RNase H-Mediated Degradation of Toxic RNA in Myotonic Dystrophy Type 1
Proc Natl Acad Sci U S A. 2012 Mar 13;109(11):4221-6
Genomic Analysis of wig-1 Pathways
PLoS One. 2012;7(2):e29429
Intracerebral Infusion of Antisense Oligonucleotides Into Prion-infected Mice
Mol Ther Nucleic Acids. 2012 Feb 7;1:e9
Mol Ther. 2011 Dec;19(12):2178-85. doi: 10.1038/mt.2011.201
Nature. 2011 Oct 5;478(7367):123-6. doi: 10.1038/nature10485.
Amyloid. 2011 Sep;18(3):136-46
Long pre-mRNA Depletion and RNA Missplicing Contribute to Neuronal Vulnerability from Loss of TDP-43
Nat Neurosci. 2011 Apr;14(4):459-68
Allele-selective Inhibition of Ataxin-3 (ATX3) Expression by Antisense Oligomers and Duplex RNAs
Biol Chem. 2011 Apr;392(4):315-25
Sci Transl Med. 2011 Mar 2;3(72):72ra18
Biochemistry. 2010 Nov 30;49(47):10166-78.
PLoS One. 2010 Sep 23;5(9):e12941
Antisense Correction of SMN2 Splicing in the CNS Rescues Necrosis in a Type III SMA Mouse Model
Genes Dev. 2010 Aug 1;24(15):1634-44
Neuroscience. 2005;131(3):705-15.
Neuroscience. 2008 Jun 26;154(3):1077-87.
Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon.
PLOS Biol. 2007 April;5(4):e73.
Antisense oligonucleotide therapy for neurodegenerative disease.
J Clin Invest. 2006 Aug;116(8):2290-6. Epub 2006 Jul 27.
ISIS-SMNRx Phase 1 Study in Patients With Spinal Muscular Atrophy Presented at American Academy of Neurology
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