慢病毒介导的小干扰RNA基因转移用于治疗亨廷顿舞蹈病
Lentiviral-mediated gene transfer of siRNAs for the treatment of Huntington's disease.
作者信息
Cambon Karine, Déglon Nicole
机构信息
Molecular Imaging Research Center, Commissariat à l'Énergie Atomique, Fontenay-aux-Roses, France.
出版信息
Methods Mol Biol. 2013;1010:95-109. doi: 10.1007/978-1-62703-411-1_7.
PMID:23754221
Abstract
This chapter describes the potential use of viral-mediated gene transfer in the central nervous system for the silencing of gene expression using RNA interference in the context of Huntington's disease (HD). Protocols provided here describe the design of small interfering RNAs, their encoding in lentiviral vectors (LVs) and viral production, as well as procedures for their stereotaxic injection in the rodent brain.
摘要
本章描述了在亨廷顿舞蹈症（HD）背景下，利用病毒介导的基因转移在中枢神经系统中通过RNA干扰实现基因表达沉默的潜在用途。这里提供的方案描述了小干扰RNA的设计、其在慢病毒载体（LVs）中的编码及病毒生产，以及将其立体定向注射到啮齿动物大脑中的程序。
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