Polyamides alleviate transcription inhibition associated with long GAA•TTC repeats in Friedreich’s ataxia. Homo sapiens

Lymphoblast cells from a patient with Freidriech's Ataxia were incubated with pyrrole-imidazole polyamides targeted to the GAA triplet repeat in the intron 1. The polyamides were shown in cell culture to increase levels of endogenous frataxin mRNA. A normal sibling derived lymphoblast cell line was used as a control. Keywords: human lymphoblast cells Overall design: Normal (GM15851) and patient (GM15850) cell lines were incubated in the presence of match polyamide FA1 at 1uM, 2uM or mismatch polyamide FA2 at 2uM for 7days prior to RNA purification and microarray analysis.

将弗里德赖希共济失调（Friedreich's Ataxia）患者的淋巴母细胞与靶向内含子1（intron 1）中GAA三核苷酸重复序列（GAA triplet repeat）的吡咯-咪唑聚酰胺（pyrrole-imidazole polyamides）共孵育。经细胞培养实验验证，该聚酰胺可提升内源性frataxin信使核糖核酸（frataxin mRNA）的表达水平。本实验以正常同胞来源的淋巴母细胞系作为对照。
关键词：人淋巴母细胞
实验设计概述：将正常（GM15851）与患者（GM15850）来源的细胞系分别用1μM、2μM浓度的匹配型聚酰胺FA1，或2μM浓度的错配型聚酰胺FA2孵育7天，随后开展RNA纯化与微阵列分析（microarray analysis）。