State-of-the-art gene-based therapies: the road ahead von Kay, Mark A.

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The Loop Position of shRNAs and Pre-miRNAs Is Critical for the Accuracy of Dicer Processing In Vivo von Gu, Shuo, Jin, Lan, Zhang, Yue, Huang, Yong, Zhang, Feijie, Valdmanis, Paul N., Kay, Mark A.

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Ruvkun and Ambros recognized for miRNAs von Kay, Mark A.

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Drugging RNAi von Haussecker, Dirk, Kay, Mark A

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Recombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference von Borel, Florie, Kay, Mark A, Mueller, Christian

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective von Sabatino, Denise E., Bushman, Frederic D., Chandler, Randy J., Crystal, Ronald G., Davidson, Beverly L., Dolmetsch, Ricardo, Eggan, Kevin C., Gao, Guangping, Gil-Farina, Irene, Kay, Mark A., McCarty, Douglas M., Montini, Eugenio, Ndu, Adora, Yuan, Jing

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model von Lisowski, Leszek, Dane, Allison P., Chu, Kirk, Zhang, Yue, Cunningham, Sharon C., Wilson, Elizabeth M., Nygaard, Sean, Grompe, Markus, Alexander, Ian E., Kay, Mark A.

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Human tRNA-derived small RNAs in the global regulation of RNA silencing von Haussecker, Dirk, Huang, Yong, Lau, Ashley, Parameswaran, Poornima, Fire, Andrew Z, Kay, Mark A

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Biological basis for restriction of microRNA targets to the 3′ untranslated region in mammalian mRNAs von Gu, Shuo, Jin, Lan, Zhang, Feijie, Sarnow, Peter, Kay, Mark A

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Slicing-Independent RISC Activation Requires the Argonaute PAZ Domain von Gu, Shuo, Jin, Lan, Huang, Yong, Zhang, Feijie, Kay, Mark A.

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Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration von Malato, Yann, Naqvi, Syed, Schürmann, Nina, Ng, Raymond, Wang, Bruce, Zape, Joan, Kay, Mark A, Grimm, Dirk, Willenbring, Holger

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Genome Editing of Isogenic Human Induced Pluripotent Stem Cells Recapitulates Long QT Phenotype for Drug Testing von Wang, Yongming, PhD, Liang, Ping, MD, PhD, Lan, Feng, PhD, Wu, Haodi, PhD, Lisowski, Leszek, PhD, Gu, Mingxia, MD, PhD, Hu, Shijun, PhD, Kay, Mark A., MD, PhD, Urnov, Fyodor D., PhD, Shinnawi, Rami, PhD, Gold, Joseph D., PhD, Gepstein, Lior, MD, PhD, Wu, Joseph C., MD, PhD

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A bright cyan-excitable orange fluorescent protein facilitates dual-emission microscopy and enhances bioluminescence imaging in vivo von Chu, Jun, Oh, Younghee, Sens, Alex, Ataie, Niloufar, Dana, Hod, Macklin, John J, Laviv, Tal, Welf, Erik S, Dean, Kevin M, Zhang, Feijie, Kim, Benjamin B, Tang, Clement Tran, Hu, Michelle, Baird, Michelle A, Davidson, Michael W, Kay, Mark A, Fiolka, Reto, Yasuda, Ryohei, Kim, Douglas S, Ng, Ho-Leung, Lin, Michael Z

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RNA structure probing reveals the structural basis of Dicer binding and cleavage von Luo, Qing-Jun, Zhang, Jinsong, Li, Pan, Wang, Qing, Zhang, Yue, Roy-Chaudhuri, Biswajoy, Xu, Jianpeng, Kay, Mark A., Zhang, Qiangfeng Cliff

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A tRNA-Derived Small RNA Regulates Ribosomal Protein S28 Protein Levels after Translation Initiation in Humans and Mice von Kim, Hak Kyun, Xu, Jianpeng, Chu, Kirk, Park, Hyesuk, Jang, Hagoon, Li, Pan, Valdmanis, Paul N., Zhang, Qiangfeng Cliff, Kay, Mark A.

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The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner von Gonzalez-Sandoval, Adriana, Pekrun, Katja, Tsuji, Shinnosuke, Zhang, Feijie, Hung, King L., Chang, Howard Y., Kay, Mark A.

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miR-122 removal in the liver activates imprinted microRNAs and enables more effective microRNA-mediated gene repression von Valdmanis, Paul N., Kim, Hak Kyun, Chu, Kirk, Zhang, Feijie, Xu, Jianpeng, Munding, Elizabeth M., Shen, Jia, Kay, Mark A.

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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways von Grimm, Dirk, Streetz, Konrad L., Jopling, Catherine L., Storm, Theresa A., Pandey, Kusum, Davis, Corrine R., Marion, Patricia, Salazar, Felix, Kay, Mark A.

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In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses von GRIMM, Dirk, LEE, Joyce S, WANG, Lora, DESAI, Tushar, AKACHE, Bassel, STORM, Theresa A, KAY, Mark A

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Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice von Azuma, Hisaya, Paulk, Nicole, Ranade, Aarati, Dorrell, Craig, Al-Dhalimy, Muhsen, Ellis, Ewa, Strom, Stephen, Kay, Mark A, Finegold, Milton, Grompe, Markus

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