首页> 外文期刊>Gene Therapy and Molecular Biology >Gene transfer into muscle for the treatment ofmuscular dystrophy and haemophilia
【24h】

Gene transfer into muscle for the treatment ofmuscular dystrophy and haemophilia

机译:基因转移到肌肉中治疗肌肉营养不良和血友病

获取原文
           

摘要

Muscle has proven to be an appropriate expression system for genes, the product of which is required in the general circulation as well as for muscle genes per se. This review deals with the design of the gene constructs including the vectors and the regulatory elements required for optimisation of expression following introduction of the relevant cDNA by intramuscular injection. The relative merits and problems associated with each type of vector including the immunogenic responses they elicit are discussed. Duchenne muscular dystrophy is used to illustrate the problems associated with gene therapy for a disease in which a muscle protein is defective or missing whilst haemophilia is chosen as an example of how a systemic protein, Factor VIII or IX, may be produced at low constitutive levels in muscle rather than liver.
机译:事实证明,肌肉是基因的合适表达系统,而基因产物本身是大体循环所必需的。这篇综述涉及基因构建体的设计,包括通过肌肉注射引入相关cDNA后优化表达最优化所需的载体和调控元件。讨论了与每种类型载体有关的相对优缺点,包括它们引起的免疫原性应答。 Duchenne肌营养不良症用于说明与基因疗法有关的疾病的问题,在该疾病中,肌肉蛋白有缺陷或缺失,而选择血友病作为以低组成水平生产全身性蛋白VIII或IX的例子在肌肉而不是肝脏。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱:kefu@zhangqiaokeyan.com

京公网安备:11010802029741号 ICP备案号:京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有
  • 客服微信

  • 服务号