共 38 条
DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA•TTC repeats in Friedreich's ataxia
被引:108
作者:

Burnett, Ryan
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机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Melander, Christian
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h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Puckett, James W.
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h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Son, Leslie S.
论文数: 0 引用数: 0
h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Wells, Robert D.
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h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Dervan, Peter B.
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h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA

Gottesfeld, Joel M.
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h-index: 0
机构: Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA
机构:
[1] Scripps Res Inst, Dept Mol Biol, La Jolla, CA 92037 USA
[2] CALTECH, Div Chem & Chem Engn, Pasadena, CA 91125 USA
[3] Texas A&M Univ, Inst Biosci & Technol, Ctr Genome Res, Syst Hlth Sci Ctr, Houston, TX 77030 USA
来源:
关键词:
gene regulation;
triplet repeat expansion;
frataxin;
D O I:
10.1073/pnas.0604939103
中图分类号:
O [数理科学和化学];
P [天文学、地球科学];
Q [生物科学];
N [自然科学总论];
学科分类号:
07 ;
0710 ;
09 ;
摘要:
The DNA abnormality found in 98% of Friedreich's ataxia (FRDA) patients is the unstable hyperexpansion of a GAA(.)TTC triplet repeat in the first intron of the frataxin gene. Expanded GAA.TTC repeats result in decreased transcription and reduced levels of frataxin protein in affected individuals. P-Alanine-linked pyrrole-imidazole polyamides bind GAA(.)TTC tracts with high affinity and disrupt the intramolecular DNA-DNA-associated region of the sticky-DNA conformation formed by long GAA(.)TTC repeats. Fluorescent polyamide-Bodipy conjugates localize in the nucleus of a lymphoid cell line derived from a FRDA patient. The synthetic ligands increase transcription of the frataxin gene in cell culture, resulting in increased levels of frataxin protein. DNA microarray analyses indicate that a limited number of genes are significantly affected in FRDA cells. Polyamides may increase transcription by altering the DNA conformation of genes harboring long GAA(.)TTC repeats or by chromatin opening.
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页码:11497 / 11502
页数:6
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