Liver-targeted gene therapy: Approaches and challenges.
Liver-targeted gene therapy: Approaches and challenges.
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DOI：
10.1002/lt.24122
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发表时间：
2015-06
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影响因子：
--
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--
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肝脏在许多遗传性和获得性遗传性疾病中发挥着重要作用。它也是治疗某些不直接导致肝脏损伤的先天性代谢缺陷的场所。由于无数不良副作用和方法学限制，基于核酸的肝脏疾病疗法的进展受到严重限制。为了解决这些问题，近年来的研究工作不断加强，利用新型遗传工具开发靶向基因方法，例如锌指核酸酶（ZFN）、转录激活子样效应核酸酶（TALEN）和成簇规则间隔短回文重复序列（CRISPR），以及各种非病毒载体，例如睡美人转座子、piggyBac转座子和PhiC31整合酶。虽然这些方法中的每一种都利用了不同的基因修饰机制，但它们都依赖于将 DNA 和 RNA 分子有效递送到细胞中。本综述将概述当前和新兴的肝脏定向基因治疗和基因修复治疗策略。
Liver plays a major role in many inherited and acquired genetic disorders. It is also the site for the treatment of certain inborn errors of metabolism that do not directly cause injury to the liver. The advancement of nucleic acid-based therapies for liver maladies has been severely limited due to the myriad of untoward side effects and methodological limitations. To address these issues, research efforts in recent years have been intensified towards the development of targeted gene approaches using novel genetic tools, such as the zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPRs), as well as various non-viral vectors, such as Sleeping Beauty transposons, piggyBac transposons and PhiC31 integrase. While each of these methods utilizes a distinct mechanism of gene modification, all of them are dependent upon the efficient delivery of DNA and RNA molecules into the cell. This review will provide an overview on current and emerging therapeutic strategies for liver-directed gene therapy and gene repair.
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影响因子：
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