而限制疾病的发作或传播。然而，递送 CRISPR-Cas9 货物并引发保护性免疫反应的策略需要安全地递送至疾病靶细胞和组织。虽然基于病毒载体的系统和病毒颗粒表现出高效和稳定的转基因表达，但每种系统的包装能力和继发不良免疫反应均受到限制。相比之下，非病毒载体脂质纳米颗粒已成功用作疫苗和治疗药物。在此，我们重点介绍每种可用的基因传递系统，用于治疗和预防各种传染病、炎症、遗传和退行性疾病。意义声明：用于疾病治疗和预防的 CRISPR-Cas9 基因编辑是一个新兴领域，可以改变许多慢性衰弱性疾病的结果。版权所有 © 2023。由 Elsevier Inc. 出版。

For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. STATEMENT OF SIGNIFICANCE: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.Copyright © 2023. Published by Elsevier Inc.