The Noncoding RNA MALAT1 Is a Critical Regulator of the Metastasis Phenotype of Lung Cancer Cells von GUTSCHNER, Tony, HÄMMERLE, Monika, ZÖRNIG, Martin, ROBERT MACLEOD, A, SPECTOR, David L, DIEDERICHS, Sven, EIBMANN, Moritz, HSU, Jeff, KIM, Youngsoo, HUNG, Gene, REVENKO, Alexey, ARUN, Gayatri, STENTRUP, Marion, GROSS, Matthias

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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study von Finkel, Richard S, Dr, Chiriboga, Claudia A, MD, Vajsar, Jiri, MD, Day, John W, MD, Montes, Jacqueline, EdD, De Vivo, Darryl C, MD, Yamashita, Mason, MD, Rigo, Frank, PhD, Hung, Gene, MD, Schneider, Eugene, MD, Norris, Daniel A, PhD, Xia, Shuting, MS, Bennett, C Frank, PhD, Bishop, Kathie M, PhD

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Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model von Hua, Yimin, Sahashi, Kentaro, Rigo, Frank, Hung, Gene, Horev, Guy, Bennett, C. Frank, Krainer, Adrian R.

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Enhanced Store-Operated Calcium Entry Leads to Striatal Synaptic Loss in a Huntington's Disease Mouse Model von Wu, Jun, Ryskamp, Daniel A, Liang, Xia, Egorova, Polina, Zakharova, Olga, Hung, Gene, Bezprozvanny, Ilya

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Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model von Hua, Yimin, Sahashi, Kentaro, Hung, Gene, Rigo, Frank, Passini, Marco A, Bennett, C Frank, Krainer, Adrian R

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Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease von Hagemann, Tracy L., Powers, Berit, Mazur, Curt, Kim, Aneeza, Wheeler, Steven, Hung, Gene, Swayze, Eric, Messing, Albee

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The lncRNA Malat1 Is Dispensable for Mouse Development but Its Transcription Plays a cis-Regulatory Role in the Adult von 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.

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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis von 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.

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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 von 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

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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 von 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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Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy von 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.

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Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models von 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.

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Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy von 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

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Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals von Swayze, Eric E, Siwkowski, Andrew M, Wancewicz, Edward V, Migawa, Michael T, Wyrzykiewicz, Tadeusz K, Hung, Gene, Monia, Brett P, Bennett, and C. Frank

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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients von 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

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Rescue of Metabolic Alterations in AR113Q Skeletal Muscle by Peripheral Androgen Receptor Gene Silencing von Giorgetti, Elisa, Yu, Zhigang, Chua, Jason P., Shimamura, Ryosuke, Zhao, Lili, Zhu, Fan, Venneti, Sriram, Pennuto, Maria, Guan, Yuanfang, Hung, Gene, Lieberman, Andrew P.

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Deficiency of the oxygen sensor prolyl hydroxylase 1 attenuates hypercholesterolaemia, atherosclerosis, and hyperglycaemia von 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

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TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals von 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

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Pathological impact of SMN2 mis‐splicing in adult SMA mice von Sahashi, Kentaro, Ling, Karen K. Y., Hua, Yimin, Wilkinson, John Erby, Nomakuchi, Tomoki, Rigo, Frank, Hung, Gene, Xu, David, Jiang, Ya‐Ping, Lin, Richard Z., Ko, Chien‐Ping, Bennett, C. Frank, Krainer, Adrian R.

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CD40 Generation 2.5 Antisense Oligonucleotide Treatment Attenuates Doxorubicin-induced Nephropathy and Kidney Inflammation von Donner, Aaron J, Yeh, Steve T, Hung, Gene, Graham, Mark J, Crooke, Rosanne M, Mullick, Adam E

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