树枝状大分子介导的工程染色体转移。

Dendrimer mediated transfer of engineered chromosomes.

作者信息

Katona Robert L

机构信息

Institute of Genetics, Biological Research Center, Hungarian Academy of Sciences, Szeged, Hungary.

出版信息

Methods Mol Biol. 2011;738:151-60. doi: 10.1007/978-1-61779-099-7_11.

DOI:10.1007/978-1-61779-099-7_11

PMID:21431726

Abstract

Gene therapy encounters important problems such as insertional mutagenesis caused by the integration of viral vectors. These problems could be circumvented by the use of mammalian artificial chromosomes (MACs) that are unique and high capacity gene delivery tools. MACs were delivered into various target cell lines including stem cells by microcell-mediated chromosome transfer (MMCT), microinjection, and cationic lipid and dendrimer mediated transfers. MACs were also cleansed to more than 95% purity before transfer with an expensive technology. We present here a method by which MACs can be delivered into murine embryonic stem (ES) cells with a nonexpensive, less tedious, but still efficient way.

摘要

基因治疗面临着一些重要问题，比如病毒载体整合导致的插入诱变。这些问题可以通过使用哺乳动物人工染色体（MACs）来规避，MACs是独特且具有高容量的基因递送工具。通过微细胞介导的染色体转移（MMCT）、显微注射以及阳离子脂质和树枝状大分子介导的转移，MACs被递送至包括干细胞在内的各种靶细胞系。在转移之前，还通过一项昂贵的技术将MACs纯化至纯度超过95%。我们在此介绍一种方法，通过该方法可以用一种成本不高、不那么繁琐但仍然高效的方式将MACs递送至小鼠胚胎干细胞（ES细胞）中。