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Results: 1 to 20 of 221

1.

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

Wu Y, Peng Y, Wang Y.

Rev Neurosci. 2015;26(1):95-104. doi: 10.1515/revneuro-2014-0040.

PMID:
25222595
[PubMed - in process]
2.

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

Skotte NH, Southwell AL, Østergaard 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]
Free PMC Article
4.

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

Southwell AL, Skotte NH, Kordasiewicz HB, Østergaard 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 Dec;22(12):2093-106. doi: 10.1038/mt.2014.153. Epub 2014 Aug 7.

PMID:
25101598
[PubMed - in process]
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.

The RNA-binding protein HuD promotes spinal GAP43 overexpression in antiretroviral-induced neuropathy.

Sanna MD, Quattrone A, Mello T, Ghelardini C, Galeotti N.

Exp Neurol. 2014 Nov;261:343-53. doi: 10.1016/j.expneurol.2014.05.017. Epub 2014 May 24.

PMID:
24861443
[PubMed - indexed for MEDLINE]
7.

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]
8.

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]
9.

Recent progress in the understanding and treatment of transthyretin amyloidosis.

Sekijima Y.

J Clin Pharm Ther. 2014 Jun;39(3):225-33. doi: 10.1111/jcpt.12145. Review.

PMID:
24749898
[PubMed - indexed for MEDLINE]
10.

Oligonucleotide-based therapy for neurodegenerative diseases.

Magen I, Hornstein E.

Brain Res. 2014 Oct 10;1584:116-28. doi: 10.1016/j.brainres.2014.04.005. Epub 2014 Apr 12.

PMID:
24727531
[PubMed - in process]
11.

Personalized gene silencing therapeutics for Huntington disease.

Kay C, Skotte NH, Southwell AL, Hayden MR.

Clin Genet. 2014 Jul;86(1):29-36. doi: 10.1111/cge.12385. Epub 2014 Apr 11. Review.

PMID:
24646433
[PubMed - indexed for MEDLINE]
12.

New developments in exon skipping and splice modulation therapies for neuromuscular diseases.

Touznik A, Lee JJ, Yokota T.

Expert Opin Biol Ther. 2014 Jun;14(6):809-19. doi: 10.1517/14712598.2014.896335. Epub 2014 Mar 12. Review.

PMID:
24620745
[PubMed - indexed for MEDLINE]
13.

Central and peripheral administration of antisense oligonucleotide targeting amyloid-β protein precursor improves learning and memory and reduces neuroinflammatory cytokines in Tg2576 (AβPPswe) mice.

Farr SA, Erickson MA, Niehoff ML, Banks WA, Morley JE.

J Alzheimers Dis. 2014;40(4):1005-16. doi: 10.3233/JAD-131883.

PMID:
24577464
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.

Siva K, Covello G, Denti MA.

Nucleic Acid Ther. 2014 Feb;24(1):69-86. doi: 10.1089/nat.2013.0461. Review.

PMID:
24506781
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

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]
16.

FAP neuropathy and emerging treatments.

Adams D, Théaudin M, Cauquil C, Algalarrondo V, Slama M.

Curr Neurol Neurosci Rep. 2014 Mar;14(3):435. doi: 10.1007/s11910-013-0435-3. Review.

PMID:
24482069
[PubMed - indexed for MEDLINE]
17.

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
18.

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]
Free PMC Article
19.

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
20.

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]
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