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Results: 1 to 20 of 235

1.

Investigational therapies for the treatment of spinal muscular atrophy.

Kaczmarek A, Schneider S, Wirth B, Riessland M.

Expert Opin Investig Drugs. 2015 Apr 24:1-15. [Epub ahead of print]

PMID:
25911060
2.

Antisense oligonucleotides in therapy for neurodegenerative disorders.

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

Adv Drug Deliv Rev. 2015 Mar 20. pii: S0169-409X(15)00043-5. doi: 10.1016/j.addr.2015.03.008. [Epub ahead of print] Review.

3.

RNA-targeted Therapeutics for ALS.

Reddy LV, Miller TM.

Neurotherapeutics. 2015 Apr;12(2):424-7. doi: 10.1007/s13311-015-0344-z.

PMID:
25753730
4.

[Gene silencing approaches for the treatment of Huntington's disease].

Merienne N, Déglon N.

Med Sci (Paris). 2015 Feb;31(2):159-67. doi: 10.1051/medsci/20153102012. Epub 2015 Mar 4. Review. French.

PMID:
25744262
5.

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
6.

A large animal model of spinal muscular atrophy and correction of phenotype.

Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH.

Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9.

PMID:
25516063
7.

[The role of RNA splicing in the pathogenesis of spinal muscular atrophy and development of its therapeutics].

Sahashi K, Sobue G.

Brain Nerve. 2014 Dec;66(12):1471-80. doi: 10.11477/mf.1416200061. Review. Japanese.

PMID:
25475034
8.

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
9.

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
10.

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
11.

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
12.

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.

13.

Epigenomic networking in drug development: from pathogenic mechanisms to pharmacogenomics.

Cacabelos R.

Drug Dev Res. 2014 Sep;75(6):348-65. doi: 10.1002/ddr.21219.

PMID:
25195579
14.

C9orf72 amyotrophic lateral sclerosis and frontotemporal dementia: gain or loss of function?

Mizielinska S, Isaacs AM.

Curr Opin Neurol. 2014 Oct;27(5):515-23. doi: 10.1097/WCO.0000000000000130. Review.

15.

Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing.

Aronin N, DiFiglia M.

Mov Disord. 2014 Sep 15;29(11):1455-61. doi: 10.1002/mds.26020. Epub 2014 Aug 27. Review.

PMID:
25164989
16.

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. Review.

17.

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
18.

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.

19.

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
20.

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
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