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G2019S-LRRK2 Expression Augments α-Synuclein Sequestration into Inclusions in Neurons.

Volpicelli-Daley LA, Abdelmotilib H, Liu Z, Stoyka L, Daher JP, Milnerwood AJ, Unni VK, Hirst WD, Yue Z, Zhao HT, Fraser K, Kennedy RE, West AB.

J Neurosci. 2016 Jul 13;36(28):7415-27. doi: 10.1523/JNEUROSCI.3642-15.2016.


Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.

Osman EY, Washington CW 3rd, Kaifer KA, Mazzasette C, Patitucci TN, Florea KM, Simon ME, Ko CP, Ebert AD, Lorson CL.

Mol Ther. 2016 Jul 12. doi: 10.1038/mt.2016.145. [Epub ahead of print]


Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.

Shimizu-Motohashi Y, Miyatake S, Komaki H, Takeda S, Aoki Y.

Am J Transl Res. 2016 Jun 15;8(6):2471-89. eCollection 2016. Review.


The long non-coding RNAs in neurodegenerative diseases: novel mechanisms of pathogenesis.

Riva P, Ratti A, Venturin M.

Curr Alzheimer Res. 2016 Jun 22. [Epub ahead of print]


C9orf72 BAC Mouse Model with Motor Deficits and Neurodegenerative Features of ALS/FTD.

Liu Y, Pattamatta A, Zu T, Reid T, Bardhi O, Borchelt DR, Yachnis AT, Ranum LP.

Neuron. 2016 May 4;90(3):521-34. doi: 10.1016/j.neuron.2016.04.005. Epub 2016 Apr 21.


Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.

Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C.

Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21.


Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.

Geraets RD, Koh Sy, Hastings ML, Kielian T, Pearce DA, Weimer JM.

Orphanet J Rare Dis. 2016 Apr 16;11:40. doi: 10.1186/s13023-016-0414-2. Review.


The first crystal structures of RNA-PNA duplexes and a PNA-PNA duplex containing mismatches--toward anti-sense therapy against TREDs.

Kiliszek A, Banaszak K, Dauter Z, Rypniewski W.

Nucleic Acids Res. 2016 Feb 29;44(4):1937-43. doi: 10.1093/nar/gkv1513. Epub 2015 Dec 29.


Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice.

Peters OM, Cabrera GT, Tran H, Gendron TF, McKeon JE, Metterville J, Weiss A, Wightman N, Salameh J, Kim J, Sun H, Boylan KB, Dickson D, Kennedy Z, Lin Z, Zhang YJ, Daughrity L, Jung C, Gao FB, Sapp PC, Horvitz HR, Bosco DA, Brown SP, de Jong P, Petrucelli L, Mueller C, Brown RH Jr.

Neuron. 2015 Dec 2;88(5):902-9. doi: 10.1016/j.neuron.2015.11.018.


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. 2016 Apr 15;25(R1):R53-64. doi: 10.1093/hmg/ddv442. Epub 2015 Oct 26. Review.


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. Review.


Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy.

Bogdanik LP, Osborne MA, Davis C, Martin WP, Austin A, Rigo F, Bennett CF, Lutz CM.

Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5863-72. doi: 10.1073/pnas.1509758112. Epub 2015 Oct 12.


Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis.

Niemietz C, Chandhok G, Schmidt H.

Molecules. 2015 Sep 30;20(10):17944-75. doi: 10.3390/molecules201017944. Review.


Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy.

Singh NN, Lee BM, DiDonato CJ, Singh RN.

Future Med Chem. 2015;7(13):1793-808. doi: 10.4155/fmc.15.101. Epub 2015 Sep 18. Review.


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.


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.


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.


Advances and challenges in developing a therapy for spinal muscular atrophy.

Anderton RS, Mastaglia FL.

Expert Rev Neurother. 2015;15(8):895-908. doi: 10.1586/14737175.2015.1059757. Review.


Developing therapies for spinal muscular atrophy.

Wertz MH, Sahin M.

Ann N Y Acad Sci. 2016 Feb;1366(1):5-19. doi: 10.1111/nyas.12813. Epub 2015 Jul 14.


Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.

Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S.

Sci Rep. 2015 Jun 30;5:11746. doi: 10.1038/srep11746.

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