Severe & Rare / Neurodegenerative

RNase H-Mediated Degradation of Toxic RNA in Myotonic Dystrophy Type 1

Lee JE, Bennett CF, Cooper TA

Proc Natl Acad Sci U S A. 2012 Mar 13;109(11):4221-6

Genomic Analysis of wig-1 Pathways

Sedaghat Y, Mazur C, Sabripour M, Hung G, Monia BP

PLoS One. 2012;7(2):e29429

Intracerebral Infusion of Antisense Oligonucleotides Into Prion-infected Mice

Friberg KN, Hung G, Wancewicz E, Giles K, Black C, Freier S, Bennett F, DeArmond SJ, Freyman Y, Lessard P, Ghaemmaghami S, Prusiner SB

Mol Ther Nucleic Acids. 2012 Feb 7;1:e9

Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene / Allele-Specific Silencing of Mutant Huntingtin

Carroll JB, Warby SC, Southwell AL, Doty CN, Greenlee S, Skotte N, Hung G, Bennett CF, Freier SM, Hayden MR

Mol Ther. 2011 Dec;19(12):2178-85. doi: 10.1038/mt.2011.201

Peripheral SMN Restoration is Essential for Long-Term Rescue of a Severe Spinal Muscular Atrophy Mouse Model

Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF, Krainer AR

Nature. 2011 Oct 5;478(7367):123-6. doi: 10.1038/nature10485.

Antisense Oligonucleotide Suppression of Serum Amyloid A Reduces Amyloid Deposition in Mice with AA Amyloidosis

Kluve-Beckerman B, Hardwick J, Du L, Benson MD, Monia BP, Watt A, Crooke RM, Mullick A

Amyloid. 2011 Sep;18(3):136-46

Long pre-mRNA Depletion and RNA Missplicing Contribute to Neuronal Vulnerability from Loss of TDP-43

Polymenidou M, Lagier-Tourenne C, Hutt KR, Huelga SC, Moran J, Liang TY, Ling SC, Sun E, Wancewicz E, Mazur C, Kordasiewicz H, Sedaghat Y, Donohue JP, Shiue L, Bennett CF, Yeo GW, Cleveland DW

Nat Neurosci. 2011 Apr;14(4):459-68

Allele-selective Inhibition of Ataxin-3 (ATX3) Expression by Antisense Oligomers and Duplex RNAs

Hu J, Gagnon KT, Liu J, Watts JK, Syeda-Nawaz J, Bennett CF, Swayze EE, Randolph J, Chattopadhyaya J, Corey DR

Biol Chem. 2011 Apr;392(4):315-25

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Passini MA, Bu J, Richards AM, Kinnecom C, Sardi SP, Stanek LM, Hua Y, Rigo F, Matson J, Hung G, Kaye EM, Shihabuddin LS, Krainer AR, Bennett CF, Cheng SH

Sci Transl Med. 2011 Mar 2;3(72):72ra18

Allele-Selective Inhibition of Mutant Huntingtin Expression with Antisense Oligonucleotides Targeting the Expanded CAG Repeat

Gagnon KT, Pendergraff HM, Deleavey GF, Swayze EE, Potier P, Randolph J, Roesch EB, Chattopadhyaya J, Damha MJ, Bennett CF, Montailler C, Lemaitre MM, Corey DR

Biochemistry. 2010 Nov 30;49(47):10166-78.

Evaluation of an Anti-Tumor Necrosis Factor Therapeutic in a Mouse Model of Niemann-Pick C Liver Disease

Vincent M, Sayre NL, Graham MJ, Crooke RM, Shealy DJ, Liscum L

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

Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR

Genes Dev. 2010 Aug 1;24(15):1634-44

Spinal Distribution and Metabolism of 2’-O-(2-methoxyethyl)-modified Oligonucleotides after Intrathecal Administration in Rats.

Butler M, Hayes CS, Chappell A, Murray SF, Yaksh TL, Hua XY.

Neuroscience. 2005;131(3):705-15.

Inhibition of Spinal Cytosolic Phospholipase A(2) Expression by an Antisense Oligonucleotide Attenuates Tissue Injury-induced Hyperalgesia.

Kim DH, Fitzsimmons B, Hefferan MP, Svensson CI, Wancewicz E, Monia BP, Hung G, Butler M, Marsala M, Hua XY, Yaksh TL .

Neuroscience. 2008 Jun 26;154(3):1077-87.

Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon.

Hua Y, Vickers TA, Baker BF, Bennett CF, Krainer AR.

PLOS Biol. 2007 April;5(4):e73.

Antisense oligonucleotide therapy for neurodegenerative disease.

Smith RA, Miller TM, Yamanaka K, Monia BP, Condon TP, Hung G, Lobsiger CS, Ward CM, McAlonis-Downes M, Wei H, Wancewicz EV, Bennett CF, Cleveland DW.

J Clin Invest. 2006 Aug;116(8):2290-6. Epub 2006 Jul 27.