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首页> 外文期刊>Expert opinion on biological therapy >Development of mammalian artificial chromosomes for the treatment of genetic diseases: Sandhoff and Krabbe diseases.
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Development of mammalian artificial chromosomes for the treatment of genetic diseases: Sandhoff and Krabbe diseases.

机译：用于治疗遗传疾病的哺乳动物人工染色体的开发：桑霍夫和克拉伯病。

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摘要

Mammalian artificial chromosomes (MACs) are being developed as alternatives to viral vectors for gene therapy applications, as they allow for the introduction of large payloads of genetic information in a non-integrating, autonomously replicating format. One class of MACs, the satellite DNA-based artificial chromosome expression vehicle (ACE), is uniquely suited for gene therapy applications, in that it can be generated denovo in cells, along with being easily purified and readily transferred into a variety of recipient cell lines and primary cells. To facilitate the rapid engineering of ACEs, the ACE System was developed, permitting the efficient and reproducible loading of pre-existing ACEs with DNA sequences and/or target gene(s). As a result, the ACE System and ACEs are unique and versatile platforms for ex vivo gene therapy strategies that circumvent and alleviate existing safety and delivery limitations surrounding conventional gene therapy vectors. This review will focus on the status of MAC technologies and, in particular, the application of the ACE System towards an ex vivo gene therapy treatment of lysosomal storage diseases, specifically Sandhoff (MIM #268800) and Krabbe (MIM #245200) diseases.
机译：哺乳动物人工染色体（MACs）正被开发为基因治疗应用中病毒载体的替代品，因为它们允许以非整合，自主复制的格式引入大量的遗传信息有效载荷。一类MAC是基于卫星DNA的人工染色体表达载体（ACE），非常适合基因治疗应用，因为它可以在细胞中产生树突状细胞，并且易于纯化并易于转移到各种受体细胞中系和原代细胞。为了促进ACE的快速工程开发，开发了ACE系统，可以有效且可重现地装载预先存在的带有DNA序列和/或靶基因的ACE。结果，ACE系统和ACE是用于离体基因治疗策略的独特且通用的平台，其规避并减轻了围绕常规基因治疗载体的现有安全性和递送限制。这篇综述将侧重于MAC技术的现状，尤其是ACE系统在溶酶体贮积病，特别是Sandhoff（MIM＃268800）和Krabbe（MIM＃245200）疾病的离体基因治疗中的应用。

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