Molecular Therapy - Table of Contents alert Volume 21 Issue 1

TABLE OF CONTENTS Volume 21, Issue 1 (January 2013) In this issue Editorial In This Issue Research Highlights Commentary Original Articles Also new AOP Sign up for e-alerts Recommend to your library Web feed Subscribe Editorial Top Gene Therapy for Hemophilia: Addressing the Coming Challenges of Affordability and Accessibility Mark W Skinner Mol Ther 2013 21: 1-2; 10.1038/mt.2012.272 Full Text In This Issue Top In This Issue Mol Ther 2013 21: 3; 10.1038/mt.2012.270 Full Text Research Highlights Top Research Highlights Mol Ther 2013 21: 4; 10.1038/mt.2012.271 Full Text Commentary Top Small but Significant: Inter- and Intrapatient Variations in iPS Cell–based Disease Modeling Malte Sgodda and Tobias Cantz Mol Ther 2013 21: 5-7; 10.1038/mt.2012.273 Full Text Original Articles Top Intra-amniotic Transient Transduction of the Periderm With a Viral Vector Encoding TGFβ3 Prevents Cleft Palate in Tgfβ3−/− Mouse Embryos Chadwick Wu, Masa Endo, Byung H Yang, Melissa A Radecki, Patrick F Davis, Philip W Zoltick, Ryan M Spivak, Alan W Flake, Richard E Kirschner and Hyun-Duck Nah Mol Ther 2013 21: 8-17; advance online publication, October 23, 2012; 10.1038/mt.2012.135 Abstract | Full Text Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice Kamal KE Gadalla, Mark ES Bailey, Rosemary C Spike, Paul D Ross, Kenton T Woodard, Sahana Nagabhushan Kalburgi, Lavanya Bachaboina, Jie V Deng, Anne E West, R Jude Samulski, Steven J Gray and Stuart R Cobb Mol Ther 2013 21: 18-30; advance online publication, September 25, 2012; 10.1038/mt.2012.200 Abstract | Full Text Lentivirus Mediated Delivery of Neurosin Promotes Clearance of Wild-type α-Synuclein and Reduces the Pathology in an α-Synuclein Model of LBD Brian Spencer, Sarah Michael, Jay Shen, Kori Kosberg, Edward Rockenstein, Christina Patrick, Anthony Adame and Eliezer Masliah Mol Ther 2013 21: 31-41; advance online publication, April 17, 2012; 10.1038/mt.2012.66 Abstract | Full Text Reducing Mitochondrial ROS Improves Disease-related Pathology in a Mouse Model of Ataxia-telangiectasia Anthony D D'Souza, Ian A Parish, Diane S Krause, Susan M Kaech and Gerald S Shadel Mol Ther 2013 21: 42-48; advance online publication, September 25, 2012; 10.1038/mt.2012.203 Abstract | Full Text Virus-mediated shRNA Knockdown of Nav1.3 in Rat Dorsal Root Ganglion Attenuates Nerve Injury-induced Neuropathic Pain Omar A Samad, Andrew M Tan, Xiaoyang Cheng, Edmund Foster, Sulayman D Dib-Hajj and Stephen G Waxman Mol Ther 2013 21: 49-56; advance online publication, August 21, 2012; 10.1038/mt.2012.169 Abstract | Full Text Inhibition of the Interaction Between NS3 Protease and HCV IRES With a Small Peptide: A Novel Therapeutic Strategy Upasana Ray, Chaitrali L Roy, Anuj Kumar, Prashant Mani, Agnel P Joseph, G Sudha, Debi P Sarkar, N Srinivasan and Saumitra Das Mol Ther 2013 21: 57-67; advance online publication, August 21, 2012; 10.1038/mt.2012.151 Abstract | Full Text Multimechanistic Tumor Targeted Oncolytic Virus Overcomes Resistance in Brain Tumors Kaoru Tamura, Hiroaki Wakimoto, Aayush S Agarwal, Samuel D Rabkin, Deepak Bhere, Robert L Martuza, Toshihiko Kuroda, Randa Kasmieh and Khalid Shah Mol Ther 2013 21: 68-77; advance online publication, August 28, 2012; 10.1038/mt.2012.175 Abstract | Full Text Targeting Gonadotropin-releasing Hormone Receptor Inhibits the Early Step of Ovarian Cancer Metastasis by Modulating Tumor-mesothelial Adhesion Lydia WT Cheung, Susan Yung, Tak-Mao Chan, Peter CK Leung and Alice ST Wong Mol Ther 2013 21: 78-90; advance online publication, November 20, 2012; 10.1038/mt.2012.187 Abstract | Full Text Cetuximab-mediated Tumor Regression Depends on Innate and Adaptive Immune Responses Xuanming Yang, Xunmin Zhang, Eric D Mortenson, Olga Radkevich-Brown, Yang Wang and Yang-Xin Fu Mol Ther 2013 21: 91-100; advance online publication, September 18, 2012; 10.1038/mt.2012.184 Abstract | Full Text Genetically Engineered Microvesicles Carrying Suicide mRNA/Protein Inhibit Schwannoma Tumor GrowthMTOpen Arda Mizrak, Mehmet Fatih Bolukbasi, Gokhan Baris Ozdener, Gary J Brenner, Sibylle Madlener, Erdogan Pekcan Erkan, Thomas Ströbel, Xandra O Breakefield and Okay Saydam Mol Ther 2013 21: 101-108; advance online publication, August 21, 2012; 10.1038/mt.2012.161 Abstract | Full Text Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer Robert C Münch, Hanna Janicki, Iris Völker, Anke Rasbach, Michael Hallek, Hildegard Büning and Christian J Buchholz Mol Ther 2013 21: 109-118; advance online publication, September 11, 2012; 10.1038/mt.2012.186 Abstract | Full Text IL10 Released by a New Inflammation-regulated Lentiviral System Efficiently Attenuates Zymosan-induced Arthritis Guillermo Garaulet, Arántzazu Alfranca, María Torrente, Amelia Escolano, Raquel López-Fontal, Sonsoles Hortelano, Juan M Redondo and Antonio Rodríguez Mol Ther 2013 21: 119-130; advance online publication, July 3, 2012; 10.1038/mt.2012.131 Abstract | Full Text Minicircle DNA Vectors Achieve Sustained Expression Reflected by Active Chromatin and Transcriptional Level Lia E. Gracey Maniar, Jay M. Maniar, Zhi-Ying Chen, Jiamiao Lu, Andrew Z. Fire and Mark A. Kay Mol Ther 2013 21: 131-138; advance online publication, November 27, 2012; 10.1038/mt.2012.244 Abstract | Full Text Infectivity-selective Oncolytic Adenovirus Developed by High-throughput Screening of Adenovirus-formatted Library Yoshiaki Miura, Satoshi Yamasaki, Julia Davydova, Eric Brown, Kazunori Aoki, Selwyn Vickers and Masato Yamamoto Mol Ther 2013 21: 139-148; advance online publication, October 2, 2012; 10.1038/mt.2012.205 Abstract | Full Text The Possible “Proton Sponge ” Effect of Polyethylenimine (PEI) Does Not Include Change in Lysosomal pH Rikke V Benjaminsen, Maria A Mattebjerg, Jonas R Henriksen, S Moein Moghimi and Thomas L Andresen Mol Ther 2013 21: 149-157; advance online publication, October 2, 2012; 10.1038/mt.2012.185 Abstract | Full Text Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses Agnieszka Ciesielska, Piotr Hadaczek, Gabriele Mittermeyer, Shangzhen Zhou, J Fraser Wright, Krystof S Bankiewicz and John Forsayeth Mol Ther 2013 21: 158-166; advance online publication, August 28, 2012; 10.1038/mt.2012.167 Abstract | Full Text Regulating Cytokine Function Enhances Safety and Activity of Genetic Cancer Therapies Hannah Chen, Padma Sampath, Weizhou Hou and Stephen H. Thorne Mol Ther 2013 21: 167-174; advance online publication, November 13, 2012; 10.1038/mt.2012.225 Abstract | Full Text Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich SyndromeMTOpen Samantha Scaramuzza, Luca Biasco, Anna Ripamonti, Maria C Castiello, Mariana Loperfido, Elena Draghici, Raisa J Hernandez, Fabrizio Benedicenti, Marina Radrizzani, Monica Salomoni, Marco Ranzani, Cynthia C Bartholomae, Elisa Vicenzi, Andrea Finocchi, Robbert Bredius, Marita Bosticardo, Manfred Schmidt, Christof von Kalle, Eugenio Montini, Alessandra Biffi, Maria G Roncarolo, Luigi Naldini, Anna Villa and Alessandro Aiuti Mol Ther 2013 21: 175-184; advance online publication, February 28, 2012; 10.1038/mt.2012.23 Abstract | Full Text Systemically Injected Exosomes Targeted to EGFR Deliver Antitumor MicroRNA to Breast Cancer Cells Shin-ichiro Ohno, Masakatsu Takanashi, Katsuko Sudo, Shinobu Ueda, Akio Ishikawa, Nagahisa Matsuyama, Koji Fujita, Takayuki Mizutani, Tadaaki Ohgi, Takahiro Ochiya, Noriko Gotoh and Masahiko Kuroda Mol Ther 2013 21: 185-191; advance online publication, October 2, 2012; 10.1038/mt.2012.180 Abstract | Full Text Functional In Vivo Delivery of Multiplexed Anti-HIV-1 siRNAs via a Chemically Synthesized Aptamer With a Sticky BridgeMTOpen Jiehua Zhou, C Preston Neff, Piotr Swiderski, Haitang Li, David D Smith, Tawfik Aboellail, Leila Remling-Mulder, Ramesh Akkina and John J Rossi Mol Ther 2013 21: 192-200; advance online publication, November 20, 2012; 10.1038/mt.2012.226 Abstract | Full Text Novel Protein Transduction Domain Mimics as Nonviral Delivery Vectors for siRNA Targeting NOTCH1 in Primary Human T cells A. Özgül Tezgel, Gabriela Gonzalez-Perez, Janice C. Telfer, Barbara A. Osborne, Lisa M. Minter and Gregory N. Tew Mol Ther 2013 21: 201-209; advance online publication, October 16, 2012; 10.1038/mt.2012.209 Abstract | Full Text Polyethylenimine-modified Pluronics (PCMs) Improve Morpholino Oligomer Delivery in Cell Culture and Dystrophic mdx Mice Mingxing Wang, Bo Wu, Peijuan Lu, Caryn Cloer, Jay D Tucker and Qilong Lu Mol Ther 2013 21: 210-216; advance online publication, November 20, 2012; 10.1038/mt.2012.236 Abstract | Full Text Embedding siRNA sequences targeting Apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacyMTOpen Piotr Maczuga, Jacek Lubelski, Richard van Logtenstein, Florie Borel, Bas Blits, Erwin Fakkert, Adalberto Costessi, Derek Butler, Sander van Deventer, Harald Petry, Annemart Koornneef and Pavlina Konstantinova Mol Ther 2013 21: 217-227; advance online publication, October 23, 2012; 10.1038/mt.2012.160 Abstract | Full Text Intrapatient Variations in Type 1 Diabetes-specific iPS Cell Differentiation Into Insulin-producing Cells Tayaramma Thatava, Yogish C Kudva, Ramakrishna Edukulla, Karen Squillace, Josep Genebriera De Lamo, Yulia Krotova Khan, Toshie Sakuma, Seiga Ohmine, Andre Terzic and Yasuhiro Ikeda Mol Ther 2013 21: 228-239; advance online publication, November 27, 2012; 10.1038/mt.2012.245 Abstract | Full Text Induced Pluripotent Mesenchymal Stromal Cell Clones Retain Donor-derived Differences in DNA Methylation Profiles Kaifeng Shao, Carmen Koch, Manoj K Gupta, Qiong Lin, Michael Lenz, Stephanie Laufs, Bernd Denecke, Manfred Schmidt, Matthias Linke, Hans C Hennies, Jürgen Hescheler, Martin Zenke, Ulrich Zechner, Tomo Šarić and Wolfgang Wagner Mol Ther 2013 21: 240-250; advance online publication, October 2, 2012; 10.1038/mt.2012.207 Abstract | Full Text Type I IFN Counteracts the Induction of Antigen-Specific Immune Responses by Lipid-Based Delivery of mRNA Vaccines Charlotte Pollard, Joanna Rejman, Winni De Haes, Bernard Verrier, Ellen Van Gulck, Thomas Naessens, Stefaan De Smedt, Pieter Bogaert, Johan Grooten, Guido Vanham and Stefaan De Koker Mol Ther 2013 21: 251-259; advance online publication, September 25, 2012; 10.1038/mt.2012.202 Abstract | Full Text Please note that you need to be a subscriber or site-license holder to enjoy full-text access to Molecular Therapy. 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