RNAi therapeutics: a potential new class of pharmaceutical drugs von Sah, Dinah W Y, Bumcrot, David, Manoharan, Muthiah, Koteliansky, Victor

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Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications von Friedland, Ari E, Baral, Reshica, Singhal, Pankhuri, Loveluck, Katherine, Shen, Shen, Sanchez, Minerva, Marco, Eugenio, Gotta, Gregory M, Maeder, Morgan L, Kennedy, Edward M, Kornepati, Anand V R, Sousa, Alexander, Collins, McKensie A, Jayaram, Hari, Cullen, Bryan R, Bumcrot, David

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Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy von Gori, Jennifer L, Hsu, Patrick D, Maeder, Morgan L, Shen, Shen, Welstead, G Grant, Bumcrot, David

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Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs von Soutschek, Jürgen, Akinc, Akin, Bramlage, Birgit, Charisse, Klaus, Constien, Rainer, Donoghue, Mary, Elbashir, Sayda, Geick, Anke, Hadwiger, Philipp, Harborth, Jens, John, Matthias, Kesavan, Venkitasamy, Lavine, Gary, Pandey, Rajendra K, Racie, Timothy, Rajeev, Kallanthottathil G, Röhl, Ingo, Toudjarska, Ivanka, Wang, Gang, Wuschko, Silvio, Bumcrot, David, Koteliansky, Victor, Limmer, Stefan, Manoharan, Muthiah, Vornlocher, Hans-Peter

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Claudin-3 gene silencing with siRNA suppresses ovarian tumor growth and metastasis von Huang, Yu-Hung, Bao, Yunhua, Peng, Weidan, Goldberg, Michael, Love, Kevin, Bumcrot, David A, Cole, Geoffrey, Langer, Robert, Anderson, Daniel G, Sawicki, Janet A

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Alpha-synuclein suppression by targeted small interfering RNA in the primate substantia nigra von McCormack, Alison L, Mak, Sally K, Henderson, Jaimie M, Bumcrot, David, Farrer, Matthew J, Di Monte, Donato A

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Transcriptional characterization of iPSC-derived microglia as a model for therapeutic development in neurodegeneration von Ramaswami, Gokul, Yuva-Aydemir, Yeliz, Akerberg, Brynn, Matthews, Bryan, Williams, Jenna, Golczer, Gabriel, Huang, Jiaqi, Al Abdullatif, Ali, Huh, Dann, Burkly, Linda C., Engle, Sandra J., Grossman, Iris, Sehgal, Alfica, Sigova, Alla A., Fremeau, Robert T., Liu, Yuting, Bumcrot, David

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Author Correction: Transcriptional characterization of iPSC-derived microglia as a model for therapeutic development in neurodegeneration von Ramaswami, Gokul, Yuva-Aydemir, Yeliz, Akerberg, Brynn, Matthews, Bryan, Williams, Jenna, Golczer, Gabriel, Huang, Jiaqi, Al Abdullatif, Ali, Huh, Dann, Burkly, Linda C., Engle, Sandra J., Grossman, Iris, Sehgal, Alfica, Sigova, Alla A., Fremeau, Robert T., Liu, Yuting, Bumcrot, David

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Discovery and Targeting of the Signaling Controls of PNPLA3 to Effectively Reduce Transcription, Expression, and Function in Pre-Clinical NAFLD/NASH Settings von Schwartz, Brian E., Rajagopal, Vaishnavi, Smith, Cynthia, Cohick, Evan, Whissell, Gavin, Gamboa, Mario, Pai, Rutuja, Sigova, Alla, Grossman, Iris, Bumcrot, David, Sasidharan, Kavitha, Romeo, Stefano, Sehgal, Alfica, Pingitore, Piero

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MicroRNA-34 mediates AR-dependent p53-induced apoptosis in prostate cancer von Rokhlin, Oskar W, Scheinker, Vladimir S, Taghiyev, Agshin F, Bumcrot, David, Glover, Rebecca A, Cohen, Michael B.

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Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway von Bumcrot, David, John, Matthias, Constien, Rainer, Akinc, Akin, Goldberg, Michael, Moon, Young-Ah, Spranger, Martina, Hadwiger, Philipp, Soutschek, Jürgen, Vornlocher, Hans-Peter, Manoharan, Muthiah, Stoffel, Markus, Langer, Robert, Anderson, Daniel G, Horton, Jay D, Koteliansky, Victor

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In vivo silencing of alpha-synuclein using naked siRNA von Lewis, Jada, Melrose, Heather, Bumcrot, David, Hope, Andrew, Zehr, Cynthia, Lincoln, Sarah, Braithwaite, Adam, He, Zhen, Ogholikhan, Sina, Hinkle, Kelly, Kent, Caroline, Toudjarska, Ivanka, Charisse, Klaus, Braich, Ravi, Pandey, Rajendra K, Heckman, Michael, Maraganore, Demetrius M, Crook, Julia, Farrer, Matthew J

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Requirement of 19K form of Sonic hedgehog for induction of distinct ventral cell types in CNS explants von Marti, Elisa, Bumcrot, David A, Takada, Ritsuko, McMahon, Andrew P

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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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JMnorm: a novel joint multi-feature normalization method for integrative and comparative epigenomics von Xiang, Guanjue, Guo, Yuchun, Bumcrot, David, Sigova, Alla

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Small-molecule modulators of Hedgehog signaling: identification and characterization of Smoothened agonists and antagonists von Frank-Kamenetsky, Maria, Zhang, Xiaoyan M, Bottega, Steve, Guicherit, Oivin, Wichterle, Hynek, Dudek, Henryk, Bumcrot, David, Wang, Frank Y, Jones, Simon, Shulok, Janine, Rubin, Lee L, Porter, Jeffery A

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Calcium/calmodulin-dependent kinase II plays an important role in prostate cancer cell survival von Rokhlin, Oskar, Taghiyev, Agshin F., Bayer, K. Ulrich, Bumcrot, David, Kotelianski, Victor E, Glover, Rebecca A., Cohen, Michael B.

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Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10 von Maeder, Morgan L., Stefanidakis, Michael, Wilson, Christopher J., Baral, Reshica, Barrera, Luis Alberto, Bounoutas, George S., Bumcrot, David, Chao, Hoson, Ciulla, Dawn M., DaSilva, Jennifer A., Dass, Abhishek, Dhanapal, Vidya, Fennell, Tim J., Friedland, Ari E., Giannoukos, Georgia, Gloskowski, Sebastian W., Glucksmann, Alexandra, Gotta, Gregory M., Jayaram, Hariharan, Haskett, Scott J., Hopkins, Bei, Horng, Joy E., Joshi, Shivangi, Marco, Eugenio, Mepani, Rina, Reyon, Deepak, Ta, Terence, Tabbaa, Diana G., Samuelsson, Steven J., Shen, Shen, Skor, Maxwell N., Stetkiewicz, Pam, Wang, Tongyao, Yudkoff, Clifford, Myer, Vic E., Albright, Charles F., Jiang, Haiyan

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Distribution of Sonic hedgehog peptides in the developing chick and mouse embryo von Martí, E, Takada, R, Bumcrot, D A, Sasaki, H, McMahon, A P

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Proteolytic processing yields two secreted forms of sonic hedgehog von Bumcrot, David A., Takada, Ritsuko, Mcmahon, Andrew P.

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