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Results: 1 to 20 of 209

1.

An insight into advances in the pathogenesis and therapeutic strategies of spinocerebellar ataxia type 3.

Wu Y, Peng Y, Wang Y.

Rev Neurosci. 2014 Sep 12. pii: /j/revneuro.ahead-of-print/revneuro-2014-0040/revneuro-2014-0040.xml. doi: 10.1515/revneuro-2014-0040. [Epub ahead of print]

PMID:
25222595
[PubMed - as supplied by publisher]
2.

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all huntington disease patients.

Skotte NH, Southwell AL, Ostergaard ME, Carroll JB, Warby SC, Doty CN, Petoukhov E, Vaid K, Kordasiewicz H, Watt AT, Freier SM, Hung G, Seth PP, Bennett CF, Swayze EE, Hayden MR.

PLoS One. 2014 Sep 10;9(9):e107434. doi: 10.1371/journal.pone.0107434. eCollection 2014.

PMID:
25207939
[PubMed - in process]
Free PMC Article
3.

Targets for future clinical trials in Huntington's disease: What's in the pipeline?

Wild EJ, Tabrizi SJ.

Mov Disord. 2014 Sep 15;29(11):1434-45. doi: 10.1002/mds.26007. Epub 2014 Aug 25.

PMID:
25155142
[PubMed - in process]
4.

In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.

Southwell AL, Skotte NH, Kordasiewicz HB, Ostergaard ME, Watt AT, Carroll JB, Doty CN, Villanueva EB, Petoukhov E, Vaid K, Xie Y, Freier SM, Swayze EE, Seth PP, Bennett CF, Hayden MR.

Mol Ther. 2014 Aug 7. doi: 10.1038/mt.2014.153. [Epub ahead of print]

PMID:
25101598
[PubMed - as supplied by publisher]
5.

Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy For Tauopathies.

Sud R, Geller ET, Schellenberg GD.

Mol Ther Nucleic Acids. 2014 Jul 29;3:e180. doi: 10.1038/mtna.2014.30.

PMID:
25072694
[PubMed]
Free PMC Article
6.

Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

Rigo F, Chun SJ, Norris DA, Hung G, Lee S, Matson J, Fey RA, Gaus H, Hua Y, Grundy JS, Krainer AR, Henry SP, Bennett CF.

J Pharmacol Exp Ther. 2014 Jul;350(1):46-55. doi: 10.1124/jpet.113.212407. Epub 2014 Apr 30.

PMID:
24784568
[PubMed - indexed for MEDLINE]
7.

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 Sep 15;23(18):4832-45. doi: 10.1093/hmg/ddu198. Epub 2014 Apr 29.

PMID:
24781211
[PubMed - in process]
8.

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]
9.

Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases.

Veltrop M, Aartsma-Rus A.

Exp Cell Res. 2014 Jul 1;325(1):50-5. doi: 10.1016/j.yexcr.2014.01.026. Epub 2014 Jan 31. Review.

PMID:
24486759
[PubMed - indexed for MEDLINE]
10.

Presymptomatic treatment with acetylcholinesterase antisense oligonucleotides prolongs survival in ALS (G93A-SOD1) mice.

Marc G, Leah R, Ofira E, Oded A, Zohar A, Hanna R.

Biomed Res Int. 2013;2013:845345. doi: 10.1155/2013/845345. Epub 2013 Dec 22.

PMID:
24455732
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

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]
12.

Biodistribution and molecular studies on orally administered nanoparticle-AON complexes encapsulated with alginate aiming at inducing dystrophin rescue in mdx mice.

Falzarano MS, Passarelli C, Bassi E, Fabris M, Perrone D, Sabatelli P, Maraldi NM, Donà S, Selvatici R, Bonaldo P, Sparnacci K, Laus M, Braghetta P, Rimessi P, Ferlini A.

Biomed Res Int. 2013;2013:527418. doi: 10.1155/2013/527418. Epub 2013 Dec 12.

PMID:
24392452
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

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]
14.

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]
15.

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
16.

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. Review.

PMID:
24140266
[PubMed - indexed for MEDLINE]
17.

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
18.

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]
19.

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]
20.

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]

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