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Results: 1 to 20 of 248

1.

Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy.

Sahashi K, Katsuno M, Hung G, Adachi H, Kondo N, Nakatsuji H, Tohnai G, Iida M, Bennett CF, Sobue G.

Hum Mol Genet. 2015 Jul 30. pii: ddv300. [Epub ahead of print]

PMID:
26231218
2.

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
3.

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
4.

Disease mechanisms and therapeutic approaches in spinal muscular atrophy.

Tisdale S, Pellizzoni L.

J Neurosci. 2015 Jun 10;35(23):8691-700. doi: 10.1523/JNEUROSCI.0417-15.2015. Review.

PMID:
26063904
5.

Investigational therapies for the treatment of spinal muscular atrophy.

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

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

PMID:
25911060
6.

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.

7.

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
8.

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.

9.

[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
10.

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
11.

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.

12.

[Therapeutic development in myotonic dystrophy].

Takahashi MP, Nakamori M, Mochizuki H.

Rinsho Shinkeigaku. 2014;54(12):1077-9. Review. Japanese.

PMID:
25519965
13.

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
14.

[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
15.

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
16.

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
17.

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
18.

Therapeutic applications of the cell-penetrating HIV-1 Tat peptide.

Rizzuti M, Nizzardo M, Zanetta C, Ramirez A, Corti S.

Drug Discov Today. 2015 Jan;20(1):76-85. doi: 10.1016/j.drudis.2014.09.017. Epub 2014 Sep 30. Review.

PMID:
25277319
19.

Delivering a disease-modifying treatment for Huntington's disease.

Godinho BM, Malhotra M, O'Driscoll CM, Cryan JF.

Drug Discov Today. 2015 Jan;20(1):50-64. doi: 10.1016/j.drudis.2014.09.011. Epub 2014 Sep 26. Review.

PMID:
25256777
20.

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
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