用于杜氏肌营养不良症基因治疗的微肌营养不良蛋白基因的密码子优化

Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy.

作者信息

Athanasopoulos Takis, Foster Helen, Foster Keith, Dickson George

机构信息

Institute of Biomedical and Life Sciences, South West London Academic Network, St. George's University of London, London, UK.

出版信息

Methods Mol Biol. 2011;709:21-37. doi: 10.1007/978-1-61737-982-6_2.

DOI:10.1007/978-1-61737-982-6_2

PMID:21194019

Abstract

Duchenne muscular dystrophy (DMD) is a severe muscle wasting X-linked genetic disease caused by dystrophin gene mutations. Gene replacement therapy aims to transfer a functional full-length dystrophin cDNA or a quasi micro/mini-gene into the muscle. A number of AAV vectors carrying microdystrophin genes have been tested in the mdx model of DMD. Further modification/optimization of these microgene vectors may improve the therapeutic potency. In this chapter, we describe a species-specific, codon optimization protocol to improve microdystrophin gene therapy in the mdx model.

摘要

杜兴氏肌营养不良症（DMD）是一种由肌营养不良蛋白基因突变引起的严重肌肉萎缩性X连锁遗传病。基因替代疗法旨在将功能性全长肌营养不良蛋白cDNA或准微/小基因导入肌肉。一些携带微肌营养不良蛋白基因的腺相关病毒（AAV）载体已在DMD的mdx模型中进行了测试。对这些微基因载体进行进一步的修饰/优化可能会提高治疗效果。在本章中，我们描述了一种物种特异性的密码子优化方案，以改善mdx模型中的微肌营养不良蛋白基因治疗。