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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.

Borel F, Gernoux G, Cardozo B, Metterville JP, Toro Cabreja GC, Song L, Su Q, Gao GP, Elmallah MK, Brown RH Jr, Mueller C.

Hum Gene Ther. 2016 Jan;27(1):19-31. doi: 10.1089/hum.2015.122.


Paradoxical Roles of Antioxidant Enzymes: Basic Mechanisms and Health Implications.

Lei XG, Zhu JH, Cheng WH, Bao Y, Ho YS, Reddi AR, Holmgren A, Arnér ES.

Physiol Rev. 2016 Jan;96(1):307-64. doi: 10.1152/physrev.00010.2014. Review.


A Tandem Oligonucleotide Approach for SNP-Selective RNA Degradation Using Modified Antisense Oligonucleotides.

Magner D, Biala E, Lisowiec-Wachnicka J, Kierzek E, Kierzek R.

PLoS One. 2015 Nov 6;10(11):e0142139. doi: 10.1371/journal.pone.0142139. eCollection 2015 Nov 6. Erratum in: PLoS One. 2016;11(9):e0163575.


Antisense gene silencing: therapy for neurodegenerative disorders?

Nielsen TT, Nielsen JE.

Genes (Basel). 2013 Sep 10;4(3):457-84. doi: 10.3390/genes4030457.


Disease-causing allele-specific silencing by RNA interference.

Hohjoh H.

Pharmaceuticals (Basel). 2013 Apr 11;6(4):522-35. doi: 10.3390/ph6040522.


Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis.

Wang H, Yang B, Qiu L, Yang C, Kramer J, Su Q, Guo Y, Brown RH Jr, Gao G, Xu Z.

Hum Mol Genet. 2014 Feb 1;23(3):668-81. doi: 10.1093/hmg/ddt454. Epub 2013 Sep 18.


Recent advances in RNA interference therapeutics for CNS diseases.

Ramachandran PS, Keiser MS, Davidson BL.

Neurotherapeutics. 2013 Jul;10(3):473-85. doi: 10.1007/s13311-013-0183-8. Review.


Neuronal exosomal miRNA-dependent translational regulation of astroglial glutamate transporter GLT1.

Morel L, Regan M, Higashimori H, Ng SK, Esau C, Vidensky S, Rothstein J, Yang Y.

J Biol Chem. 2013 Mar 8;288(10):7105-16. doi: 10.1074/jbc.M112.410944. Epub 2013 Jan 30.


Neuroprotection for amyotrophic lateral sclerosis: role of stem cells, growth factors, and gene therapy.

Pandya RS, Mao LL, Zhou EW, Bowser R, Zhu Z, Zhu Y, Wang X.

Cent Nerv Syst Agents Med Chem. 2012 Mar;12(1):15-27. Review.


Restoration of normal BMP signaling levels and osteogenic differentiation in FOP mesenchymal progenitor cells by mutant allele-specific targeting.

Kaplan J, Kaplan FS, Shore EM.

Gene Ther. 2012 Jul;19(7):786-90. doi: 10.1038/gt.2011.152. Epub 2011 Oct 20.


Efficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi.

de Yñigo-Mojado L, Martín-Ruíz I, Sutherland JD.

PLoS One. 2011;6(6):e21352. doi: 10.1371/journal.pone.0021352. Epub 2011 Jun 21.


A comprehensive assessment of the SOD1G93A low-copy transgenic mouse, which models human amyotrophic lateral sclerosis.

Acevedo-Arozena A, Kalmar B, Essa S, Ricketts T, Joyce P, Kent R, Rowe C, Parker A, Gray A, Hafezparast M, Thorpe JR, Greensmith L, Fisher EM.

Dis Model Mech. 2011 Sep;4(5):686-700. doi: 10.1242/dmm.007237. Epub 2011 May 2.


TDP-43 potentiates alpha-synuclein toxicity to dopaminergic neurons in transgenic mice.

Tian T, Huang C, Tong J, Yang M, Zhou H, Xia XG.

Int J Biol Sci. 2011 Mar 4;7(2):234-43.


Genetic therapy for the nervous system.

Bowers WJ, Breakefield XO, Sena-Esteves M.

Hum Mol Genet. 2011 Apr 15;20(R1):R28-41. doi: 10.1093/hmg/ddr110. Epub 2011 Mar 23. Review.


Transgenic rat model of neurodegeneration caused by mutation in the TDP gene.

Zhou H, Huang C, Chen H, Wang D, Landel CP, Xia PY, Bowser R, Liu YJ, Xia XG.

PLoS Genet. 2010 Mar 26;6(3):e1000887. doi: 10.1371/journal.pgen.1000887.


Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype.

Scholefield J, Greenberg LJ, Weinberg MS, Arbuthnot PB, Abdelgany A, Wood MJ.

PLoS One. 2009 Sep 30;4(9):e7232. doi: 10.1371/journal.pone.0007232.


Huntington's disease: silencing a brutal killer.

Pfister EL, Zamore PD.

Exp Neurol. 2009 Dec;220(2):226-9. doi: 10.1016/j.expneurol.2009.09.017. Epub 2009 Sep 25. Review. No abstract available.


Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses.

Kaidonis X, Liaw WC, Roberts AD, Ly M, Anson D, Byers S.

Eur J Hum Genet. 2010 Feb;18(2):194-9. doi: 10.1038/ejhg.2009.143. Epub 2009 Aug 19.


Allele-specific RNAi mitigates phenotypic progression in a transgenic model of Alzheimer's disease.

Rodríguez-Lebrón E, Gouvion CM, Moore SA, Davidson BL, Paulson HL.

Mol Ther. 2009 Sep;17(9):1563-73. doi: 10.1038/mt.2009.123. Epub 2009 Jun 16.

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