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Items: 1 to 20 of 149

1.

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform.

Bennett CF, Swayze EE.

Annu Rev Pharmacol Toxicol. 2010;50:259-93. doi: 10.1146/annurev.pharmtox.010909.105654. Review.

PMID:
20055705
2.

Expanded CAG repeats in the crosshairs.

Aronin N.

Nat Biotechnol. 2009 May;27(5):451-2. doi: 10.1038/nbt0509-451. No abstract available.

PMID:
19430450
3.

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

Hu J, Matsui M, Gagnon KT, Schwartz JC, Gabillet S, Arar K, Wu J, Bezprozvanny I, Corey DR.

Nat Biotechnol. 2009 May;27(5):478-84. doi: 10.1038/nbt.1539. Epub 2009 May 3.

4.

Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.

Pfister EL, Kennington L, Straubhaar J, Wagh S, Liu W, DiFiglia M, Landwehrmeyer B, Vonsattel JP, Zamore PD, Aronin N.

Curr Biol. 2009 May 12;19(9):774-8. doi: 10.1016/j.cub.2009.03.030. Epub 2009 Apr 9.

5.

Sustained effects of nonallele-specific Huntingtin silencing.

Drouet V, Perrin V, Hassig R, Dufour N, Auregan G, Alves S, Bonvento G, Brouillet E, Luthi-Carter R, Hantraye P, D├ęglon N.

Ann Neurol. 2009 Mar;65(3):276-85. doi: 10.1002/ana.21569.

PMID:
19334076
6.

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.

Lombardi MS, Jaspers L, Spronkmans C, Gellera C, Taroni F, Di Maria E, Donato SD, Kaemmerer WF.

Exp Neurol. 2009 Jun;217(2):312-9. doi: 10.1016/j.expneurol.2009.03.004. Epub 2009 Mar 13.

PMID:
19289118
7.

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup.

Warby SC, Montpetit A, Hayden AR, Carroll JB, Butland SL, Visscher H, Collins JA, Semaka A, Hudson TJ, Hayden MR.

Am J Hum Genet. 2009 Mar;84(3):351-66. doi: 10.1016/j.ajhg.2009.02.003. Epub 2009 Feb 26.

8.

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi.

McBride JL, Boudreau RL, Harper SQ, Staber PD, Monteys AM, Martins I, Gilmore BL, Burstein H, Peluso RW, Polisky B, Carter BJ, Davidson BL.

Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5868-73. doi: 10.1073/pnas.0801775105. Epub 2008 Apr 8.

9.

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.

DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DW, Zamore PD, Aronin N.

Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9. Epub 2007 Oct 16.

10.

Transvascular delivery of small interfering RNA to the central nervous system.

Kumar P, Wu H, McBride JL, Jung KE, Kim MH, Davidson BL, Lee SK, Shankar P, Manjunath N.

Nature. 2007 Jul 5;448(7149):39-43. Epub 2007 Jun 17.

PMID:
17572664
11.

RNAi silencing in mouse models of neurodegenerative diseases.

Farah MH.

Curr Drug Deliv. 2007 Apr;4(2):161-7. Review.

PMID:
17456035
12.

Evaluation of locked nucleic acid-modified small interfering RNA in vitro and in vivo.

Mook OR, Baas F, de Wissel MB, Fluiter K.

Mol Cancer Ther. 2007 Mar;6(3):833-43.

13.

Strategies for silencing human disease using RNA interference.

Kim DH, Rossi JJ.

Nat Rev Genet. 2007 Mar;8(3):173-84. Review.

PMID:
17304245
14.

Huntingtin inhibits caspase-3 activation.

Zhang Y, Leavitt BR, van Raamsdonk JM, Dragatsis I, Goldowitz D, MacDonald ME, Hayden MR, Friedlander RM.

EMBO J. 2006 Dec 13;25(24):5896-906. Epub 2006 Nov 23.

15.

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

Schwarz DS, Ding H, Kennington L, Moore JT, Schelter J, Burchard J, Linsley PS, Aronin N, Xu Z, Zamore PD.

PLoS Genet. 2006 Sep 8;2(9):e140. Epub 2006 Jul 24.

16.

Antisense oligonucleotide therapy for neurodegenerative disease.

Smith RA, Miller TM, Yamanaka K, Monia BP, Condon TP, Hung G, Lobsiger CS, Ward CM, McAlonis-Downes M, Wei H, Wancewicz EV, Bennett CF, Cleveland DW.

J Clin Invest. 2006 Aug;116(8):2290-6. Epub 2006 Jul 27.

17.

Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras.

McNamara JO 2nd, Andrechek ER, Wang Y, Viles KD, Rempel RE, Gilboa E, Sullenger BA, Giangrande PH.

Nat Biotechnol. 2006 Aug;24(8):1005-15. Epub 2006 Jun 25.

PMID:
16823371
18.

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.

Grimm D, Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR, Marion P, Salazar F, Kay MA.

Nature. 2006 May 25;441(7092):537-41.

PMID:
16724069
19.

Antisense oligonucleotides: from design to therapeutic application.

Chan JH, Lim S, Wong WS.

Clin Exp Pharmacol Physiol. 2006 May-Jun;33(5-6):533-40. Review.

PMID:
16700890
20.

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse.

Machida Y, Okada T, Kurosawa M, Oyama F, Ozawa K, Nukina N.

Biochem Biophys Res Commun. 2006 Apr 28;343(1):190-7. Epub 2006 Mar 3.

PMID:
16530728

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