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Results: 1 to 20 of 200

1.

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.

Osman EY, Miller MR, Robbins KL, Lombardi AM, Atkinson AK, Brehm AJ, Lorson CL.

Hum Mol Genet. 2014 Apr 29. pii: ddu198. [Epub ahead of print]

PMID:
24781211
[PubMed - as supplied by publisher]
2.

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]
3.

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA).

Zanetta C, Riboldi G, Nizzardo M, Simone C, Faravelli I, Bresolin N, Comi GP, Corti S.

J Cell Mol Med. 2014 Feb;18(2):187-96. doi: 10.1111/jcmm.12224. Epub 2014 Jan 8. Review.

PMID:
24400925
[PubMed - indexed for MEDLINE]
4.

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]
5.

Neurodegenerative disease: C9orf72 RNA foci--a therapeutic target for ALS and FTD?

Wood H.

Nat Rev Neurol. 2013 Dec;9(12):659. doi: 10.1038/nrneurol.2013.244. Epub 2013 Nov 26. No abstract available.

PMID:
24275931
[PubMed - indexed for MEDLINE]
6.

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]
Free PMC Article
7.

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]
8.

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

Donnelly CJ, Zhang PW, Pham JT, Haeusler 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. Erratum in: Neuron. 2013 Nov 20;80(4):1102. Heusler, Aaron R [corrected to Haeusler, Aaron R].

PMID:
24139042
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

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]
10.

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. Review.

PMID:
24068583
[PubMed - indexed for MEDLINE]
11.

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]
12.

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]
13.

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
14.

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
15.

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
16.

[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]
17.

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]
18.

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
19.

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]
20.

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]

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