CRISPR/Cas 基因组编辑组件的眼部递送来治疗眼部疾病。
Ocular delivery of CRISPR/Cas genome editing components for treatment of eye diseases.
机构信息
Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, 6 Center Drive, Room 306, Bethesda, MD 20892, USA.
Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, 6 Center Drive, Room 306, Bethesda, MD 20892, USA.
出版信息
Adv Drug Deliv Rev. 2021 Jan;168:181-195. doi: 10.1016/j.addr.2020.06.011. Epub 2020 Jun 27.
A variety of inherited or multifactorial ocular diseases call for novel treatment paradigms. The newly developed genome editing technology, CRISPR, has shown great promise in treating these diseases, but delivery of the CRISPR/Cas components to target ocular tissues and cells requires appropriate use of vectors and routes of administration to ensure safety, efficacy and specificity. Although adeno-associated viral (AAV) vectors are thus far the most commonly used tool for ocular gene delivery, sustained expression of CRISPR/Cas components may cause immune reactions and an increased risk of off-target editing. In this review, we summarize the ocular administration routes and discuss the advantages and disadvantages of viral and non-viral vectors for delivery of CRISPR/Cas components to the eye. We review the existing studies of CRISPR/Cas genome editing for ocular diseases and discuss the major challenges of the technology in ocular applications. We also discuss the most recently developed CRISPR tools such as base editing and prime editing which may be used for future ocular applications.
多种遗传性或多因素眼病需要新的治疗模式。新开发的基因组编辑技术 CRISPR 在治疗这些疾病方面显示出巨大的潜力，但将 CRISPR/Cas 成分递送到目标眼组织和细胞需要适当使用载体和给药途径，以确保安全性、有效性和特异性。尽管腺相关病毒 (AAV) 载体迄今为止是最常用于眼部基因传递的工具，但 CRISPR/Cas 成分的持续表达可能会引起免疫反应和增加脱靶编辑的风险。在这篇综述中，我们总结了眼部给药途径，并讨论了用于将 CRISPR/Cas 成分递送到眼睛的病毒和非病毒载体的优缺点。我们回顾了用于眼部疾病的 CRISPR/Cas 基因组编辑的现有研究，并讨论了该技术在眼部应用中的主要挑战。我们还讨论了最近开发的 CRISPR 工具，如碱基编辑和先导编辑，它们可能用于未来的眼部应用。
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