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Review Article

基于基因组 DNA 的基因治疗中非编码元件的前景

卷 22, 期 2, 2022

发表于: 19 April, 2021

页: [89 - 103] 页: 15

弟呕挨: 10.2174/1566523221666210419090357

价格: $65

摘要

自几十年前第一次临床试验开始以来，基因治疗取得了重大发展，并且无论免疫反应和插入诱变等障碍如何，基因治疗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) 与目前用于基因治疗的互补 DNA (cDNA) 或小基因相比的优势。 这篇综述的重点是概述 gDNA 基因座在基因治疗中的潜在用途的进展和未来，扩大分子医学的治疗库

关键词: 基因治疗、cDNA、基因组DNA、非编码DNA、基因表达、多基因疾病

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图形摘要

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