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Published in Cancer research (Chicago, Ill.) (01.02.2013)
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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
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Published in The Lancet (British edition) (17.12.2016)
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Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
Hua, Yimin, Sahashi, Kentaro, Hung, Gene, Rigo, Frank, Passini, Marco A, Bennett, C Frank, Krainer, Adrian R
Published in Genes & development (01.08.2010)
Published in Genes & development (01.08.2010)
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Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease
Hagemann, Tracy L., Powers, Berit, Mazur, Curt, Kim, Aneeza, Wheeler, Steven, Hung, Gene, Swayze, Eric, Messing, Albee
Published in Annals of neurology (01.01.2018)
Published in Annals of neurology (01.01.2018)
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The lncRNA Malat1 Is Dispensable for Mouse Development but Its Transcription Plays a cis-Regulatory Role in the Adult
Zhang, Bin, Arun, Gayatri, Mao, Yuntao S., Lazar, Zsolt, Hung, Gene, Bhattacharjee, Gourab, Xiao, Xiaokun, Booth, Carmen J., Wu, Jie, Zhang, Chaolin, Spector, David L.
Published in Cell reports (Cambridge) (26.07.2012)
Published in Cell reports (Cambridge) (26.07.2012)
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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Kordasiewicz, Holly B., Stanek, Lisa M., Wancewicz, Edward V., Mazur, Curt, McAlonis, Melissa M., Pytel, Kimberly A., Artates, Jonathan W., Weiss, Andreas, Cheng, Seng H., Shihabuddin, Lamya S., Hung, Gene, Bennett, C. Frank, Cleveland, Don W.
Published in Neuron (Cambridge, Mass.) (21.06.2012)
Published in Neuron (Cambridge, Mass.) (21.06.2012)
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Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates
Rigo, Frank, Chun, Seung J, Norris, Daniel A, Hung, Gene, Lee, Sam, Matson, John, Fey, Robert A, Gaus, Hans, Hua, Yimin, Grundy, John S, Krainer, Adrian R, Henry, Scott P, Bennett, C Frank
Published in The Journal of pharmacology and experimental therapeutics (01.07.2014)
Published in The Journal of pharmacology and experimental therapeutics (01.07.2014)
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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy
Passini, Marco A, Bu, Jie, Richards, Amy M, Kinnecom, Cathrine, Sardi, S Pablo, Stanek, Lisa M, Hua, Yimin, Rigo, Frank, Matson, John, Hung, Gene, Kaye, Edward M, Shihabuddin, Lamya S, Krainer, Adrian R, Bennett, C Frank, Cheng, Seng H
Published in Science translational medicine (02.03.2011)
Published in Science translational medicine (02.03.2011)
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Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts
Burel, Sebastien A, Hart, Christopher E, Cauntay, Patrick, Hsiao, Jill, Machemer, Todd, Katz, Melanie, Watt, Andy, Bui, Huynh-Hoa, Younis, Husam, Sabripour, Mahyar, Freier, Susan M, Hung, Gene, Dan, Amy, Prakash, T P, Seth, Punit P, Swayze, Eric E, Bennett, C Frank, Crooke, Stanley T, Henry, Scott P
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Published in Nucleic acids research (18.03.2016)
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Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7
Niu, Chenchen, Prakash, Thazah P, Kim, Aneeza, Quach, John L, Huryn, Laryssa A, Yang, Yuechen, Lopez, Edith, Jazayeri, Ali, Hung, Gene, Sopher, Bryce L, Brooks, Brian P, Swayze, Eric E, Bennett, C Frank, La Spada, Albert R
Published in Science translational medicine (31.10.2018)
Published in Science translational medicine (31.10.2018)
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Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy
Cortes, Constanza J., Ling, Shuo-Chien, Guo, Ling T., Hung, Gene, Tsunemi, Taiji, Ly, Linda, Tokunaga, Seiya, Lopez, Edith, Sopher, Bryce L., Bennett, C. Frank, Shelton, G. Diane, Cleveland, Don W., La Spada, Albert R.
Published in Neuron (Cambridge, Mass.) (16.04.2014)
Published in Neuron (Cambridge, Mass.) (16.04.2014)
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Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
Moore, Lauren R., Rajpal, Gautam, Dillingham, Ian T., Qutob, Maya, Blumenstein, Kate G., Gattis, Danielle, Hung, Gene, Kordasiewicz, Holly B., Paulson, Henry L., McLoughlin, Hayley S.
Published in Molecular therapy. Nucleic acids (16.06.2017)
Published in Molecular therapy. Nucleic acids (16.06.2017)
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Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy
Lieberman, Andrew P., Yu, Zhigang, Murray, Sue, Peralta, Raechel, Low, Audrey, Guo, Shuling, Yu, Xing Xian, Cortes, Constanza J., Bennett, C. Frank, Monia, Brett P., La Spada, Albert R., Hung, Gene
Published in Cell reports (Cambridge) (01.05.2014)
Published in Cell reports (Cambridge) (01.05.2014)
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Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals
Swayze, Eric E, Siwkowski, Andrew M, Wancewicz, Edward V, Migawa, Michael T, Wyrzykiewicz, Tadeusz K, Hung, Gene, Monia, Brett P, Bennett, and C. Frank
Published in Nucleic acids research (01.01.2007)
Published in Nucleic acids research (01.01.2007)
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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients
Skotte, Niels H, Southwell, Amber L, Østergaard, Michael E, Carroll, Jeffrey B, Warby, Simon C, Doty, Crystal N, Petoukhov, Eugenia, Vaid, Kuljeet, Kordasiewicz, Holly, Watt, Andrew T, Freier, Susan M, Hung, Gene, Seth, Punit P, Bennett, C Frank, Swayze, Eric E, Hayden, Michael R
Published in PloS one (10.09.2014)
Published in PloS one (10.09.2014)
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Rescue of Metabolic Alterations in AR113Q Skeletal Muscle by Peripheral Androgen Receptor Gene Silencing
Giorgetti, Elisa, Yu, Zhigang, Chua, Jason P., Shimamura, Ryosuke, Zhao, Lili, Zhu, Fan, Venneti, Sriram, Pennuto, Maria, Guan, Yuanfang, Hung, Gene, Lieberman, Andrew P.
Published in Cell reports (Cambridge) (27.09.2016)
Published in Cell reports (Cambridge) (27.09.2016)
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Deficiency of the oxygen sensor prolyl hydroxylase 1 attenuates hypercholesterolaemia, atherosclerosis, and hyperglycaemia
Marsch, Elke, Demandt, Jasper A F, Theelen, Thomas L, Tullemans, Bibian M E, Wouters, Kristiaan, Boon, Mariëtte R, van Dijk, Theo H, Gijbels, Marion J, Dubois, Ludwig J, Meex, Steven J R, Mazzone, Massimiliano, Hung, Gene, Fisher, Edward A, Biessen, Erik A L, Daemen, Mat J A P, Rensen, Patrick C N, Carmeliet, Peter, Groen, Albert K, Sluimer, Judith C
Published in European heart journal (14.10.2016)
Published in European heart journal (14.10.2016)
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TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals
Sahashi, Kentaro, Hua, Yimin, Ling, Karen K Y, Hung, Gene, Rigo, Frank, Horev, Guy, Katsuno, Masahisa, Sobue, Gen, Ko, Chien-Ping, Bennett, C Frank, Krainer, Adrian R
Published in Genes & development (15.08.2012)
Published in Genes & development (15.08.2012)
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