Therapeutic Antisense Oligonucleotides Are Coming of Age von Bennett, C. Frank

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Antisense Oligonucleotide Therapies for Neurodegenerative Diseases von Bennett, C. Frank, Krainer, Adrian R, Cleveland, Don W

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Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration von Melamed, Ze’ev, López-Erauskin, Jone, Baughn, Michael W., Zhang, Ouyang, Drenner, Kevin, Sun, Ying, Freyermuth, Fernande, McMahon, Moira A., Beccari, Melinda S., Artates, Jon W., Ohkubo, Takuya, Rodriguez, Maria, Lin, Nianwei, Wu, Dongmei, Bennett, C. Frank, Rigo, Frank, Da Cruz, Sandrine, Ravits, John, Lagier-Tourenne, Clotilde, Cleveland, Don W.

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Selective tissue targeting of synthetic nucleic acid drugs von Seth, Punit P, Tanowitz, Michael, Bennett, C Frank

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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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Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides von Scharner, Juergen, Ma, Wai Kit, Zhang, Qian, Lin, Kuan-Ting, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

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Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models von Hua, Yimin, Liu, Ying Hsiu, Sahashi, Kentaro, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

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Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome von Puttaraju, Madaiah, Jackson, Michaela, Klein, Stephanie, Shilo, Asaf, Bennett, C. Frank, Gordon, Leslie, Rigo, Frank, Misteli, Tom

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MaTAR25 lncRNA regulates the Tensin1 gene to impact breast cancer progression von Chang, Kung-Chi, Diermeier, Sarah D., Yu, Allen T., Brine, Lily D., Russo, Suzanne, Bhatia, Sonam, Alsudani, Habeeb, Kostroff, Karen, Bhuiya, Tawfiqul, Brogi, Edi, Pappin, Darryl J., Bennett, C. Frank, Rigo, Frank, Spector, David L.

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Targeting Huntingtin Expression in Patients with Huntington’s Disease von Tabrizi, Sarah J, Leavitt, Blair R, Landwehrmeyer, G. Bernhard, Wild, Edward J, Saft, Carsten, Barker, Roger A, Blair, Nick F, Craufurd, David, Priller, Josef, Rickards, Hugh, Rosser, Anne, Kordasiewicz, Holly B, Czech, Christian, Swayze, Eric E, Norris, Daniel A, Baumann, Tiffany, Gerlach, Irene, Schobel, Scott A, Paz, Erika, Smith, Anne V, Bennett, C. Frank, Lane, Roger M

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Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration von Lagier-Tourenne, Clotilde, Baughn, Michael, Rigo, Frank, Sun, Shuying, Liu, Patrick, Li, Hai-Ri, Jiang, Jie, Watt, Andrew T, Chun, Seung, Katz, Melanie, Qiu, Jinsong, Sun, Ying, Ling, Shuo-Chien, Zhu, Qiang, Polymenidou, Magdalini, Drenner, Kevin, Artates, Jonathan W, McAlonis-Downes, Melissa, Markmiller, Sebastian, Hutt, Kasey R, Pizzo, Donald P, Cady, Janet, Harms, Matthew B, Baloh, Robert H, Vandenberg, Scott R, Yeo, Gene W, Fu, Xiang-Dong, Bennett, C Frank, Cleveland, Don W, Ravits, John

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Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model von Sheng, Lei, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R, Hua, Yimin

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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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Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS von Nagata, Tetsuya, Dwyer, Chrissa A., Yoshida-Tanaka, Kie, Ihara, Kensuke, Ohyagi, Masaki, Kaburagi, Hidetoshi, Miyata, Haruka, Ebihara, Satoe, Yoshioka, Kotaro, Ishii, Takashi, Miyata, Kanjiro, Miyata, Kenichi, Powers, Berit, Igari, Tomoko, Yamamoto, Syunsuke, Arimura, Naoto, Hirabayashi, Hideki, Uchihara, Toshiki, Hara, Rintaro Iwata, Wada, Takeshi, Bennett, C. Frank, Seth, Punit P., Rigo, Frank, Yokota, Takanori

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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs von Jiang, Jie, Zhu, Qiang, Gendron, Tania F., Saberi, Shahram, McAlonis-Downes, Melissa, Seelman, Amanda, Stauffer, Jennifer E., Jafar-nejad, Paymaan, Drenner, Kevin, Schulte, Derek, Chun, Seung, Sun, Shuying, Ling, Shuo-Chien, Myers, Brian, Engelhardt, Jeffery, Katz, Melanie, Baughn, Michael, Platoshyn, Oleksandr, Marsala, Martin, Watt, Andy, Heyser, Charles J., Ard, M. Colin, De Muynck, Louis, Daughrity, Lillian M., Swing, Deborah A., Tessarollo, Lino, Jung, Chris J., Delpoux, Arnaud, Utzschneider, Daniel T., Hedrick, Stephen M., de Jong, Pieter J., Edbauer, Dieter, Van Damme, Philip, Petrucelli, Leonard, Shaw, Christopher E., Bennett, C. Frank, Da Cruz, Sandrine, Ravits, John, Rigo, Frank, Cleveland, Don W., Lagier-Tourenne, Clotilde

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RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention von Donnelly, Christopher J., Zhang, Ping-Wu, Pham, Jacqueline T., Haeusler, Aaron R., Mistry, Nipun A., Vidensky, Svetlana, Daley, Elizabeth L., Poth, Erin M., Hoover, Benjamin, Fines, Daniel M., Maragakis, Nicholas, Tienari, Pentti J., Petrucelli, Leonard, Traynor, Bryan J., Wang, Jiou, Rigo, Frank, Bennett, C. Frank, Blackshaw, Seth, Sattler, Rita, Rothstein, Jeffrey D.

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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment von Ramos, Daniel M, d'Ydewalle, Constantin, Gabbeta, Vijayalakshmi, Dakka, Amal, Klein, Stephanie K, Norris, Daniel A, Matson, John, Taylor, Shannon J, Zaworski, Phillip G, Prior, Thomas W, Snyder, Pamela J, Valdivia, David, Hatem, Christine L, Waters, Ian, Gupte, Nikhil, Swoboda, Kathryn J, Rigo, Frank, Bennett, C Frank, Naryshkin, Nikolai, Paushkin, Sergey, Crawford, Thomas O, Sumner, Charlotte J

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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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Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB von Maimon, Roy, Chillon-Marinas, Carlos, Snethlage, Cedric E., Singhal, Sarthak M., McAlonis-Downes, Melissa, Ling, Karen, Rigo, Frank, Bennett, C. Frank, Da Cruz, Sandrine, Hnasko, Thomas S., Muotri, Alysson R., Cleveland, Don W.

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy von Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.

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