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17 Article(s) on File
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Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models
Hua Y,
Liu YH,
Sahashi K,
Rigo F,
Bennett CF,
Krainer AR

Genes Dev
  (0): Jan 12 2015   Nlm
[Abstract]
[DOI] [Full Text]

Pharmacology of a central nervous system delivered 2′-O-methoxyethyl- modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
Rigo F,
Chun SJ,
Norris DA,
Hung G,
Lee S,
Matson J,
Fey RA,
Gaus H,
Hua Y,
Grundy JS,
Krainer AR,
Henry SP,
Bennett CF

Journal of Pharmacology and Experimental Therapeutics
  350 (1): 46-55; Jul 2014 2014   NLM
[Abstract]
[DOI] [Full Text]

Pathological impact of SMN2 mis-splicing in adult SMA mice
Sahashi K,
Ling KK,
Hua Y,
Wilkinson JE,
Nomakuchi T,
Rigo F,
Hung G,
Xu D,
Jiang YP,
Lin RZ,
Ko CP,
Bennett CF,
Krainer AR

EMBO Mol Med
  5 (10): 1586-601; Oct 2013   NLM
[Abstract]
[DOI] [Full Text]

Pathological impact of SMN2 mis-splicing in adult SMA mice
Sahashi K,
Ling KK,
Hua Y,
Wilkinson JE,
Nomakuchi T,
Rigo F,
Hung G,
Xu D,
Jiang YP,
Lin RZ,
Ko CP,
Bennett CF,
Krainer AR

EMBO Mol Med
  5 (10): 1586-1601; Sep 9 2013   NLM
[Abstract]
[DOI] [Full Text]

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides
Wang Z,
Jeon HY,
Rigo F,
Bennett CF,
Krainer AR

Open Biol
  2 (10): 120133; Oct 2012   NLM
[Abstract]
[DOI] [Full Text]

Antisense-based therapy for the treatment of spinal muscular atrophy
Rigo F,
Hua YM,
Krainer AR,
Bennett CF

Journal of Cell Biology
  199 (1): 21-25; Oct 1 2012  
[Abstract]
[DOI] [Full Text]

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals
Sahashi K,
Hua Y,
Ling KK,
Hung G,
Rigo F,
Horev G,
Katsuno M,
Sobue G,
Ko CP,
Bennett CF,
Krainer AR

Genes Dev
  26 (16): 1874-84; Aug 15 2012   NLM
[Abstract]
[DOI] [Full Text]

Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing
Rigo F,
Hua YM,
Chun SJ,
Prakash TP,
Krainer AR,
Bennett CF

Nature Chemical Biology
  8 (6): 555-561; Jun 2012  
[Abstract]
[DOI] [Full Text]

Widespread recognition of 5 splice sites by noncanonical base-pairing to U1 snRNA involving bulged nucleotides
Roca X,
Akerman M,
Gaus H,
Berdeja A,
Bennett CF,
Krainer AR

Genes Development
  26 (10): 1098-109; May 15 2012   Nlm
[Abstract]
[DOI] [Full Text]

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
  478 (7367): 123-6; Oct 6 2011   NLM
[Abstract]
[DOI] [Full Text]

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 ,
Rigo F,
Matson J,
Hung G,
Kaye EM,
Shihabuddin LS,
Krainer AR,
Bennett CF,
Cheng SH

Science Translational Medicine
  3 (72): Mar 2 2011   Elsevier PMC3140425
[Abstract]
[DOI]

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
  24 (15): 1634-44; Aug 1 2010   PMCID: PMC2912561
[Abstract]
[DOI] [Full Text] [PDF]

Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
Hua Y,
Vickers TA,
Okunola HL,
Bennett CF,
Krainer AR

Am J Hum Genet
  82 (4): 834-48; Apr 2008   PMCID: PMC2427210
[Abstract]
[DOI] [Full Text] [PDF]

Enhancement of SMN2 Exon 7 inclusion by antisense oligonucleotides targeting the exon
Hua YM,
Vickers TA,
Baker BF,
Bennett CF,
Krainer AR

PLoS Biology
  5 (4): e73; Apr 2007   PMCID: PMC1820610
[Abstract]
[DOI] [Full Text] [PDF]

Regulating gene expression through RNA nuclear retention
Prasanth KV,
Prasanth SG,
Xuan ZY,
Hearn S,
Freier SM,
Bennett CF,
Zhang MQ,
Spector DL

Cell
  123 (2): 249-263; Oct 2005  
[Abstract]
[DOI] [Full Text] [PDF]

Nucleocytoplasmic shuttling: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides
Lorenz P,
Misteli T,
Baker BF,
Bennett CF,
Spector DL

Nucleic Acids Research
  28 (2): 582-592; Jan 2000  
[Abstract]
[Full Text] [PDF]

Nonhistone protein BA is a glutathione S-transferase localized to interchromatinic regions of the cell nucleus
Bennett CF,
Spector DL,
Yeoman LC

J Cell Biol
  102 (2): 600-9; Feb 1986  
[Abstract]
[Full Text] [PDF]


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