Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector von Bowles, Dawn E, McPhee, Scott WJ, Li, Chengwen, Gray, Steven J, Samulski, Jade J, Camp, Angelique S, Li, Juan, Wang, Bing, Monahan, Paul E, Rabinowitz, Joseph E, Grieger, Joshua C, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Xiao, Xiao, Samulski, R Jude

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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression von Konkle, Barbara A., Walsh, Christopher E., Escobar, Miguel A., Josephson, Neil C., Young, Guy, von Drygalski, Annette, McPhee, Scott W.J., Samulski, R. Jude, Bilic, Ivan, de la Rosa, Maurus, Reipert, Birgit M., Rottensteiner, Hanspeter, Scheiflinger, Friedrich, Chapin, John C., Ewenstein, Bruce, Monahan, Paul E.

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Mechanistic insights of evaporation-induced actuation in supramolecular crystals von Piotrowska, Roxana, Hesketh, Travis, Wang, Haozhen, Martin, Alan R. G., Bowering, Deborah, Zhang, Chunqiu, Hu, Chunhua T., McPhee, Scott A., Wang, Tong, Park, Yaewon, Singla, Pulkit, McGlone, Thomas, Florence, Alastair, Tuttle, Tell, Ulijn, Rein V., Chen, Xi

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-assoc... von Monahan, Paul E, Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B, Wichlan, David G, Wu, Zhijian, Grieger, Joshua C, Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W, Booth, Carmen J, Samulski, Jade J, Kafri, Tal, McPhee, Scott W J, Samulski, R Jude

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Crystal structure and mechanistic investigation of the twister ribozyme von Liu, Yijin, Wilson, Timothy J, McPhee, Scott A, Lilley, David M J

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Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments von Lampel, Ayala, McPhee, Scott A., Kassem, Salma, Sementa, Deborah, Massarano, Tlalit, Aramini, James M., He, Ye, Ulijn, Rein V.

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Polymeric peptide pigments with sequence-encoded properties von Lampel, Ayala, McPhee, Scott A., Park, Hang-Ah, Scott, Gary G., Humagain, Sunita, Hekstra, Doeke R., Yoo, Barney, Frederix, Pim W. J. M., Li, Tai-De, Abzalimov, Rinat R., Greenbaum, Steven G., Tuttle, Tell, Hu, Chunhua, Bettinger, Christopher J., Ulijn, Rein V.

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Emergence of Cooperative Glucose-Binding Networks in Adaptive Peptide Systems von Kassem, Salma, McPhee, Scott A., Berisha, Naxhije, Ulijn, Rein V.

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Dystrophin Immunity in Duchenne's Muscular Dystrophy von Mendell, Jerry R, Campbell, Katherine, Rodino-Klapac, Louise, Sahenk, Zarife, Shilling, Chris, Lewis, Sarah, Bowles, Dawn, Gray, Steven, Li, Chengwen, Li, Juan, Galloway, Gloria, Malik, Vinod, Coley, Brian, Clark, K. Reed, Xiao, Xiao, Samulski, R. Jude, Samulski, Jade, McPhee, Scott W, Walker, Christopher M

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A critical base pair in k-turns that confers folding characteristics and correlates with biological function von McPhee, Scott A., Huang, Lin, Lilley, David M. J.

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The Structure of Sulfoindocarbocyanine 3 Terminally Attached to dsDNA via a Long, Flexible Tether von Urnavicius, Linas, McPhee, Scott A., Lilley, David M.J., Norman, David G.

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A structural database for k-turn motifs in RNA von Schroeder, Kersten T, McPhee, Scott A, Ouellet, Jonathan, Lilley, David M J

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Fluorescent RNA cytosine analogue – an internal probe for detailed structure and dynamics investigations von Füchtbauer, Anders Foller, Preus, Søren, Börjesson, Karl, McPhee, Scott A., Lilley, David M. J., Wilhelmsson, L. Marcus

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Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle von Asokan, Aravind, Conway, Julia C, Phillips, Jana L, Li, Chengwen, Hegge, Julia, Sinnott, Rebecca, Yadav, Swati, DiPrimio, Nina, Nam, Hyun-Joo, Agbandje-McKenna, Mavis, McPhee, Scott, Wolff, Jon, Samulski, R Jude

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Author Correction: Mechanistic insights of evaporation-induced actuation in supramolecular crystals von Piotrowska, Roxana, Hesketh, Travis, Wang, Haozhen, Martin, Alan R. G., Bowering, Deborah, Zhang, Chunqiu, Hu, Chunhua T., McPhee, Scott A., Wang, Tong, Park, Yaewon, Singla, Pulkit, McGlone, Thomas, Florence, Alastair, Tuttle, Tell, Ulijn, Rein V., Chen, Xi

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The Impact of Tyrosine Iodination on the Aggregation and Cleavage Kinetics of MMP-9-Responsive Peptide Sequences von MacPherson, Douglas S, McPhee, Scott A, Zeglis, Brian M, Ulijn, Rein V

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Unbiased Discovery of Dynamic Peptide‐ATP Complexes von Kroiss, Daniela, Aramini, James M., McPhee, Scott A., Tuttle, Tell, Ulijn, Rein V.

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Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments von Lampel, Ayala, McPhee, Scott A., Kassem, Salma, Sementa, Deborah, Massarano, Tlalit, Aramini, James M., He, Ye, Ulijn, Rein V.

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Unbiased Discovery of Dynamic Peptide‐ATP Complexes von Kroiss, Daniela, Aramini, James M., McPhee, Scott A., Tuttle, Tell, Ulijn, Rein V.

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Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene ( ASPA ) to the Human Brain von Janson, Christopher, McPhee, Scott, Bilaniuk, Larissa, Haselgrove, John, Testaiuti, Mark, Freese, Andrew, Wang, Dah-Jyuu, Shera, David, Hurh, Peter, Rupin, Joan, Saslow, Elizabeth, Goldfarb, Olga, Goldberg, Michael, Larijani, Ghassem, Sharrar, William, Liouterman, Larisa, Camp, Angelique, Kolodny, Edwin, Samulski, Jude, Leone, Paola

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