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Results: 1 to 20 of 228

1.

Antisense oligonucleotides in therapy for neurodegenerative disorders.

Evers MM, Toonen LJ, van Roon-Mom WM.

Adv Drug Deliv Rev. 2015 Mar 19. pii: S0169-409X(15)00043-5. doi: 10.1016/j.addr.2015.03.008. [Epub ahead of print] Review.

2.

RNA-targeted Therapeutics for ALS.

Reddy LV, Miller TM.

Neurotherapeutics. 2015 Mar 10. [Epub ahead of print]

PMID:
25753730
3.

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.

Hua Y, Liu YH, Sahashi K, Rigo F, Bennett CF, Krainer AR.

Genes Dev. 2015 Feb 1;29(3):288-97. doi: 10.1101/gad.256644.114. Epub 2015 Jan 12.

PMID:
25583329
4.

Huntington's disease: an update of therapeutic strategies.

Kumar A, Kumar Singh S, Kumar V, Kumar D, Agarwal S, Rana MK.

Gene. 2015 Feb 10;556(2):91-7. doi: 10.1016/j.gene.2014.11.022. Epub 2014 Nov 12. Review.

PMID:
25447911
5.

Targeting miR-155 restores abnormal microglia and attenuates disease in SOD1 mice.

Butovsky O, Jedrychowski MP, Cialic R, Krasemann S, Murugaiyan G, Fanek Z, Greco DJ, Wu PM, Doykan CE, Kiner O, Lawson RJ, Frosch MP, Pochet N, Fatimy RE, Krichevsky AM, Gygi SP, Lassmann H, Berry J, Cudkowicz ME, Weiner HL.

Ann Neurol. 2015 Jan;77(1):75-99. doi: 10.1002/ana.24304. Epub 2014 Nov 27.

PMID:
25381879
6.

Spinal muscular atrophy: diagnosis and management in a new therapeutic era.

Arnold WD, Kassar D, Kissel JT.

Muscle Nerve. 2015 Feb;51(2):157-67. doi: 10.1002/mus.24497. Epub 2014 Dec 16. Review.

PMID:
25346245
7.

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
8.

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.

9.

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.

10.

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
11.

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.

12.

An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis.

Obici L, Merlini G.

Expert Opin Investig Drugs. 2014 Sep;23(9):1239-51. doi: 10.1517/13543784.2014.922541. Epub 2014 Jul 8. Review.

PMID:
25003808
13.

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
14.

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
15.

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
16.

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
17.

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
18.

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
19.

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
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