Expanded CAG repeats in the crosshairs

被引：1

作者：

Aronin, Neil ^{[1
]}

机构：

[1] Univ Massachusetts, Sch Med, Worcester, MA 01605 USA

来源：

NATURE BIOTECHNOLOGY | 2009年 / 27卷 / 05期

关键词：

HUNTINGTONS-DISEASE; ALLELE;

D O I：

10.1038/nbt0509-451

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 [微生物学]; 090105 [作物生产系统与生态工程];

摘要：

Antisense oligomers targeted to CAG repeats allow allele-specific knockdown of the gene that causes Huntington's disease.

引用

页码：451 / 452

页数：2

共 9 条

[1]

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease [J].

Alves, Sandro ;

Nascimento-Ferreira, Isabel ;

Auregan, Gwennaelle ;

Hassig, Raymonde ;

Dufour, Noelle ;

Brouillet, Emmanuel ;

de Lima, Maria C. Pedroso ;

Hantraye, Philippe ;

de Almeida, Luis Pereira ;

Deglon, Nicole .

PLOS ONE, 2008, 3 (10)

[2]

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs [J].

Hu, Jiaxin ;

Matsui, Masayuki ;

Gagnon, Keith T. ;

Schwartz, Jacob C. ;

Gabillet, Sylvie ;

Arar, Khalil ;

Wu, Jun ;

Bezprozvanny, Ilya ;

Corey, David R. .

NATURE BIOTECHNOLOGY, 2009, 27 (05) :478-484

[3]

Miller VM, 2003, P NATL ACAD SCI USA, V100, P7195, DOI 10.1073/pnas.1231012100

[4]

Trinucleotide repeat disorders [J].

Orr, Harry T. ;

Zoghbi, Huda Y. .

ANNUAL REVIEW OF NEUROSCIENCE, 2007, 30 :575-621

[5]

PFISTER EL, CURR BIOL

[6]

Designing siRNA that distinguish between genes that differ by a single nucleotide [J].

Schwarz, Dianne S. ;

Ding, Hongliu ;

Kennington, Lori ;

Moore, Jessica T. ;

Schelter, Janell ;

Burchard, Julja ;

Linsley, Peter S. ;

Aronin, Neil ;

Xu, Zuoshang ;

Zamore, Phillip D. .

PLOS GENETICS, 2006, 2 (09) :1307-1318

[7]

RELATIONSHIP BETWEEN TRINUCLEOTIDE REPEAT EXPANSION AND PHENOTYPIC VARIATION IN HUNTINGTONS-DISEASE [J].

SNELL, RG ;

MACMILLAN, JC ;

CHEADLE, JP ;

FENTON, I ;

LAZAROU, LP ;

DAVIES, P ;

MACDONALD, ME ;

GUSELLA, JF ;

HARPER, PS ;

SHAW, DJ .

NATURE GENETICS, 1993, 4 (04) :393-397

[8]

Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts [J].

van Bilsen, P. H. J. ;

Jaspers, L. ;

Lombardi, M. S. ;

Odekerken, J. C. E. ;

Burright, E. N. ;

Kaemmerer, W. F. .

HUMAN GENE THERAPY, 2008, 19 (07) :710-718

[9]

Allele-specific silencing of mutant Huntington's disease gene [J].

Zhang, Yu ;

Engelman, Joshua ;

Friedlander, Robert M. .

JOURNAL OF NEUROCHEMISTRY, 2009, 108 (01) :82-90

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