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Results: 1 to 20 of 196

1.

Oligonucleotide-based therapy for neurodegenerative diseases.

Magen I, Hornstein E.

Brain Res. 2014 Apr 12. pii: S0006-8993(14)00482-X. doi: 10.1016/j.brainres.2014.04.005. [Epub ahead of print]

PMID:
24727531
[PubMed - as supplied by publisher]
2.

Drugging unconventional targets: insights from Huntington's disease.

Yu S, Liang Y, Palacino J, Difiglia M, Lu B.

Trends Pharmacol Sci. 2014 Feb;35(2):53-62. doi: 10.1016/j.tips.2013.12.001. Epub 2014 Jan 2. Review.

PMID:
24388390
[PubMed - indexed for MEDLINE]
3.

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.

Lagier-Tourenne C, Baughn M, Rigo F, Sun S, Liu P, Li HR, Jiang J, Watt AT, Chun S, Katz M, Qiu J, Sun Y, Ling SC, Zhu Q, Polymenidou M, Drenner K, Artates JW, McAlonis-Downes M, Markmiller S, Hutt KR, Pizzo DP, Cady J, Harms MB, Baloh RH, Vandenberg SR, Yeo GW, Fu XD, Bennett CF, Cleveland DW, Ravits J.

Proc Natl Acad Sci U S A. 2013 Nov 19;110(47):E4530-9. doi: 10.1073/pnas.1318835110. Epub 2013 Oct 29.

PMID:
24170860
[PubMed - indexed for MEDLINE]
4.

Taking a risk: a therapeutic focus on ataxin-2 in amyotrophic lateral sclerosis?

van den Heuvel DM, Harschnitz O, van den Berg LH, Pasterkamp RJ.

Trends Mol Med. 2014 Jan;20(1):25-35. doi: 10.1016/j.molmed.2013.09.001. Epub 2013 Oct 16.

PMID:
24140266
[PubMed - in process]
5.

RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.

Donnelly CJ, Zhang PW, Pham JT, Heusler AR, Mistry NA, Vidensky S, Daley EL, Poth EM, Hoover B, Fines DM, Maragakis N, Tienari PJ, Petrucelli L, Traynor BJ, Wang J, Rigo F, Bennett CF, Blackshaw S, Sattler R, Rothstein JD.

Neuron. 2013 Oct 16;80(2):415-28. doi: 10.1016/j.neuron.2013.10.015.

PMID:
24139042
[PubMed - indexed for MEDLINE]
6.

In-silico analysis for RNA-interference mechanism of α-synuclein to treat Parkinson's disease.

Seema S, Seenivasagam R, Hemavathi K.

Int J Bioinform Res Appl. 2013;9(6):557-75. doi: 10.1504/IJBRA.2013.056630.

PMID:
24084237
[PubMed - indexed for MEDLINE]
7.

Epigenetics and ncRNAs in brain function and disease: mechanisms and prospects for therapy.

Varela MA, Roberts TC, Wood MJ.

Neurotherapeutics. 2013 Oct;10(4):621-31. doi: 10.1007/s13311-013-0212-7.

PMID:
24068583
[PubMed - in process]
8.

TigarB causes mitochondrial dysfunction and neuronal loss in PINK1 deficiency.

Flinn LJ, Keatinge M, Bretaud S, Mortiboys H, Matsui H, De Felice E, Woodroof HI, Brown L, McTighe A, Soellner R, Allen CE, Heath PR, Milo M, Muqit MM, Reichert AS, Köster RW, Ingham PW, Bandmann O.

Ann Neurol. 2013 Dec;74(6):837-47. doi: 10.1002/ana.23999.

PMID:
24027110
[PubMed - indexed for MEDLINE]
9.

Spinal muscular atrophy: an update on therapeutic progress.

Seo J, Howell MD, Singh NN, Singh RN.

Biochim Biophys Acta. 2013 Dec;1832(12):2180-90. doi: 10.1016/j.bbadis.2013.08.005. Epub 2013 Aug 27. Review.

PMID:
23994186
[PubMed - indexed for MEDLINE]
10.

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.

Østergaard ME, Southwell AL, Kordasiewicz H, Watt AT, Skotte NH, Doty CN, Vaid K, Villanueva EB, Swayze EE, Bennett CF, Hayden MR, Seth PP.

Nucleic Acids Res. 2013 Nov;41(21):9634-50. doi: 10.1093/nar/gkt725. Epub 2013 Aug 19.

PMID:
23963702
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

Ultrasound-enhanced delivery of morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice.

Koebis M, Kiyatake T, Yamaura H, Nagano K, Higashihara M, Sonoo M, Hayashi Y, Negishi Y, Endo-Takahashi Y, Yanagihara D, Matsuda R, Takahashi MP, Nishino I, Ishiura S.

Sci Rep. 2013;3:2242. doi: 10.1038/srep02242.

PMID:
23873129
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

An intronic structure enabled by a long-distance interaction serves as a novel target for splicing correction in spinal muscular atrophy.

Singh NN, Lawler MN, Ottesen EW, Upreti D, Kaczynski JR, Singh RN.

Nucleic Acids Res. 2013 Sep;41(17):8144-65. doi: 10.1093/nar/gkt609. Epub 2013 Jul 16.

PMID:
23861442
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

[Familial amyloid polyneuropathies: therapeutic issues].

Adams D, Cauquil C, Théaudin M.

Bull Acad Natl Med. 2012 Oct;196(7):1333-45; discussion 1345-7. Review. French.

PMID:
23815018
[PubMed - indexed for MEDLINE]
14.

Potential therapeutic applications of RNA cap analogs.

Ziemniak M, Strenkowska M, Kowalska J, Jemielity J.

Future Med Chem. 2013 Jun;5(10):1141-72. doi: 10.4155/fmc.13.96. Review.

PMID:
23795970
[PubMed - indexed for MEDLINE]
15.

Involvement of microRNA in microglia-mediated immune response.

Guedes J, Cardoso AL, Pedroso de Lima MC.

Clin Dev Immunol. 2013;2013:186872. doi: 10.1155/2013/186872. Epub 2013 May 23. Review.

PMID:
23762086
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice.

Koval ED, Shaner C, Zhang P, du Maine X, Fischer K, Tay J, Chau BN, Wu GF, Miller TM.

Hum Mol Genet. 2013 Oct 15;22(20):4127-35. doi: 10.1093/hmg/ddt261. Epub 2013 Jun 4.

PMID:
23740943
[PubMed - indexed for MEDLINE]
17.

The DcpS inhibitor RG3039 improves motor function in SMA mice.

Van Meerbeke JP, Gibbs RM, Plasterer HL, Miao W, Feng Z, Lin MY, Rucki AA, Wee CD, Xia B, Sharma S, Jacques V, Li DK, Pellizzoni L, Rusche JR, Ko CP, Sumner CJ.

Hum Mol Genet. 2013 Oct 15;22(20):4074-83. doi: 10.1093/hmg/ddt257. Epub 2013 May 31.

PMID:
23727836
[PubMed - indexed for MEDLINE]
18.

Antisense oligonucleotides: treating neurodegeneration at the level of RNA.

DeVos SL, Miller TM.

Neurotherapeutics. 2013 Jul;10(3):486-97. doi: 10.1007/s13311-013-0194-5. Review.

PMID:
23686823
[PubMed - indexed for MEDLINE]
19.

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon.

Evers MM, Tran HD, Zalachoras I, Pepers BA, Meijer OC, den Dunnen JT, van Ommen GJ, Aartsma-Rus A, van Roon-Mom WM.

Neurobiol Dis. 2013 Oct;58:49-56. doi: 10.1016/j.nbd.2013.04.019. Epub 2013 May 6.

PMID:
23659897
[PubMed - indexed for MEDLINE]
20.

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

Mitrpant C, Porensky P, Zhou H, Price L, Muntoni F, Fletcher S, Wilton SD, Burghes AH.

PLoS One. 2013 Apr 22;8(4):e62114. doi: 10.1371/journal.pone.0062114. Print 2013.

PMID:
23630626
[PubMed - indexed for MEDLINE]
Free PMC Article

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