Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis von Coelho, Teresa, Adams, David, Silva, Ana, Lozeron, Pierre, Hawkins, Philip N, Mant, Timothy, Perez, Javier, Chiesa, Joseph, Warrington, Steve, Tranter, Elizabeth, Munisamy, Malathy, Falzone, Rick, Harrop, Jamie, Cehelsky, Jeffrey, Bettencourt, Brian R, Geissler, Mary, Butler, James S, Sehgal, Alfica, Meyers, Rachel E, Chen, Qingmin, Borland, Todd, Hutabarat, Renta M, Clausen, Valerie A, Alvarez, Rene, Fitzgerald, Kevin, Gamba-Vitalo, Christina, Nochur, Saraswathy V, Vaishnaw, Akshay K, Sah, Dinah W.Y, Gollob, Jared A, Suhr, Ole B

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Tumor heterogeneity is an active process maintained by a mutant EGFR-induced cytokine circuit in glioblastoma von Inda, Maria-del-Mar, Bonavia, Rudy, Mukasa, Akitake, Narita, Yoshitaka, Sah, Dinah W Y, Vandenberg, Scott, Brennan, Cameron, Johns, Terrance G, Bachoo, Robert, Hadwiger, Philipp, Tan, Pamela, Depinho, Ronald A, Cavenee, Webster, Furnari, Frank

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Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms von Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G, Cantley, William L, Dorkin, J Robert, Butler, James S, Qin, LiuLiang, Racie, Timothy, Sprague, Andrew, Fava, Eugenio, Zeigerer, Anja, Hope, Michael J, Zerial, Marino, Sah, Dinah WY, Fitzgerald, Kevin, Tracy, Mark A, Manoharan, Muthiah, Koteliansky, Victor, Fougerolles, Antonin de, Maier, Martin A

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Blockade of Nogo-66, Myelin-Associated Glycoprotein, and Oligodendrocyte Myelin Glycoprotein by Soluble Nogo-66 Receptor Promotes Axonal Sprouting and Recovery after Spinal Injury von Li, Shuxin, Liu, Betty P, Budel, Stephane, Li, Mingwei, Ji, Benxiu, Walus, Lee, Li, Weiwei, Jirik, Adrienna, Rabacchi, Sylvia, Choi, Eugene, Worley, Dane, Sah, Dinah W. Y, Pepinsky, Blake, Lee, Daniel, Relton, Jane, Strittmatter, Stephen M

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Widespread suppression of huntingtin with convection-enhanced delivery of siRNA von Stiles, David K., Zhang, Zhiming, Ge, Pei, Nelson, Brian, Grondin, Richard, Ai, Yi, Hardy, Peter, Nelson, Peter T., Guzaev, Andrei P., Butt, Mark T., Charisse, Klaus, Kosovrasti, Verbena, Tchangov, Lubomir, Meys, Michael, Maier, Martin, Nechev, Lubomir, Manoharan, Muthiah, Kaemmerer, William F., Gwost, Douglas, Stewart, Gregory R., Gash, Don M., Sah, Dinah W.Y.

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Therapeutic potential of RNA interference for neurological disorders von Sah, Dinah W.Y.

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Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery von Chen, Qingmin, Butler, David, Querbes, William, Pandey, Rajendra K., Ge, Pei, Maier, Martin A., Zhang, Ligang, Rajeev, Kallanthottathil G., Nechev, Lubomir, Kotelianski, Victor, Manoharan, Muthiah, Sah, Dinah W.Y.

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Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles von Nakayama, Tomoko, Butler, James S, Sehgal, Alfica, Severgnini, Mariano, Racie, Tim, Sharman, Jennifer, Ding, Feng, Morskaya, Svetlana Shulga, Brodsky, Joshua, Tchangov, Lubomir, Kosovrasti, Verbena, Meys, Mike, Nechev, Lubomir, Wang, Gang, Peng, Chang Geng, Fang, Yupang, Maier, Martin, Rajeev, Kallanthottathil G, Li, Robert, Hettinger, Julia, Barros, Scott, Clausen, Valerie, Zhang, Xuemei, Wang, Qianfan, Hutabarat, Renta, Dokholyan, Nikolay V, Wolfrum, Christian, Manoharan, Muthiah, Kotelianski, Victor, Stoffel, Markus, Sah, Dinah Wy

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Tissue-specific gene silencing monitored in circulating RNA von Sehgal, Alfica, Chen, Qingmin, Gibbings, Derrick, Sah, Dinah W Y, Bumcrot, David

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Neurotrophic factors as novel therapeutics for neuropathic pain von Porreca, Frank, Sah, Dinah W.Y, Ossipo, Michael H

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siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cells von Hewett, Jeffrey W., Nery, Flávia C., Niland, Brian, Ge, Pei, Tan, Pamela, Hadwiger, Philipp, Tannous, Bakhos A., Sah, Dinah W.Y., Breakefield, Xandra O.

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Targeting the Nogo Receptor to Treat Central Nervous System Injuries von Lee, Daniel H. S, Strittmatter, Stephen M, Sah, Dinah W. Y

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Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA von Grondin, Richard, Ge, Pei, Chen, Qingmin, Sutherland, Jessica E, Zhang, Zhiming, Gash, Don M, Stiles, David K, Stewart, Gregory R, Sah, Dinah W Y, Kaemmerer, William F

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GFRalpha3 is expressed predominantly in nociceptive sensory neurons von Orozco, Olivia E., Walus, Lee, Sah, Dinah W. Y., Pepinsky, R. Blake, Sanicola, Michele

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RNAi-Mediated Inhibition of Tmprss6 Elevates Hamp1 Expression and Reduces Serum Iron Levels in Mice von Toudjarska, Ivanka, Cai, Zuhua, Racie, Tim, Milstein, Stuart, Bettencourt, Brian R, Hettinger, Julia, Sah, Dinah WY, Vaishnaw, Akshay, Bumcrot, David

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RNAi-Mediated Inhibition of Activated Protein C- A New Approach for Hemophilia Treatment von Toudjarska, Ivanka, Cai, Zuhua, Racie, Tim, Hettinger, Julia, Milstein, Stuart, Bettencourt, Brian R, Sah, Dinah WY, Bumcrot, David, Maraganore, John, Vaishnaw, Akshay

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A Neutralizing Anti-Nogo66 Receptor Monoclonal Antibody Reverses Inhibition of Neurite Outgrowth by Central Nervous System Myelin von Li, Weiwei, Walus, Lee, Rabacchi, Sylvia A, Jirik, Adrienna, Chang, Ernie, Schauer, Jessica, Zheng, Betty H, Benedetti, Nancy J, Liu, Betty P, Choi, Eugene, Worley, Dane, Silvian, Laura, Mo, Wenjun, Mullen, Colleen, Yang, Weixing, Strittmatter, Stephen M, Sah, Dinah W Y, Pepinsky, Blake, Lee, Daniel H S

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Liver Specific Delivery of siRNA Targeting EGLN Prolyl Hydroxylases Activates Hepatic Erythropoietin Production and Stimulates Erythropoiesis von Querbes, William, Bogorad, Roman, Moslehi, Javid, Akinc, Akin, Wong, Jamie, Zurenko, Chris, Qin, June, Hettinger, Julia, Kuchimanchi, Satya, Charisse, Klaus, Sah, Dinah WY, Fitzgerald, Kevin, Kotelianski, Victor, Kaelin, William G.

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Abstract 3126: Tumor heterogeneity in glioblastoma is an active process driven by a mutant EGFR-induced paracrine circuit von Inda, Maria-del-Mar, Bonavia, Rudy, Sah, Dinah WY, Vandenberg, Scott, Brennan, Cameron, Johns, Terrance G., Hadwiger, Philipp, Tan, Pamela, Cavenee, Webster K., Furnari, Frank B.

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Distribution of GDNF family receptor alpha 3 and RET in rat and human non-neural tissues von Yang, Chunhua, Hutto, David, Sah, Dinah WY

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