Submit your best article to Molecular Therapy (MT) Impact Factor 7.041* Submission Benefits at a glance include: - Quick and easy online submission - Fast editorial turnaround - Advance Online Publication within 28 business days of acceptance - Open Access option - Inclusion in the leading abstracting and indexing services - High visibility *2012 Journal Citation Report (Thomson Reuters, 2013) | | | | | | Editorial | Top | | Pathway for Approval of a Gene Therapy Orphan Product: Treading New GroundBarry J Byrne Mol Ther 2013 21: 1465-1466; 10.1038/mt.2013.157 Full Text | | In This Issue | Top | | In This IssueMol Ther 2013 21: 1468; 10.1038/mt.2013.158 Full Text | | Research Highlights | Top | | Research HighlightsMol Ther 2013 21: 1469; 10.1038/mt.2013.159 Full Text | | Letter to the Editor | Top | | Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9Casey A Maguire, Matheus HW Crommentuijn, Dakai Mu, Eloise Hudry, Alberto Serrano-Pozo, Bradley T Hyman and Bakhos A Tannous Mol Ther 2013 21: 1470-1471; 10.1038/mt.2013.95 Full Text | | Commentary | Top | | Immunotherapy Exposes Cancer Stem Cell Resistance and a New Synthetic LethalityLaszlo Radvanyi Mol Ther 2013 21: 1472-1474; 10.1038/mt.2013.160 Full Text | | | | Toward Position-independent Retroviral Vector Expression in Pluripotent Stem CellsAxel Schambach and Thomas Moritz Mol Ther 2013 21: 1474-1477; 10.1038/mt.2013.161 Full Text | | Review | Top | | Managing MicroRNAs with Vector-Encoded Decoy-Type InhibitorsRasmus O Bak, Anne Kruse Hollensen and Jacob Giehm Mikkelsen Mol Ther 2013 21: 1478-1485; advance online publication, June 11, 2013; 10.1038/mt.2013.113 Abstract | Full Text | | Original Articles | Top | | Viral Delivery of Antioxidant Genes as a Therapeutic Strategy in Experimental Models of Amyotrophic Lateral SclerosisMTOpenAikaterini Nanou, Adrian Higginbottom, Chiara F Valori, Matthew Wyles, Ke Ning, Pamela Shaw and Mimoun Azzouz Mol Ther 2013 21: 1486-1496; advance online publication, June 4, 2013; 10.1038/mt.2013.115 Abstract | Full Text | | | | Acat1 Knockdown Gene Therapy Decreases Amyloid-β in a Mouse Model of Alzheimer’s DiseaseStephanie R Murphy, Catherine CY Chang, Godwin Dogbevia, Elena Y Bryleva, Zachary Bowen, Mazahir T Hasan and Ta-Yuan Chang Mol Ther 2013 21: 1497-1506; advance online publication, June 18, 2013; 10.1038/mt.2013.118 Abstract | Full Text | | | | Functional Cloning of Recurrence-specific Antigens Identifies Molecular Targets to Treat Tumor RelapseNicolas Boisgerault, Timothy Kottke, Jose Pulido, Jill Thompson, Rosa Maria Diaz, Diana Rommelfanger-Konkol, Addie Embry, Dyana Saenz, Eric Poeschla, Hardev Pandha, Kevin Harrington, Alan Melcher, Peter Selby and Richard Vile Mol Ther 2013 21: 1507-1516; advance online publication, June 11, 2013; 10.1038/mt.2013.116 Abstract | Full Text | | | | Systemic Delivery of SapC-DOPS Has Antiangiogenic and Antitumor Effects Against GlioblastomaJeffrey Wojton, Zhengtao Chu, Haritha Mathsyaraja, Walter H Meisen, Nicholas Denton, Chang-Hyuk Kwon, Lionel ML Chow, Mary Palascak, Robert Franco, Tristan Bourdeau, Sherry Thornton, Michael C Ostrowski, Balveen Kaur and Xiaoyang Qi Mol Ther 2013 21: 1517-1525; advance online publication, June 4, 2013; 10.1038/mt.2013.114 Abstract | Full Text | | | | Minicircle DNA is Superior to Plasmid DNA in Eliciting Antigen-specific CD8+ T-cell ResponsesWynette M Dietz, Nicole E B Skinner, Sara E Hamilton, Michelle D Jund, Suzanne M Heitfeld, Adam J Litterman, Patrick Hwu, Zhi-Ying Chen, Andres M Salazar, John R Ohlfest, Bruce R Blazar, Christopher A Pennell and Mark J Osborn Mol Ther 2013 21: 1526-1535; advance online publication, May 21, 2013; 10.1038/mt.2013.85 Abstract | Full Text | | | | Kinetics and Epigenetics of Retroviral Silencing in Mouse Embryonic Stem Cells Defined by Deletion of the D4Z4 ElementMTOpenSylvie Rival-Gervier, Mandy YM Lo, Shahryar Khattak, Peter Pasceri, Matthew C Lorincz and James Ellis Mol Ther 2013 21: 1536-1550; advance online publication, June 11, 2013; 10.1038/mt.2013.131 Abstract | Full Text | | | | AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping TherapyMaëva Le Hir, Aurélie Goyenvalle, Cécile Peccate, Guillaume Précigout, Kay E Davies, Thomas Voit, Luis Garcia and Stéphanie Lorain Mol Ther 2013 21: 1551-1558; advance online publication, June 11, 2013; 10.1038/mt.2013.121 Abstract | Full Text | | | | Codelivery of VEGF siRNA and Gemcitabine Monophosphate in a Single Nanoparticle Formulation for Effective Treatment of NSCLCYuan Zhang, Nicole MJ Schwerbrock, Arlin B Rogers, William Y Kim and Leaf Huang Mol Ther 2013 21: 1559-1569; advance online publication, June 18, 2013; 10.1038/mt.2013.120 Abstract | Full Text | | | | Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi TherapeuticsMartin A Maier, Muthusamy Jayaraman, Shigeo Matsuda, Ju Liu, Scott Barros, William Querbes, Ying K Tam, Steven M Ansell, Varun Kumar, June Qin, Xuemei Zhang, Qianfan Wang, Sue Panesar, Renta Hutabarat, Mary Carioto, Julia Hettinger, Pachamuthu Kandasamy, David Butler, Kallanthottathil G Rajeev, Bo Pang, Klaus Charisse, Kevin Fitzgerald, Barbara L Mui, Xinyao Du, Pieter Cullis, Thomas D Madden, Michael J Hope, Muthiah Manoharan and Akin Akinc Mol Ther 2013 21: 1570-1578; advance online publication, June 25, 2013; 10.1038/mt.2013.124 Abstract | Full Text | | | | Recovery From Experimental Parkinsonism by Semaphorin-guided Axonal Growth of Grafted Dopamine NeuronsN Emmanuel Díaz-Martínez, Elisa Tamariz, N Fabián Díaz, Claudia M García-Peña, Alfredo Varela-Echavarría and Iván Velasco Mol Ther 2013 21: 1579-1591; advance online publication, June 4, 2013; 10.1038/mt.2013.78 Abstract | Full Text | | | | Genetically Engineered Human Islets Protected From CD8-mediated Autoimmune Destruction In VivoArnaud Zaldumbide, Gonnie Alkemade, Françoise Carlotti, Tatjana Nikolic, Joana RF Abreu, Marten A Engelse, Anja Skowera, Eelco J de Koning, Mark Peakman, Bart O Roep, Rob C Hoeben and Emmanuel JHJ Wiertz Mol Ther 2013 21: 1592-1601; advance online publication, May 21, 2013; 10.1038/mt.2013.105 Abstract | Full Text | | | | Synergistic Effects of GDNF and VEGF on Lifespan and Disease Progression in a Familial ALS Rat ModelDan Krakora, Patrick Mulcrone, Michael Meyer, Christina Lewis, Ksenija Bernau, Genevieve Gowing, Chad Zimprich, Patrick Aebischer, Clive N Svendsen and Masatoshi Suzuki Mol Ther 2013 21: 1602-1610; advance online publication, May 28, 2013; 10.1038/mt.2013.108 Abstract | Full Text | | | | Antitumor Effects of Chimeric Receptor Engineered Human T Cells Directed to Tumor StromaSunitha Kakarla, Kevin KH Chow, Melinda Mata, Donald R Shaffer, Xiao-Tong Song, Meng-Fen Wu, Hao Liu, Lisa L Wang, David R Rowley, Klaus Pfizenmaier and Stephen Gottschalk Mol Ther 2013 21: 1611-1620; advance online publication, June 4, 2013; 10.1038/mt.2013.110 Abstract | Full Text | | | | Systemic Delivery of Fusogenic Membrane Glycoprotein-expressing Neural Stem Cells to Selectively Kill Tumor CellsDetu Zhu, Dang Hoang Lam, Yovita Ida Purwanti, Sal Lee Goh, Chunxiao Wu, Jieming Zeng, Weimin Fan and Shu Wang Mol Ther 2013 21: 1621-1630; advance online publication, June 11, 2013; 10.1038/mt.2013.123 Abstract | Full Text | | | | Stem Cell–based Therapy for Prevention of Delayed Fracture Union: A Randomized and Prospective Preliminary StudyMeir Liebergall, Josh Schroeder, Rami Mosheiff, Zulma Gazit, Zilberman Yoram, Linda Rasooly, Anat Daskal, Amal Khoury, Yoram Weil and Shaul Beyth Mol Ther 2013 21: 1631-1638; advance online publication, June 4, 2013; 10.1038/mt.2013.109 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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