Molecular Therapy - Table of Contents alert Volume 24 Issue 6

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TABLE OF CONTENTS Volume 24, Issue 6 (June 2016) In this issue Editorial In This Issue Research Highlights Commentary Original Articles Corrigendum Also new AOP Sign up for e-alerts Recommend to your library Web feed Subscribe   Advertisement The Fred Hutchinson Cancer Research Center (FHCRC) is recruiting a full-time faculty member at the Assistant or Associate Member rank on the Physician/Scientist pathway in the Clinical Research Division.  Learn more & Apply   Editorial Top ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization Seppo Ylä-Herttuala Mol Ther 2016 24: 1013-1014; 10.1038/mt.2016.98 Full Text In This Issue Top In This Issue Mol Ther 2016 24: 1015; 10.1038/mt.2016.99 Full Text Research Highlights Top Research Highlights Mol Ther 2016 24: 1016; 10.1038/mt.2016.100 Full Text Commentary Top Timing of Gene Therapy Interventions: The Earlier, the Better Steven J Gray Mol Ther 2016 24: 1017-1018; 10.1038/mt.2016.20 Full Text Original Articles Top CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher DiseaseMTOPEN John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard and Seng H Cheng Mol Ther 2016 24: 1019-1029; advance online publication, March 7, 2016; 10.1038/mt.2016.53 Abstract | Full Text rAAV Gene Therapy in a Canavan’s Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System Seemin Seher Ahmed, Stefan A Schattgen, Ashley E Frakes, Elif M Sikoglu, Qin Su, Jia Li, Thomas G Hampton, Andrew R Denninger, Daniel A Kirschner, Brian Kaspar, Reuben Matalon and Guangping Gao Mol Ther 2016 24: 1030-1041; advance online publication, April 4, 2016; 10.1038/mt.2016.68 Abstract | Full Text Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid Koen Vercauteren, Brad E Hoffman, Irene Zolotukhin, Geoffrey D Keeler, Jing W Xiao, Etiena Basner-Tschakarjan, Katherine A High, Hildegund CJ Ertl, Charles M Rice, Arun Srivastava, Ype P de Jong and Roland W Herzog Mol Ther 2016 24: 1042-1049; advance online publication, March 29, 2016; 10.1038/mt.2016.61 Abstract | Full Text Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries Jakob Körbelin, Timo Sieber, Stefan Michelfelder, Lars Lunding, Elmar Spies, Agnes Hunger, Malik Alawi, Kleopatra Rapti, Daniela Indenbirken, Oliver J Müller, Renata Pasqualini, Wadih Arap, Jürgen A Kleinschmidt and Martin Trepel Mol Ther 2016 24: 1050-1061; advance online publication, March 28, 2016; 10.1038/mt.2016.62 Abstract | Full Text Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector Wei Huang, Travis McMurphy, Xianglan Liu, Chuansong Wang and Lei Cao Mol Ther 2016 24: 1062-1069; advance online publication, February 9, 2016; 10.1038/mt.2016.34 Abstract | Full Text Engineering Periodic shRNA for Enhanced Silencing Efficacy Connie Wu, Kevin E Shopsowitz and Paula T Hammond Mol Ther 2016 24: 1070-1077; advance online publication, April 7, 2016; 10.1038/mt.2016.69 Abstract | Full Text Stable, Nonviral Expression of Mutated Tumor Neoantigen-specific T-cell Receptors Using the Sleeping Beauty Transposon/Transposase System Drew C Deniger, Anna Pasetto, Eric Tran, Maria R Parkhurst, Cyrille J Cohen, Paul F Robbins, Laurence JN Cooper and Steven A Rosenberg Mol Ther 2016 24: 1078-1089; advance online publication, March 5, 2016; 10.1038/mt.2016.51 Abstract | Full Text Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured ThymocytesMTOPEN Sheng Zhou, Soghra Fatima, Zhijun Ma, Yong-Dong Wang, Taihe Lu, Laura J Janke, Yang Du and Brian P Sorrentino Mol Ther 2016 24: 1090-1099; advance online publication, March 9, 2016; 10.1038/mt.2016.55 Abstract | Full Text Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients Irene Gil-Farina, Raffaele Fronza, Christine Kaeppel, Esperanza Lopez-Franco, Valerie Ferreira, Delia D’Avola, Alberto Benito, Jesus Prieto, Harald Petry, Gloria Gonzalez-Aseguinolaza and Manfred Schmidt Mol Ther 2016 24: 1100-1105; advance online publication, March 7, 2016; 10.1038/mt.2016.52 Abstract | Full Text Targeted Delivery of C/EBPα -saRNA by Pancreatic Ductal Adenocarcinoma-specific RNA Aptamers Inhibits Tumor Growth In VivoMTOPEN Sorah Yoon, Kai-Wen Huang, Vikash Reebye, Paul Mintz, Yu-Wen Tien, Hong-Shiee Lai, Pål Sætrom, Isabella Reccia, Piotr Swiderski, Brian Armstrong, Agnieszka Jozwiak, Duncan Spalding, Long Jiao, Nagy Habib and John J Rossi Mol Ther 2016 24: 1106-1116; advance online publication, March 17, 2016; 10.1038/mt.2016.60 Abstract | Full Text Protein Kinase C-α is a Critical Protein for Antisense Oligonucleotide-mediated Silencing in Mammalian Cells Daniela Castanotto, Min Lin, Claudia Kowolik, Troels Koch, Bo Rode Hansen, Henrik Oerum and Cy A Stein Mol Ther 2016 24: 1117-1125; advance online publication, March 10, 2016; 10.1038/mt.2016.54 Abstract | Full Text Allospecific Tregs Expanded After Anergization Remain Suppressive in Inflammatory Conditions but Lack Expression of Gut-homing MoleculesMTOPEN Eleni Kotsiou, John G Gribben and Jeff K Davies Mol Ther 2016 24: 1126-1134; advance online publication, April 6, 2016; 10.1038/mt.2016.64 Abstract | Full Text GD2-specific CAR T Cells Undergo Potent Activation and Deletion Following Antigen Encounter but can be Protected From Activation-induced Cell Death by PD-1 Blockade Tessa Gargett, Wenbo Yu, Gianpietro Dotti, Eric S Yvon, Susan N Christo, John D Hayball, Ian D Lewis, Malcolm K Brenner and Michael P Brown Mol Ther 2016 24: 1135-1149; advance online publication, March 29, 2016; 10.1038/mt.2016.63 Abstract | Full Text Randomized Phase 2 Trial of the Oncolytic Virus Pelareorep (Reolysin) in Upfront Treatment of Metastatic Pancreatic Adenocarcinoma Anne M Noonan, Matthew R Farren, Susan M Geyer, Ying Huang, Sanaa Tahiri, Daniel Ahn, Sameh Mikhail, Kristen K Ciombor, Shubham Pant, Santiago Aparo, Jennifer Sexton, John L Marshall, Thomas A Mace, Christina S Wu, Bassel El-Rayes, Cynthia D Timmers, James Zwiebel, Gregory B Lesinski, Miguel A Villalona-Calero and Tanios S Bekaii-Saab Mol Ther 2016 24: 1150-1158; advance online publication, April 4, 2016; 10.1038/mt.2016.66 Abstract | Full Text Corrigendum Top Corrigendum to “Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome” Mol Ther 2016 24: 1159; 10.1038/mt.2016.96 Full Text Corrigendum to “T-cell Subsets in Peripheral Blood and Tumors of Patients Treated With Oncolytic Adenoviruses” Mol Ther 2016 24: 1159; 10.1038/mt.2016.65 Full Text Advertisement Molecular Therapy - Methods & Clinical Development Special Issue: Production of Gene Therapy Vectors As gene therapy and related molecular medicines progress from bench to bedside and from small early clinical studies to larger trials, production of these novel drugs has become a key area of research and development. The articles in this open access special issue cover innovative technologies that are being developed in academic centers and in the biotech sector for manufacturing of gene therapy vectors and nucleotide-based drugs. Explore the complete special issue in full. Please note that you need to be a subscriber or site-license holder to enjoy full-text access to Molecular Therapy. In order to do so, please purchase a subscription. You have been sent this Table of Contents Alert because you have opted in to receive it. You can change or discontinue your e-mail alerts at any time, by modifying your preferences on your nature.com account at: www.nature.com/nams/svc/myaccount (You will need to log in to be recognised as a nature.com registrant). For further technical assistance, please contact our registration department. For print subscription enquiries, please contact our subscription department. For other enquiries, please contact our customer feedback department. 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