Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, John Radcliffe Hospital, Oxford, United Kingdom.
Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, John Radcliffe Hospital, Oxford, United Kingdom; Oxford Eye Hospital, John Radcliffe, Oxford, United Kingdom.
Int Rev Cell Mol Biol. 2022;372:175-205. doi: 10.1016/bs.ircmb.2022.04.009. Epub 2022 Aug 17.
RNA editing allows correction of pathological point mutations without permanently altering genomic DNA. Theoretically targetable to any RNA type and site, its flexibility and reversibility makes it a potentially powerful gene editing tool. RNA editing offers a host of potential advantages in specific niches when compared to currently available alternative gene manipulation techniques. Unlike DNA editors, which are currently too large to be delivered in vivo using a viral vector, smaller RNA editors fit easily within the capabilities of an adeno-associated virus (AAV). Unlike gene augmentation, which is limited by gene size and viral packaging constraints, RNA editing may correct transcripts too long to fit within a viral vector. In this article we examine the development of RNA editing and discuss potential applications and pitfalls. We argue that, although in its infancy, an RNA editing approach can offer unique advantages for selected retinal diseases.
RNA 编辑允许在不永久改变基因组 DNA 的情况下纠正病理性点突变。理论上可靶向任何 RNA 类型和位点，其灵活性和可逆性使其成为一种潜在强大的基因编辑工具。与目前可用的替代基因操作技术相比，RNA 编辑在特定领域具有许多潜在优势。与目前太大而无法使用病毒载体在体内递送的 DNA 编辑器不同，较小的 RNA 编辑器很容易适应腺相关病毒 (AAV) 的能力。与基因增强不同，RNA 编辑不受基因大小和病毒包装限制的限制，它可以纠正太长而无法容纳在病毒载体中的转录本。在本文中，我们研究了 RNA 编辑的发展，并讨论了潜在的应用和陷阱。我们认为，尽管处于起步阶段，但 RNA 编辑方法可为选定的视网膜疾病提供独特的优势。
