Molecular Therapy - Table of Contents alert Volume 21 Issue 2

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With an Impact Factor of 13.668*, Molecular Psychiatry continues to be the #1 journal in psychiatry, publishing high-impact work that elucidates key issues in psychiatry and related fields. Sign up to receive the FREE e-alert and stay up-to-date on the latest research. *2011 Journal Citation Report (Thomson Reuters, 2012)   Editorial Top “Special Exemptions”: Should They Be Put on Trial? H Bobby Gaspar, Sue Swift and Adrian J Thrasher Mol Ther 2013 21: 261-262; 10.1038/mt.2013.1 Full Text In This Issue Top In This Issue Mol Ther 2013 21: 264; 10.1038/mt.2013.2 Full Text Research Highlights Top Research Highlights Mol Ther 2013 21: 265; 10.1038/mt.2013.3 Full Text Letter to the Editor Top Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases Jacques P Tremblay, Xiao Xiao, Annemieke Aartsma-Rus, Carlos Barbas, Helen M Blau, Adam J Bogdanove, Kym Boycott, Serge Braun, Xandra O Breakefield, Juan A Bueren, Michael Buschmann, Barry J Byrne, Michele Calos, Toni Cathomen, Jeffrey Chamberlain, Marinee Chuah, Kenneth Cornetta, Kay E Davies, J George Dickson, Philippe Duchateau, Terence R Flotte, Daniel Gaudet, Charles A Gersbach, Renald Gilbert, Joseph Glorioso, Roland W Herzog, Katherine A High, Wenlin Huang, Johnny Huard, J Keith Joung, Depei Liu, Dexi Liu, Hanns Lochmüller, Lawrence Lustig, Jeffrey Martens, Bernard Massie, Fulvio Mavilio, Jerry R Mendell, Amit Nathwani, Katherine Ponder, Matthew Porteus, Jack Puymirat, Jude Samulski, Shin’ichi Takeda, Adrian Thrasher, Thierry VandenDriessche, Yuquan Wei, James M Wilson, Steve D Wilton, John H Wolfe and Guangping Gao Mol Ther 2013 21: 266-268; 10.1038/mt.2013.4 Full Text Commentary Top Flushing Out Antibodies to Make AAV Gene Therapy Available to More Patients Steven E Raper, James M Wilson and Frederick A Nunes Mol Ther 2013 21: 269-271; 10.1038/mt.2013.5 Full Text Review Top Assessing the Risks of Genotoxicity in the Therapeutic Development of Induced Pluripotent Stem Cells So Gun Hong, Cynthia E Dunbar and Thomas Winkler Mol Ther 2013 21: 272-281; advance online publication, December 4, 2012; 10.1038/mt.2012.255 Abstract | Full Text Original Articles Top Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice Sofia Benkhelifa-Ziyyat, Aurore Besse, Marianne Roda, Sandra Duque, Stéphanie Astord, Romain Carcenac, Thibaut Marais and Martine Barkats Mol Ther 2013 21: 282-290; advance online publication, January 8, 2013; 10.1038/mt.2012.261 Abstract | Full Text Transient Removal of CD46 Is Safe and Increases B-cell Depletion by Rituximab in CD46 Transgenic Mice and Macaques Ines Beyer, Hua Cao, Jonas Persson, Hongjie Wang, Ying Liu, Roma Yumul, Zongyi Li, Douglas Woodle, Ronald Manger, Michael Gough, Diane Rocha, Jaclyn Bogue, Audrey Baldessari, Ronald Berenson, Darrick Carter and André Lieber Mol Ther 2013 21: 291-299; advance online publication, October 23, 2012; 10.1038/mt.2012.212 Abstract | Full Text Systemic TNFα Gene Therapy Synergizes With Liposomal Doxorubicine in the Treatment of Metastatic Cancer Baowei Su, Arzu Cengizeroglu, Katarina Farkasova, Joana R. Viola, Martina Anton, Joachim W. Ellwart, Rudolf Haase, Ernst Wagner and Manfred Ogris Mol Ther 2013 21: 300-308; advance online publication, November 13, 2012; 10.1038/mt.2012.229 Abstract | Full Text Particle Tracking Analysis for the Intracellular Trafficking of Nanoparticles Modified with African Swine Fever Virus Protein p54-derived Peptide Hidetaka Akita, Kaoru Enoto, Hiroki Tanaka and Hideyoshi Harashima Mol Ther 2013 21: 309-317; advance online publication, November 20, 2012; 10.1038/mt.2012.235 Abstract | Full Text Minimizing the Inhibitory Effect of Neutralizing Antibody for Efficient Gene Expression in the Liver With Adeno-associated Virus 8 Vectors Jun Mimuro, Hiroaki Mizukami, Shuji Hishikawa, Tomokazu Ikemoto, Akira Ishiwata, Asuka Sakata, Tsukasa Ohmori, Seiji Madoiwa, Fumiko Ono, Keiya Ozawa and Yoichi Sakata Mol Ther 2013 21: 318-323; advance online publication, December 18, 2012; 10.1038/mt.2012.258 Abstract | Full Text The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis Ali Nowrouzi, Wing T Cheung, Tingting Li, Xuegong Zhang, Anne Arens, Anna Paruzynski, Simon N Waddington, Emma Osejindu, Safia Reja, Christof von Kalle, Yoahe Wang, Faisal Al-Allaf, Lisa Gregory, Matthew Themis, Maxine Holder, Niraja Dighe, Alaine Ruthe, Suzanne MK Buckley, Brian Bigger, Eugenio Montini, Adrian J Thrasher, Robert Andrews, Terry P Roberts, Robert F Newbold, Charles Coutelle, Manfred Schmidt and Mike Themis Mol Ther 2013 21: 324-337; advance online publication, November 13, 2012; 10.1038/mt.2012.224 Abstract | Full Text Multifaceted Therapeutic Targeting of Ovarian Peritoneal Carcinomatosis Through Virus-induced Immunomodulation Shashi Gujar, Rebecca Dielschneider, Derek Clements, Erin Helson, Maya Shmulevitz, Paola Marcato, Da Pan, Lu-zhe Pan, Dae-Gyun Ahn, Abdulaziz Alawadhi and Patrick WK Lee Mol Ther 2013 21: 338-347; advance online publication, November 13, 2012; 10.1038/mt.2012.228 Abstract | Full Text The Efficacy Versus Toxicity Profile of Combination Virotherapy and TLR Immunotherapy Highlights the Danger of Administering TLR Agonists to Oncolytic Virus-treated Mice Diana M Rommelfanger, Marta C Grau, Rosa M Diaz, Elizabeth Ilett, Luis Alvarez-Vallina, Jill M Thompson, Timothy J Kottke, Alan Melcher and Richard G Vile Mol Ther 2013 21: 348-357; advance online publication, September 25, 2012; 10.1038/mt.2012.204 Abstract | Full Text Systemic Delivery of Modified mRNA Encoding Herpes Simplex Virus 1 Thymidine Kinase for Targeted Cancer Gene Therapy Yuhua Wang, Hsing-hao Su, Yang Yang, Yunxia Hu, Lu Zhang, Pilar Blancafort and Leaf Huang Mol Ther 2013 21: 358-367; advance online publication, December 11, 2012; 10.1038/mt.2012.250 Abstract | Full Text Identification and Functional Characterization of tRNA-derived RNA Fragments (tRFs) in Respiratory Syncytial Virus Infection Qingrong Wang, Inhan Lee, Junping Ren, Subramanian Shankar Ajay, Yong Sun Lee and Xiaoyong Bao Mol Ther 2013 21: 368-379; advance online publication, November 27, 2012; 10.1038/mt.2012.237 Abstract | Full Text RNA Interference Targeting CUG Repeats in a Mouse Model of Myotonic Dystrophy Krzysztof Sobczak, Thurman M Wheeler, Wenli Wang and Charles A Thornton Mol Ther 2013 21: 380-387; advance online publication, November 27, 2012; 10.1038/mt.2012.222 Abstract | Full Text Smad7 suppresses renal fibrosis via altering expression of TGF-β/Smad3-regulated microRNAs Arthur C.K. Chung, Yuan Dong, Weiqin Yang, Xiang Zhong, Rong Li and Hui Y. Lan Mol Ther 2013 21: 388-398; advance online publication, December 4, 2012; 10.1038/mt.2012.251 Abstract | Full Text Aging-related Decrease of Human ASC Angiogenic Potential Is Reversed by Hypoxia Preconditioning Through ROS Production Sandra De Barros, Stéphanie Dehez, Emmanuelle Arnaud, Corinne Barreau, Alexandre Cazavet, Guillaume Perez, Anne Galinier, Louis Casteilla and Valérie Planat-Bénard Mol Ther 2013 21: 399-408; advance online publication, October 16, 2012; 10.1038/mt.2012.213 Abstract | Full Text IFN-γ-induced iNOS Expression in Mouse Regulatory Macrophages Prolongs Allograft Survival in Fully Immunocompetent Recipients Paloma Riquelme, Stefan Tomiuk, Anja Kammler, Fred Fändrich, Hans J Schlitt, Edward K Geissler and James A Hutchinson Mol Ther 2013 21: 409-422; advance online publication, August 28, 2012; 10.1038/mt.2012.168 Abstract | Full Text Hematopoietic Stem Cell Regeneration Enhanced by Ectopic Expression of ROS-detoxifying Enzymes in Transplant Mice Weimin Miao, Richard XuFeng, Moo-Rim Park, Haihui Gu, Linping Hu, Jin Wook Kang, Shihui Ma, Paulina H Liang, Yanxin Li, Haizi Cheng, Hui Yu, Michael Epperly, Joel Greenberger and Tao Cheng Mol Ther 2013 21: 423-432; advance online publication, January 8, 2013; 10.1038/mt.2012.232 Abstract | Full Text Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis Frank Harrison, Brian A Yeagy, Celine J Rocca, Donald B Kohn, Daniel R Salomon and Stephanie Cherqui Mol Ther 2013 21: 433-444; advance online publication, October 23, 2012; 10.1038/mt.2012.214 Abstract | Full Text Adipose-derived Stromal Cells Overexpressing Vascular Endothelial Growth Factor Accelerate Mouse Excisional Wound Healing Allison Nauta, Catharina Seidel, Lorenzo Deveza, Daniel Montoro, Monica Grova, Sae Hee Ko, Jeong Hyun, Geoffrey C Gurtner, Michael T Longaker and Fan Yang Mol Ther 2013 21: 445-455; advance online publication, November 20, 2012; 10.1038/mt.2012.234 Abstract | Full Text Gene-modified Mesenchymal Stem Cells Protect Against Radiation-induced Lung Injury Jianxin Xue, Xin Li, You Lu, Lu Gan, Lin Zhou, Yongsheng Wang, Jie Lan, Shurui Liu, Lan Sun, Li Jia, Xianming Mo and Jian Li Mol Ther 2013 21: 456-465; advance online publication, November 13, 2012; 10.1038/mt.2012.183 Abstract | Full Text Graft-versus-leukemia Effect of HLA-haploidentical Central-memory T-cells Expanded With Leukemic APCs and Modified With a Suicide Gene Monica Casucci, Serena Kimi Perna, Laura Falcone, Barbara Camisa, Zulma Magnani, Massimo Bernardi, Alessandro Crotta, Cristina Tresoldi, Katharina Fleischhauer, Maurilio Ponzoni, Silvia Gregori, Federico Caligaris Cappio, Fabio Ciceri, Claudio Bordignon, Alessandro Cignetti, Attilio Bondanza and Chiara Bonini Mol Ther 2013 21: 466-475; advance online publication, November 13, 2012; 10.1038/mt.2012.227 Abstract | Full Text Quality Controls in Cellular Immunotherapies: Rapid Assessment of Clinical Grade Dendritic Cells by Gene Expression Profiling Luciano Castiello, Marianna Sabatino, Yingdong Zhao, Barbara Tumaini, Jiaqiang Ren, Jin Ping, Ena Wang, Lauren V Wood, Francesco M Marincola, Raj K Puri and David F Stroncek Mol Ther 2013 21: 476-484; advance online publication, November 13, 2012; 10.1038/mt.2012.89 Abstract | Full Text Virus-like Particles Containing Multiple M2 Extracellular Domains Confer Improved Cross-protection Against Various Subtypes of Influenza Virus Min-Chul Kim, Jae-Min Song, Eunju O, Young-Man Kwon, Youn-Jeong Lee, Richard W Compans and Sang-Moo Kang Mol Ther 2013 21: 485-492; advance online publication, December 18, 2012; 10.1038/mt.2012.246 Abstract | Full Text Corrigendum Top Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles Mol Ther 2013 21: 493; 10.1038/mt.2012.275 Full Text Design and Selection of Toca 511 for Clinical Use: Modified Retroviral Replicating Vector With Improved Stability and Gene Expression Mol Ther 2013 21: 494; 10.1038/mt.2013.6 Full Text Erratum Top Erratum to “Quality Controls in Cellular Immunotherapies: Rapid Assessment of Clinical Grade Dendritic Cells by Gene Expression Profiling” Mol Ther 2013 21: 495; 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