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Items: 1 to 20 of 256

1.

Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.

Keiser MS, Kordasiewicz HB, McBride JL.

Hum Mol Genet. 2015 Oct 26. pii: ddv442. [Epub ahead of print] Review.

PMID:
26503961
2.

Clinical and genetic basis of familial amyotrophic lateral sclerosis.

Souza PV, Pinto WB, Chieia MA, Oliveira AS.

Arq Neuropsiquiatr. 2015 Dec;73(12):1026-37. doi: 10.1590/0004-282X20150161. Epub 2015 Oct 13.

3.

Identification and characterization of modified antisense oligonucleotides targeting DMPK in mice and nonhuman primates for the treatment of myotonic dystrophy type 1.

Pandey SK, Wheeler TM, Justice SL, Kim A, Younis HS, Gattis D, Jauvin D, Puymirat J, Swayze EE, Freier SM, Bennett CF, Thornton CA, MacLeod AR.

J Pharmacol Exp Ther. 2015 Nov;355(2):329-40. doi: 10.1124/jpet.115.226969. Epub 2015 Sep 1.

PMID:
26330536
4.

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 Nov 1;24(21):5985-94. doi: 10.1093/hmg/ddv300. Epub 2015 Jul 30.

PMID:
26231218
5.

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 Nov;23(11):1759-71. doi: 10.1038/mt.2015.128. Epub 2015 Jul 23.

PMID:
26201449
6.

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
7.

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.

8.

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
9.

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.

10.

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
11.

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.

12.

[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
13.

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
14.

Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7.

Staropoli JF, Li H, Chun SJ, Allaire N, Cullen P, Thai A, Fleet CM, Hua Y, Bennett CF, Krainer AR, Kerr D, McCampbell A, Rigo F, Carulli JP.

Genomics. 2015 Apr;105(4):220-8. doi: 10.1016/j.ygeno.2015.01.007. Epub 2015 Jan 31.

15.

Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments.

Sekijima Y.

J Neurol Neurosurg Psychiatry. 2015 Sep;86(9):1036-43. doi: 10.1136/jnnp-2014-308724. Epub 2015 Jan 20. Review.

PMID:
25604431
16.

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.

17.

[Therapeutic development in myotonic dystrophy].

Takahashi MP, Nakamori M, Mochizuki H.

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

PMID:
25519965
18.

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
19.

Liver transplantation in transthyretin amyloidosis: issues and challenges.

Carvalho A, Rocha A, Lobato L.

Liver Transpl. 2015 Mar;21(3):282-92. doi: 10.1002/lt.24058. Epub 2015 Feb 2. Review.

PMID:
25482846
20.

[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
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