用于递送治疗性基因的工程染色体。

Engineering chromosomes for delivery of therapeutic genes.

作者信息

Irvine Danielle V, Shaw Margaret L, Choo K H Andy, Saffery Richard

机构信息

Chromosome Research Group, Murdoch Childrens Research Institute, Royal Children's Hospital, Department of Paediatrics, University of Melbourne, Flemington Road, Parkville 3052, Australia.

出版信息

Trends Biotechnol. 2005 Dec;23(12):575-83. doi: 10.1016/j.tibtech.2005.10.001. Epub 2005 Oct 19.

DOI:10.1016/j.tibtech.2005.10.001

PMID:16242803

Abstract

The ability to create fully functional human chromosome vectors represents a potentially exciting gene-delivery system for the correction of human genetic disorders with several advantages over viral delivery systems. However, for the full potential of chromosome-based gene-delivery vectors to be realized, several key obstacles must be overcome. Methods must be developed to insert therapeutic genes reliably and efficiently and to enable the stable transfer of the resulting chromosomal vectors to different therapeutic cell types. Research to achieve these outcomes continues to encounter major challenges; however recent developments have reiterated the potential of chromosome-based vectors for therapeutic gene delivery. Here we review the different strategies under development and discuss the advantages and problems associated with each.

摘要

创建具有完全功能的人类染色体载体的能力代表了一种潜在的令人兴奋的基因传递系统，用于纠正人类遗传疾病，与病毒传递系统相比具有几个优势。然而，要实现基于染色体的基因传递载体的全部潜力，必须克服几个关键障碍。必须开发出可靠且高效地插入治疗性基因的方法，并使所得染色体载体能够稳定地转移到不同的治疗细胞类型中。为实现这些成果所做的研究仍面临重大挑战；然而，最近的进展再次强调了基于染色体的载体用于治疗性基因传递的潜力。在这里，我们回顾正在开发的不同策略，并讨论每种策略的优点和问题。