Department of Ophthalmology, the University of North Carolina at Chapel Hill, Chapel Hill, NC27599, USA.
Carolina Institute for NanoMedicine, the University of North Carolina at Chapel Hill, Chapel Hill, NC27599, USA.
Curr Gene Ther. 2022;22(2):89-103. doi: 10.2174/1566523221666210419090357.
Gene therapy has made significant development since the commencement of the first clinical trials a few decades ago and has remained a dynamic area of research regardless of obstacles such as immune response and insertional mutagenesis. Progression in various technologies like next-generation sequencing (NGS) and nanotechnology has established the importance of non-- coding segments of a genome, thereby taking gene therapy to the next level. In this review, we have summarized the importance of non-coding elements, highlighting the advantages of using full- length genomic DNA loci (gDNA) compared to complementary DNA (cDNA) or minigene, currently used in gene therapy. The focus of this review is to provide an overview of the advances and the future of potential use of gDNA loci in gene therapy, expanding the therapeutic repertoire in molecular medicine.
自几十年前首例临床试验开始以来，基因治疗取得了重大进展，并且尽管存在免疫反应和插入突变等障碍，它仍然是一个充满活力的研究领域。下一代测序（NGS）和纳米技术等各种技术的进步确立了基因组非编码片段的重要性，从而将基因治疗提升到了一个新的水平。在这篇综述中，我们总结了非编码元件的重要性，强调了使用全长基因组 DNA 基因座（gDNA）相对于目前用于基因治疗的 cDNA 或小基因的优势。本综述的重点是提供基因治疗中 gDNA 基因座的应用的进展和未来的概述，从而扩展分子医学的治疗范围。
