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1) Role of HCP5-miR-139-RUNX1 Feedback Loop in Regulating Malignant Behavior of Glioma Cells (Molecular Therapy) Tuesday July 19th 2016
Hao Teng, Ping Wang, Yixue Xue, Xiaobai Liu, Jun Ma, Heng Cai, Zhuo Xi, Zhen Li, Yunhui Liu

2) Long Non-coding RNA BGas Regulates the Cystic Fibrosis Transmembrane Conductance Regulator (Molecular Therapy) Tuesday July 19th 2016
Sheena M Saayman, Amanda Ackley, Jon Burdach, Matthew Clemson, Dieter C Gruenert, Kiyoshi Tachikawa, Pad Chivukula, Marc S Weinberg, Kevin V Morris

3) Development of an Attenuated Tat Protein as a Highly-effective Agent to Specifically Activate HIV-1 Latency (Molecular Therapy) Tuesday July 19th 2016
Guannan Geng, Bingfeng Liu, Cancan Chen, Kang Wu, Jun Liu, Yijun Zhang, Ting Pan, Jun Li, Yue Yin, Junsong Zhang, Feng Huang, Fei Yu, Jingliang Chen, Xiancai Ma, Jie Zhou, Ersheng Kuang, Chao Liu, Weiping Cai, Hui Zhang

4) Extracellular Antibody Drug Conjugates Exploiting the Proximity of Two Proteins (Molecular Therapy) Tuesday July 19th 2016
David J Marshall, Scott S Harried, John L Murphy, Chad A Hall, Mohammed S Shekhani, Christophe Pain, Conner A Lyons, Antonella Chillemi, Fabio Malavasi, Homer L Pearce, Jon S Thorson, James R Prudent

5) Inducible HGF-secreting Human Umbilical Cord Blood-derived MSCs Produced via TALEN-mediated Genome Editing Promoted Angiogenesis (Molecular Therapy) Tuesday July 19th 2016
Hyun-Kyung Chang, Pyung-Hwan Kim, Hyun-Min Cho, Soo-Young Yum, Young-Jin Choi, YeonSung Son, DaBin Lee, InSung Kang, Kyung-Sun Kang, Goo Jang, Je-Yoel Cho

6) Intratumoral Infection with Murine Cytomegalovirus Synergizes with PD-L1 Blockade to Clear Melanoma Lesions and Induce Long-term Immunity (Molecular Therapy) Tuesday July 19th 2016
Dan A Erkes, Guangwu Xu, Constantine Daskalakis, Katherine A Zurbach, Nicole A Wilski, Toktam Moghbeli, Ann B Hill, Christopher M Snyder

7) Coadministration of a Plasmid Encoding HIV-1 Gag Enhances the Efficacy of Cancer DNA Vaccines (Molecular Therapy) Tuesday July 19th 2016
Laure Lambricht, Kevin Vanvarenberg, Ans De Beuckelaer, Lien Van Hoecke, Johan Grooten, Bernard Ucakar, Pascale Lipnik, Niek N Sanders, Stefan Lienenklaus, V, Ga

8) Anti-apoptotic Effects of Human Wharton (Molecular Therapy) Tuesday July 19th 2016
SooJin Kwon, Soo Mi Ki, Sang Eon Park, Min-Jeong Kim, Brian Hyung, Na Kyung Lee, Sangmi Shim, Byung-Ok Choi, Duk L Na, Ji Eun Lee, Jong Wook Chang

9) Specific Delivery of MiRNA for High Efficient Inhibition of Prostate Cancer by RNA Nanotechnology (Molecular Therapy) Tuesday July 19th 2016
Daniel W Binzel, Yi Shu, Hui Li, Meiyan Sun, Qunshu Zhang, Dan Shu, Bin Guo, Peixuan Guo

10) Three-year Follow up of GMCSF/bi-shRNAfurin DNA-transfected Autologous Tumor Immunotherapy (Vigil) in Metastatic Advanced Ewing (Molecular Therapy) Tuesday July 19th 2016
Maurizio Ghisoli, Minal Barve, Robert Mennel, Carl Lenarsky, Staci Horvath, Gladice Wallraven, Beena O Pappen, Sam Whiting, Donald Rao, Neil Senzer, John Nemunaitis

11) Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing (Molecular Therapy) Tuesday July 19th 2016
Donald D. Rao, Christopher Jay, Zhaohui Wang, Xiuquan Luo, Padmasini Kumar, Hilary Eysenbach, Maurizio Ghisoli, Neil Senzer, John Nemunaitis

12) Phase 1 Study of Intravenous Oncolytic Poxvirus (vvDD) in Patients With Advanced Solid Cancers (Molecular Therapy) Tuesday July 12th 2016
Stephanie Downs-Canner, Zong Sheng Guo, Roshni Ravindranathan, Caroline J Breitbach, Mark E O, Heather L Jones, Anne Moon, Judith Andrea McCart, Yongli Shuai, Herbert J Zeh, David L Bartlett

13) Neural Stem Cells Engineered to Express Three Therapeutic Factors Mediate Recovery from Chronic Stage CNS Autoimmunity (Molecular Therapy) Tuesday July 12th 2016
Xing Li, Yuan Zhang, Yaping Yan, Bogoljub Ciric, Cun-Gen Ma, Bruno Gran, Mark Curtis, Abdolmohamad Rostami, Guang-Xian Zhang

14) Structural Determinants of Sleeping Beauty Transposase Activity (Molecular Therapy) Tuesday July 12th 2016
Gy, Stephen R Yant, Andr, Joseph A Marsh, Lajos M, Zsuzsanna Izsv, Orsolya Barab, Zolt

15) Elimination of Latently HIV-infected Cells from Antiretroviral Therapy-suppressed Subjects by Engineered Immune-mobilizing T-cell Receptors (Molecular Therapy) Tuesday July 12th 2016
Hongbing Yang, Sandrine Buisson, Giovanna Bossi, Zo, Gemma Hancock, Chun So, Rebecca Ashfield, Annelise Vuidepot, Tara Mahon, Peter Molloy, Joanne Oates, Samantha J Paston, Milos Aleksic, Namir J Hassan, Bent K Jakobsen, Lucy Dorrell

16) CD123-Engager T Cells as a Novel Immunotherapeutic for Acute Myeloid Leukemia (Molecular Therapy) Tuesday July 12th 2016
Challice L Bonifant, Arpad Szoor, David Torres, Nicholos Joseph, Mireya Paulina Velasquez, Kota Iwahori, Amos Gaikwad, Phuong Nguyen, Caroline Arber, Xiao-Tong Song, Michele Redell, Stephen Gottschalk

17) Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics (Molecular Therapy) Wednesday July 6th 2016
Jennifer CJ Chen, Oliver D King, Yuanfan Zhang, Nicholas P Clayton, Carrie Spencer, Bruce M Wentworth, Charles P Emerson, Kathryn R Wagner

18) Oral Delivery of Protein Drugs Bioencapsulated in Plant Cells (Molecular Therapy) Wednesday July 6th 2016
Kwang-Chul Kwon, Henry Daniell

19) Boosting Central Nervous System Axon Regeneration by Circumventing Limitations of Natural Cytokine Signaling (Molecular Therapy) Tuesday June 28th 2016
Marco Leibinger, Anastasia Andreadaki, Philipp Gobrecht, Evgeny Levin, Heike Diekmann, Dietmar Fischer

20) High Efficiency CRISPR/Cas9-mediated Gene Editing in Primary Human T-cells Using Mutant Adenoviral E4orf6/E1b55k (Molecular Therapy) Tuesday June 28th 2016
Kamila S Gwiazda, Alexandra E Grier, Jaya Sahni, Stephen M Burleigh, Unja Martin, Julia G Yang, Nicholas A Popp, Michelle C Krutein, Iram F Khan, Kyle Jacoby, Michael C Jensen, David J Rawlings, Andrew M Scharenberg

21) CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa (Molecular Therapy) Tuesday June 28th 2016
Wen-Hsuan Wu, Yi-Ting Tsai, Sally Justus, Ting-Ting Lee, Lijuan Zhang, Chyuan-Sheng Lin, Alexander G Bassuk, Vinit B Mahajan, Stephen H Tsang

22) Targeting of Discoidin Domain Receptor 2 (DDR2) Prevents Myofibroblast Activation and Neovessel Formation During Pulmonary Fibrosis (Molecular Therapy) Tuesday June 28th 2016
Hu Zhao, Huan Bian, Xin Bu, Shuya Zhang, Pan Zhang, Jiangtian Yu, Xiaofeng Lai, Di Li, Chuchao Zhu, Libo Yao, Jin Su

23) Superior Therapeutic Index in Lymphoma Therapy: CD30+ CD34+ Hematopoietic Stem Cells Resist a Chimeric Antigen Receptor T-cell Attack (Molecular Therapy) Tuesday June 28th 2016
Andreas A Hombach, Andr, Markus Chmielewski, Florian Murke, Janine Kimpel, Bernd Giebel, Hinrich Abken

24) Safety and Immunogenicity of ChAd63 and MVA ME-TRAP in West African Children and Infants (Molecular Therapy) Tuesday June 28th 2016
Muhammed O Afolabi, Alfred B Tiono, Uche J Adetifa, Jean Baptiste Yaro, Abdoulie Drammeh, Issa N, Carly Bliss, Susanne H Hodgson, Nicholas A Anagnostou, Guillaume S Sanou, Ya Jankey Jagne, Oumarou Ouedraogo, Casimir Tamara, Nicolas Ouedraogo, Mirielle Ouedraogo, Jainaba Njie-Jobe, Amidou Diarra, Christopher JA Duncan, Riccardo Cortese, Alfredo Nicosia, Rachel Roberts, Nicola K Viebig, Odile Leroy, Alison M Lawrie, Katie L Flanagan, Beate Kampman, Philip Bejon, Egeruan B Imoukhuede, Katie J Ewer, Adrian VS Hill, Kalifa Bojang, Sodiomon B Sirima

25) Sequence-defined cMET/HGFR-targeted Polymers as Gene Delivery Vehicles for the Theranostic Sodium Iodide Symporter (NIS) Gene (Molecular Therapy) Tuesday June 28th 2016
Sarah Urnauer, Stephan Morys, Ana Krhac Levacic, Andrea M M, Christina Schug, Kathrin A Schmohl, Nathalie Schwenk, Christian Zach, Janette Carlsen, Peter Bartenstein, Ernst Wagner, Christine Spitzweg

26) Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy (Molecular Therapy) Tuesday June 28th 2016
Yuanzheng Gao, Xiuming Guo, Katherine Santostefano, Yanlin Wang, Tammy Reid, Desmond Zeng, Naohiro Terada, Tetsuo Ashizawa, Guangbin Xia

27) Strand and Cell Type-specific Function of microRNA-126 in Angiogenesis (Molecular Therapy) Tuesday June 21st 2016
Qinbo Zhou, Chastain Anderson, Jakub Hanus, Fangkun Zhao, Jing Ma, Akihiko Yoshimura, Shusheng Wang

28) Mesenchymal Stem Cells Deliver Exogenous MicroRNA-let7c via Exosomes to Attenuate Renal Fibrosis (Molecular Therapy) Tuesday June 21st 2016
Bo Wang, Kevin Yao, Brooke M Huuskes, Hsin-Hui Shen, Junli Zhuang, Catherine Godson, Eoin P Brennan, Jennifer L Wilkinson-Berka, Andrea F Wise, Sharon D Ricardo

29) Complete Spectrum of CRISPR/Cas9-induced Mutations on HBV cccDNA (Molecular Therapy) Tuesday June 21st 2016
Christoph Seeger, Ji A Sohn

30) Translational Implications for Off-the-shelf Immune Cells Expressing Chimeric Antigen Receptors (Molecular Therapy) Tuesday June 14th 2016
Hiroki Torikai, Laurence JN Cooper

31) Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency (Molecular Therapy) Tuesday June 14th 2016
Maria Garcia-Gomez, Andrea Calabria, Maria Garcia-Bravo, Fabrizio Benedicenti, Penelope Kosinski, Sergio L, Collin Hill, Mar, Miguel A Mart, Israel Orman, Joan-LLuis Vives-Corrons, Charles Kung, Axel Schambach, Shengfang Jin, Juan A Bueren, Eugenio Montini, Susana Navarro, Jose C Segovia

32) Enhanced ADCC and NK Cell Activation of an Anticarcinoma Bispecific Antibody by Genetic Insertion of a Modified IL-15 Cross-linker (Molecular Therapy) Tuesday June 14th 2016
Joerg U Schmohl, Martin Felices, Elizabeth Taras, Jeff S Miller, Daniel A Vallera

33) Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform (Molecular Therapy) Tuesday June 14th 2016
Juwita H, Shahabuddin S Hoseini, Julia D Suerth, Dirk Hoffmann, Marcel Maluski, Jessica Herbst, Holger Maul, Arnab Ghosh, Britta Eiz-Vesper, Qinggong Yuan, Michael Ott, Michael Heuser, Axel Schambach, Martin G Sauer

34) In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy (Molecular Therapy) Tuesday June 7th 2016
Sourav R Choudhury, Zachary Fitzpatrick, Anne F Harris, Stacy A Maitland, Jennifer S Ferreira, Yuanfan Zhang, Shan Ma, Rohit B Sharma, Heather L Gray-Edwards, Jacob A Johnson, Aime K Johnson, Laura C Alonso, Claudio Punzo, Kathryn R Wagner, Casey A Maguire, Robert M Kotin, Douglas R Martin, Miguel Sena-Esteves

35) Rapid and Efficient Stable Gene Transfer to Mesenchymal Stromal Cells Using a Modified Foamy Virus Vector (Molecular Therapy) Tuesday June 7th 2016
Nathan Paul Sweeney, Cathy Regan, Jiahui Liu, Antonio Galleu, Francesco Dazzi, Dirk Lindemann, Charles Anthony Rupar, Myra Olga McClure

36) Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy (Molecular Therapy) Tuesday June 7th 2016
Olivier Bornert, Tobias K, Jeroen Bremer, Peter C van den Akker, Anna MG Pasmooij, Alexander Nystr

37) Development of Third-generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-cells (Molecular Therapy) Tuesday May 24th 2016
Olivier Humbert, Don W. Gisch, Martin E. Wohlfahrt, Amie B. Adams, Phil D. Greenberg, Tom M. Schmitt, Grant D. Trobridge, Hans-Peter Kiem

38) Development of Novel Small Hairpin RNAs That do not Require Processing by Dicer or AGO2 (Molecular Therapy) Tuesday May 24th 2016
Shin-ichiro Ohno, Karen Itano, Yuichirou Harada, Koutaro Asada, Keiki Oikawa, Mikie Kashiwazako, Hikaru Okuyama, Katsuyoshi Kumagai, Masakatsu Takanashi, Katsuko Sudo, Norihiko Ikeda, Masahiko Kuroda

39) Vastatin, an Endogenous Antiangiogenesis Polypeptide That Is Lost in Hepatocellular Carcinoma, Effectively Inhibits Tumor Metastasis (Molecular Therapy) Tuesday May 17th 2016
Zan Shen, Chen Yao, Zifeng Wang, Lu Yue, Zheping Fang, Hong Yao, Feng Lin, Hui Zhao, Yuan-Jue Sun, Xiu-wu Bian, Wenqi Jiang, Xiaomei Wang, Yi Li, Gang Lu, Wai Sang Poon, Hsiang-Fu Kung, Marie Chia-mi Lin

40) Engineering Periodic shRNA for Enhanced Silencing Efficacy (Molecular Therapy) Tuesday May 10th 2016
Connie Wu, Kevin E Shopsowitz, Paula T Hammond

41) CRNDE Promotes Malignant Progression of Glioma by Attenuating miR-384/PIWIL4/STAT3 Axis (Molecular Therapy) Tuesday May 10th 2016
Jian Zheng, Xiaobai Liu, Ping Wang, Yixue Xue, Jun Ma, Chengbin Qu, Yunhui Liu

42) Allospecific Tregs Expanded After Anergization Remain Suppressive in Inflammatory Conditions but Lack Expression of Gut-homing Molecules (Molecular Therapy) Tuesday May 3rd 2016
Eleni Kotsiou, John G Gribben, Jeff K Davies

43) Randomized Phase 2 Trial of the Oncolytic Virus Pelareorep (Reolysin) in Upfront Treatment of Metastatic Pancreatic Adenocarcinoma (Molecular Therapy) Tuesday May 3rd 2016
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, Tanios S Bekaii-Saab

44) Predictive and Prognostic Clinical Variables in Cancer Patients Treated With Adenoviral Oncolytic Immunotherapy (Molecular Therapy) Tuesday May 3rd 2016
Kristian Taipale, Ilkka Liikanen, Anniina Koski, Raita Heiskanen, Anna Kanerva, Otto Hemminki, Minna Oksanen, Susanna Gr, Kari Hemminki, Timo Joensuu, Akseli Hemminki

45) rAAV Gene Therapy in a Canavan (Molecular Therapy) Tuesday May 3rd 2016
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, Guangping Gao

46) Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries (Molecular Therapy) Tuesday April 26th 2016
Jakob K, Timo Sieber, Stefan Michelfelder, Lars Lunding, Elmar Spies, Agnes Hunger, Malik Alawi, Kleopatra Rapti, Daniela Indenbirken, Oliver J M, Renata Pasqualini, Wadih Arap, J, Martin Trepel

47) Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid (Molecular Therapy) Tuesday April 19th 2016
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, Roland W Herzog

48) 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 (Molecular Therapy) Tuesday April 19th 2016
Tessa Gargett, Wenbo Yu, Gianpietro Dotti, Eric S Yvon, Susan N Christo, John D Hayball, Ian D Lewis, Malcolm K Brenner, Michael P Brown

49) Protein Kinase C- (Molecular Therapy) Tuesday April 12th 2016
Daniela Castanotto, Min Lin, Claudia Kowolik, Troels Koch, Bo Rode Hansen, Henrik Oerum, Cy A Stein

50) Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes (Molecular Therapy) Tuesday April 12th 2016
Sheng Zhou, Soghra Fatima, Zhijun Ma, Yong-Dong Wang, Taihe Lu, Laura J Janke, Yang Du, Brian P Sorrentino

51) Targeted Delivery of C/EBP (Molecular Therapy) Tuesday April 12th 2016
Sorah Yoon, Kai-Wen Huang, Vikash Reebye, Paul Mintz, Yu-Wen Tien, Hong-Shiee Lai, P, Isabella Reccia, Piotr Swiderski, Brian Armstrong, Agnieszka Jozwiak, Duncan Spalding, Long Jiao, Nagy Habib, John J Rossi

52) A Role for the Long Noncoding RNA SENCR in Commitment and Function of Endothelial Cells (Molecular Therapy) Tuesday April 5th 2016
Mounia Boulberdaa, Elizabeth Scott, Margaret Ballantyne, Raquel Garcia, Betty Descamps, Gianni D Angelini, Mairi Brittan, Amanda Hunter, Martin McBride, John McClure, Joseph M Miano, Costanza Emanueli, Nicholas L Mills, Joanne C Mountford, Andrew H Baker

53) Stable, Nonviral Expression of Mutated Tumor Neoantigen-specific T-cell Receptors Using the Sleeping Beauty Transposon/Transposase System (Molecular Therapy) Tuesday April 5th 2016
Drew C Deniger, Anna Pasetto, Eric Tran, Maria R Parkhurst, Cyrille J Cohen, Paul F Robbins, Laurence JN Cooper, Steven A Rosenberg

54) Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients (Molecular Therapy) Tuesday April 5th 2016
Irene Gil-Farina, Raffaele Fronza, Christine Kaeppel, Esperanza Lopez-Franco, Valerie Ferreira, Delia D, Alberto Benito, Jesus Prieto, Harald Petry, Gloria Gonzalez-Aseguinolaza, Manfred Schmidt

55) CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease (Molecular Therapy) Tuesday April 5th 2016
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, Seng H Cheng

56) Inhibiting Notch Activity in Breast Cancer Stem Cells by Glucose Functionalized Nanoparticles Carrying (Molecular Therapy) Tuesday March 29th 2016
Veronika Mamaeva, Rasmus Niemi, Michaela Beck, Ezgi, Diti Desai, Sebastian Landor, Tove Gronroos, Pauliina Kronqvist, Ina K N Pettersen, Emmet McCormack, Jessica M Rosenholm, Mika Linden, Cecilia Sahlgren

57) Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics (Molecular Therapy) Tuesday March 22nd 2016
Masayuki Matsui, Thazha P Prakash, David R Corey

58) Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington (Molecular Therapy) Wednesday March 16th 2016
Kari Pollock, Heather Dahlenburg, Haley Nelson, Kyle D Fink, Whitney Cary, Kyle Hendrix, Geralyn Annett, Audrey Torrest, Peter Deng, Joshua Gutierrez, Catherine Nacey, Karen Pepper, Stefanos Kalomoiris, Johnathon D Anderson, Jeannine McGee, William Gruenloh, Brian Fury, Gerhard Bauer, Alexandria Duffy, Theresa Tempkin, Vicki Wheelock, Jan A Nolta

59) Gene Transfer of Brain-derived Neurotrophic Factor (BDNF) Prevents Neurodegeneration Triggered by FXN Deficiency (Molecular Therapy) Wednesday March 16th 2016
Yurika Katsu-Jim, Frida Lor, Juan Carlos Corona, Javier D

60) Chop Deficiency Protects Mice Against Bleomycin-induced Pulmonary Fibrosis by Attenuating M2 Macrophage Production (Molecular Therapy) Wednesday March 16th 2016
Yingying Yao, Yi Wang, Zhijun Zhang, Long He, Jianghui Zhu, Meng Zhang, Xiaoyu He, Zhenshun Cheng, Qilin Ao, Yong Cao, Ping Yang, Yunchao Su, Jianping Zhao, Shu Zhang, Qilin Yu, Qin Ning, Xudong Xiang, Weining Xiong, Cong-Yi Wang, Yongjian Xu

61) Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy (Molecular Therapy) Wednesday March 16th 2016
Soeren Turan, Alfonso P Farruggio, Waracharee Srifa, John W Day, Michele P Calos

62) Progress With Developing Use of Gene Editing To Cure Chronic Infection With Hepatitis B Virus (Molecular Therapy) Wednesday March 16th 2016
Abdullah Ely, Buhle Moyo, Patrick Arbuthnot

63) Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent (Molecular Therapy) Tuesday March 8th 2016
Monir Shababi, Zhihua Feng, Eric Villalon, Christine M Sibigtroth, Erkan Y Osman, Madeline R Miller, Patricka A Williams-Simon, Abby Lombardi, Thalia H Sass, Arleigh K Atkinson, Michael L Garcia, Chien-Ping Ko, Christian L Lorson

64) In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA (Molecular Therapy) Tuesday March 8th 2016
Dustin J Landau, Elizabeth Drake Brooks, Pablo Perez-Pinera, Hiruni Amarasekara, Adam Mefferd, Songtao Li, Andrew Bird, Charles A Gersbach, Dwight D Koeberl

65) Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy (Molecular Therapy) Tuesday March 8th 2016
Eirini P Papapetrou, Axel Schambach

66) Human Mesenchymal Stem Cell Delivery System Modulates Ischemic Cardiac Remodeling With an Increase of Coronary Artery Blood Flow (Molecular Therapy) Tuesday March 1st 2016
Young Sook Lee, Wan Seok Joo, Hyun Soo Kim, Sung Wan Kim

67) H7N9 Live Attenuated Influenza Vaccine Is Highly Immunogenic, Prevents Virus Replication, and Protects Against Severe Bronchopneumonia in Ferrets (Molecular Therapy) Tuesday March 1st 2016
J, Irina Isakova-Sivak, Harry van Dijken, Sanne Spijkers, Justin Mouthaan, Rineke de Jong, Tatiana Smolonogina, Paul Roholl, Larisa Rudenko

68) Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector (Molecular Therapy) Tuesday March 1st 2016
Wei Huang, Travis McMurphy, Xianglan Liu, Chuansong Wang, Lei Cao

69) AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6 (Molecular Therapy) Tuesday March 1st 2016
Virginie Pichard, Nathalie Provost, Alexandra Mendes-Madeira, Lyse Libeau, Philippe Hulin, Kizito-Tshitoko Tshilenge, Marine Biget, Baptiste Ameline, Jack-Yves Deschamps, Michel Weber, Guyl, Marie-Anne Colle, Philippe Moullier, Fabienne Rolling

70) Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing (Molecular Therapy) Tuesday February 16th 2016
Ciaran M Lee, Thomas J Cradick, Eli J Fine, Gang Bao

71) Genome-editing Technologies for Gene and Cell Therapy (Molecular Therapy) Tuesday February 16th 2016
Morgan L Maeder, Charles A Gersbach

72) Salient Features of Endonuclease Platforms for Therapeutic Genome Editing (Molecular Therapy) Tuesday February 16th 2016
Michael T Certo, Richard A Morgan

73) CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus Escape (Molecular Therapy) Tuesday February 16th 2016
Gang Wang, Na Zhao, Ben Berkhout, Atze T Das

74) Induced Apoptosis Investigation in Wild-type and FLT3-ITD Acute Myeloid Leukemia Cells by Nanochannel Electroporation and Single-cell qRT-PCR (Molecular Therapy) Tuesday February 16th 2016
Keliang Gao, Xiaomeng Huang, Chi-Ling Chiang, Xinmei Wang, Lingqian Chang, Pouyan Boukany, Guido Marcucci, Robert Lee, Ly James Lee

75) Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 Promoter (Molecular Therapy) Tuesday February 16th 2016
Haiyan Ji, Zhengtao Jiang, Panpan Lu, Li Ma, Chuan Li, Hanyu Pan, Zheng Fu, Xiying Qu, Pengfei Wang, Junxiao Deng, Xinyi Yang, Jianhua Wang, Huanzhang Zhu

76) The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells (Molecular Therapy) Tuesday February 16th 2016
Ciaran M Lee, Thomas J Cradick, Gang Bao

77) PTH Induces Systemically Administered Mesenchymal Stem Cells to Migrate to and Regenerate Spine Injuries (Molecular Therapy) Tuesday February 9th 2016
Dmitriy Sheyn, Galina Shapiro, Wafa Tawackoli, Douk Soo Jun, Youngdo Koh, Kyu Bok Kang, Susan Su, Xiaoyu Da, Shiran Ben-David, Maxim Bez, Eran Yalon, Ben Antebi, Pablo Avalos, Tomer Stern, Elazar Zelzer, Edward M Schwarz, Zulma Gazit, Gadi Pelled, Hyun M Bae, Dan Gazit

78) Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I (Molecular Therapy) Tuesday February 9th 2016
Cristina Martin-Higueras, Sergio Luis-Lima, Eduardo Salido

79) Actin-resistant DNAse I Expression From Oncolytic Adenovirus Enadenotucirev Enhances Its Intratumoral Spread and Reduces Tumor Growth (Molecular Therapy) Tuesday February 9th 2016
Alison Tedcastle, Sam Illingworth, Alice Brown, Leonard W Seymour, Kerry D Fisher

80) Smooth Muscle Cell (Molecular Therapy) Tuesday February 9th 2016
William H Thiel, Carla L Esposito, David D Dickey, Justin P Dassie, Matthew E Long, Joshua Adam, Jennifer Streeter, Brandon Schickling, Maysam Takapoo, Katie S Flenker, Julia Klesney-Tait, Vittorio de Franciscis, Francis J Miller, Paloma H Giangrande

81) Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T Cells (Molecular Therapy) Tuesday February 9th 2016
Andreas Gogol-D, Ismahen Ammar, Saumyashree Gupta, Mario Bunse, Csaba Miskey, Wei Chen, Wolfgang Uckert, Thomas F Schulz, Zsuzsanna Izsv, Zolt

82) MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy (Molecular Therapy) Tuesday February 9th 2016
Naemeh Pourshafie, Philip R Lee, Ke-lian Chen, George G Harmison, Laura C Bott, Masahisa Katsuno, Gen Sobue, Barrington G Burnett, Kenneth H Fischbeck, Carlo Rinaldi

83) Development and Validation of a Small Single-domain Antibody That Effectively Inhibits Matrix Metalloproteinase 8 (Molecular Therapy) Tuesday February 9th 2016
Delphine Demeestere, Eline Dejonckheere, Sophie Steeland, Paco Hulpiau, Jurgen Haustraete, Nick Devoogdt, Rielana Wichert, Christoph Becker-Pauly, Elien Van Wonterghem, Sylviane Dewaele, Griet Van Imschoot, Jeroen Aerts, Lutgarde Arckens, Yvan Saeys, Claude Libert, Roosmarijn E Vandenbroucke

84) Novel Conserved-region T-cell Mosaic Vaccine With High Global HIV-1 Coverage Is Recognized by Protective Responses in Untreated Infection (Molecular Therapy) Tuesday February 9th 2016
Beatrice Ondondo, Hayato Murakoshi, Genevieve Clutton, Sultan Abdul-Jawad, Edmund G-T Wee, Hiroyuki Gatanaga, Shinichi Oka, Andrew J McMichael, Masafumi Takiguchi, Bette Korber, Tom

85) Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1 (Molecular Therapy) Tuesday February 9th 2016
Chaitali Dutta, Nicole Avitahl-Curtis, Natalie Pursell, Marita Larsson Cohen, Benjamin Holmes, Rohan Diwanji, Wei Zhou, Luciano Apponi, Martin Koser, Bo Ying, Dongyu Chen, Xue Shui, Utsav Saxena, Wendy A Cyr, Anee Shah, Naim Nazef, Weimin Wang, Marc Abrams, Henryk Dudek, Eduardo Salido, Bob D Brown, Chengjung Lai

86) Current Progress in Therapeutic Gene Editing for Monogenic Diseases (Molecular Therapy) Tuesday February 9th 2016
Versha Prakash, Marc Moore, Rafael J Y

87) SSRP1 Contributes to the Malignancy of Hepatocellular Carcinoma and Is Negatively Regulated by miR-497 (Molecular Therapy) Tuesday February 9th 2016
Qianshan Ding, Ke He, Tao Luo, Yunchao Deng, Hanning Wang, Hao Liu, Jinqian Zhang, Kaiyun Chen, Jinfeng Xiao, Xiaopeng Duan, Rui Huang, Zhenglin Xia, Wenjie Zhou, Jinliang He, Honggang Yu, Xingyuan Jiao, Guoan Xiang

88) Modulation of Protein (Molecular Therapy) Tuesday February 2nd 2016
Ioanna Petta, Sam Lievens, Claude Libert, Jan Tavernier, Karolien De Bosscher

89) Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human Cells (Molecular Therapy) Tuesday February 2nd 2016
Jaichandran Sivalingam, Dimitar Kenanov, Hao Han, Ajit Johnson Nirmal, Wai Har Ng, Sze Sing Lee, Jeyakumar Masilamani, Toan Thang Phan, Sebastian Maurer-Stroh, Oi Lian Kon

90) Serial Activation of the Inducible Caspase 9 Safety Switch After Human Stem Cell Transplantation (Molecular Therapy) Tuesday February 2nd 2016
Xiaoou Zhou, Swati Naik, Olga Dakhova, Gianpietro Dotti, Helen E. Heslop, Malcolm K. Brenner

91) Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia (Molecular Therapy) Tuesday January 26th 2016
Tytteli A K Turunen, Jere Kurkipuro, Tommi Heikura, Taina Vuorio, Elisa Hyt, Zsuzsanna Izsv, Seppo Yl

92) Relationship Between the Physicochemical Properties of Lipid Nanoparticles and the Quality of siRNA Delivery to Liver Cells (Molecular Therapy) Tuesday January 26th 2016
Yusuke Sato, Hiroto Hatakeyama, Mamoru Hyodo, Hideyoshi Harashima

93) Re-expression of Selected Epigenetically Silenced Candidate Tumor Suppressor Genes in Cervical Cancer by TET2-directed Demethylation (Molecular Therapy) Tuesday January 26th 2016
Christian Huisman, Monique G P van der Wijst, Matthijs Schokker, Pilar Blancafort, Martijn M Terpstra, Klaas Kok, Ate GJ van der Zee, Ed Schuuring, G Bea A Wisman, Marianne G Rots

94) Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector (Molecular Therapy) Tuesday January 26th 2016
Sourav R Choudhury, Anne F Harris, Damien J Cabral, Allison M Keeler, Ellen Sapp, Jennifer S Ferreira, Heather L Gray-Edwards, Jacob A Johnson, Aime K Johnson, Qin Su, Lorelei Stoica, Marian DiFiglia, Neil Aronin, Douglas R Martin, Guangping Gao, Miguel Sena-Esteves

95) Development of Nonviral Vectors Targeting the Brain as a Therapeutic Approach For Parkinson (Molecular Therapy) Tuesday January 26th 2016
Hayate Javed, Sindhu A Menon, Karima M Al-Mansoori, Abdelmojib Al-Wandi, Nour K Majbour, Mustafa T Ardah, Shiji Varghese, Nishant N Vaikath, M Emdadul Haque, Mimoun Azzouz, Omar MA El-Agnaf

96) SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically (Molecular Therapy) Tuesday January 19th 2016
Tianming Li, Qidi Zheng, Jiahui An, Mengying Wu, Haiyan Li, Xin Gui, Hu Pu, Dongdong Lu

97) Genetic Immunization With In Vivo Dendritic Cell-targeting Liposomal DNA Vaccine Carrier Induces Long-lasting Antitumor Immune Response (Molecular Therapy) Tuesday January 19th 2016
Arup Garu, Gopikrishna Moku, Suresh Kumar Gulla, Arabinda Chaudhuri

98) In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa (Molecular Therapy) Tuesday January 19th 2016
Benjamin Bakondi, Wenjian Lv, Bin Lu, Melissa K Jones, Yuchun Tsai, Kevin J Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J Breunig, Clive N Svendsen, Shaomei Wang

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