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Results: 1 to 20 of 242

1.

Huntingtin Haplotypes Provide Prioritized Target Panels for Allele Specific Silencing in Huntington Disease Patients of European Ancestry.

Kay C, Collins JA, Skotte NH, Southwell AL, Warby SC, Caron NS, Doty CN, Nguyen B, Griguoli A, Ross CJ, Squitieri F, Hayden MR.

Mol Ther. 2015 Jul 23. doi: 10.1038/mt.2015.128. [Epub ahead of print]

PMID:
26201449
2.

Developing therapies for spinal muscular atrophy.

Wertz MH, Sahin M.

Ann N Y Acad Sci. 2015 Jul 14. doi: 10.1111/nyas.12813. [Epub ahead of print]

PMID:
26173388
3.

Investigational therapies for the treatment of spinal muscular atrophy.

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

Expert Opin Investig Drugs. 2015 Jul;24(7):867-81. doi: 10.1517/13543784.2015.1038341. Epub 2015 Apr 24.

PMID:
25911060
4.

Antisense oligonucleotides in therapy for neurodegenerative disorders.

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

Adv Drug Deliv Rev. 2015 Jun 29;87:90-103. doi: 10.1016/j.addr.2015.03.008. Epub 2015 Mar 20. Review.

5.

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
6.

Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.

Wojtkowiak-Szlachcic A, Taylor K, Stepniak-Konieczna E, Sznajder LJ, Mykowska A, Sroka J, Thornton CA, Sobczak K.

Nucleic Acids Res. 2015 Mar 31;43(6):3318-31. doi: 10.1093/nar/gkv163. Epub 2015 Mar 9.

7.

[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
8.

Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.

Singh NN, Lee BM, Singh RN.

Ann N Y Acad Sci. 2015 Apr;1341:176-87. doi: 10.1111/nyas.12727. Epub 2015 Feb 27.

PMID:
25727246
9.

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
10.

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
11.

[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
12.

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
13.

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. Erratum in: Ann Neurol. 2015 Jun;77(6):1085.

PMID:
25381879
14.

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
15.

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
16.

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.

17.

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
18.

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.

19.

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
20.

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.

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