Severe & Rare / Neurodegenerative

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.