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1) Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs (Molecular Therapy) Tuesday December 16th 2014
Cory Smith, Leire Abalde-Atristain, Chaoxia He, Brett R Brodsky, Evan M Braunstein, Pooja Chaudhari, Yoon-Young Jang, Linzhao Cheng, Zhaohui Ye

2) Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose (Molecular Therapy) Tuesday December 9th 2014
Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, Lyndsey Braun, Vicki McGovern, Shibi Likhite, Olivia Michels, Alessandra Govoni, Julie Fitzgerald, Pablo Morales, Kevin D Foust, Jerry R Mendell, Arthur H M Burghes, Brian K Kaspar

3) Case (Molecular Therapy) Tuesday December 9th 2014
Anna Kanerva, Anniina Koski, Ilkka Liikanen, Minna Oksanen, Timo Joensuu, Otto Hemminki, Juni Palmgren, Kari Hemminki, Akseli Hemminki

4) Selective Inhibition of Tumor Growth by Clonal NK Cells Expressing an ErbB2/HER2-Specific Chimeric Antigen Receptor (Molecular Therapy) Tuesday December 9th 2014
Kurt Sch, Christiane Sahm, Congcong Zhang, Sonja Naundorf, Christian Brendel, Marcus Odendahl, Paulina Nowakowska, Halvard B, Ulrike K, Stephan Kloess, Sylvia K, Heidi Holtgreve-Grez, Anna Jauch, Manfred Schmidt, Ralf Schubert, Klaus K, Erhard Seifried, Hans G Klingemann, Michael A Rieger, Torsten Tonn, Manuel Grez, Winfried S Wels

5) Let-7 microRNAs Regenerate Peripheral Nerve Regeneration by Targeting Nerve Growth Factor (Molecular Therapy) Tuesday December 9th 2014
Shiying Li, Xinghui Wang, Yun Gu, Chu Chen, Yaxian Wang, Jie Liu, Wen Hu, Bin Yu, Yongjun Wang, Fei Ding, Yan Liu, Xiaosong Gu

6) Development of Novel Activin-Targeted Therapeutics (Molecular Therapy) Tuesday December 9th 2014
Justin L Chen, Kelly L Walton, Sara L Al-Musawi, Emily K Kelly, Hongwei Qian, Mylinh La, Louis Lu, George Lovrecz, Mark Ziemann, Ross Lazarus, Assam El-Osta, Paul Gregorevic, Craig A Harrison

7) Cyclooxygenase-2 Silencing for the Treatment of Colitis: A Combined In Vivo Strategy Based on RNA Interference and Engineered Escherichia Coli (Molecular Therapy) Tuesday December 9th 2014
Enzo Spisni, Maria C Valerii, Luigia De Fazio, Elena Cavazza, Francesca Borsetti, Annamaria Sgromo, Marco Candela, Manuela Centanni, Fernando Rizello, Antonio Strillacci

8) Implantation of rAAV5-IGF-I Transduced Autologous Chondrocytes Improves Cartilage Repair in Full-thickness Defects in the Equine Model (Molecular Therapy) Tuesday December 2nd 2014
Kyla F Ortved, Laila Begum, Hussni O Mohammed, Alan J Nixon

9) Enhanced Efficacy of the CDNF/MANF Family by Combined Intranigral Overexpression in the 6-OHDA Rat Model of Parkinson (Molecular Therapy) Tuesday December 2nd 2014
Óscar Cordero-Llana, Benjamin C Houghton, Federica Rinaldi, Hannah Taylor, Rafael J Y, James B Uney, Liang-Fong Wong, Maeve A Caldwell

10) Mesenchymal Stem Cells Correct Inappropriate Epithelial (Molecular Therapy) Tuesday December 2nd 2014
Manabu Ono, Shinya Ohkouchi, Masahiko Kanehira, Naoki Tode, Makoto Kobayashi, Masahito Ebina, Toshihiro Nukiwa, Toshiya Irokawa, Hiromasa Ogawa, Takaaki Akaike, Yoshinori Okada, Hajime Kurosawa, Toshiaki Kikuchi, Masakazu Ichinose

11) Targeted 25-hydroxyvitamin D3 1 (Molecular Therapy) Tuesday November 25th 2014
Bo Li, David J Baylink, Michael H Walter, Kin-Hing William Lau, Xianmei Meng, Jun Wang, Andriy Cherkas, Xiaolei Tang, Xuezhong Qin

12) Multiplexing Seven miRNA-Based shRNAs to Suppress HIV Replication (Molecular Therapy) Tuesday November 25th 2014
Jang-Gi Choi, Preeti Bharaj, Sojan Abraham, Hongming Ma, Guohua Yi, Chunting Ye, Ying Dang, N Manjunath, Haoquan Wu, Premlata Shankar

13) Broadly-specific Cytotoxic T Cells Targeting Multiple HIV Antigens Are Expanded From HIV+ Patients: Implications for Immunotherapy (Molecular Therapy) Tuesday November 25th 2014
Sharon Lam, Julia Sung, Conrad Cruz, Paul Castillo-Caro, Minhtran Ngo, Carolina Garrido, Joann Kuruc, Nancie Archin, Cliona Rooney, David Margolis, Catherine Bollard

14) Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas (Molecular Therapy) Tuesday November 25th 2014
Sung Hugh Choi, Kaoru Tamura, Rajiv Kumar Khajuria, Deepak Bhere, Irina Nesterenko, Jack Lawler, Khalid Shah

15) Recurrence of Melanoma Following T Cell Treatment: Continued Antigen Expression in a Tumor That Evades T Cell Recruitment (Molecular Therapy) Tuesday November 25th 2014
Trudy Straetemans, Cor Berrevoets, Miriam Coccoris, Elike Treffers-Westerlaken, Rebecca Wijers, David K Cole, Valerie Dardalhon, Andrew K Sewell, Naomi Taylor, Jaap Verweij, Reno Debets

16) A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy (Molecular Therapy) Tuesday November 18th 2014
Jerry R Mendell, Zarife Sahenk, Vinod Malik, Ana M Gomez, Kevin M Flanigan, Linda P Lowes, Lindsay N Alfano, Katherine Berry, Eric Meadows, Sarah Lewis, Lyndsey Braun, Kim Shontz, Maria Rouhana, Kelly Reed Clark, Xiomara Q Rosales, Samiah Al-Zaidy, Alessandra Govoni, Louise R Rodino-Klapac, Mark J Hogan, Brian K Kaspar

17) Encapsulated Stem Cells Loaded With Hyaluronidase-expressing Oncolytic Virus for Brain Tumor Therapy (Molecular Therapy) Tuesday November 18th 2014
Jordi Martinez-Quintanilla, Derek He, Hiroaki Wakimoto, Ramon Alemany, Khalid Shah

18) Exosome-mediated Delivery of the Intrinsic C-terminus Domain of PTEN Protects It From Proteasomal Degradation and Ablates Tumorigenesis (Molecular Therapy) Tuesday November 11th 2014
Syed Feroj Ahmed, Nilanjana Das, Moumita Sarkar, Uttara Chatterjee, Sandip Chatterjee, Mrinal Kanti Ghosh

19) Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice (Molecular Therapy) Tuesday November 4th 2014


20) An AAVS1-Targeted Minigene Platform for Correction of iPSCs From All Five Types of Chronic Granulomatous Disease (Molecular Therapy) Tuesday November 4th 2014
Randall K Merling, Colin L Sweeney, Jessica Chu, Aaron Bodansky, Uimook Choi, Debra Long Priel, Douglas B Kuhns, Hongmei Wang, Sam Vasilevsky, Suk See De Ravin, Thomas Winkler, Cynthia E Dunbar, Jizhong Zou, Kol A Zarember, John I Gallin, Steven M Holland, Harry L Malech

21) SCL/TAL1-mediated Transcriptional Network Enhances Megakaryocytic Specification of Human Embryonic Stem Cells (Molecular Therapy) Tuesday November 4th 2014
Miguel G Toscano, Oscar Navarro-Montero, Veronica Ayllon, Veronica Ramos-Mejia, Xiomara Guerrero-Carreno, Clara Bueno, Tamara Romero, Mar Lamolda, Marien Cobo, Francisco Martin, Pablo Menendez, Pedro J Real

22) miR-382 Inhibits Osteosarcoma Metastasis and Relapse by Targeting Y Box-Binding Protein 1 (Molecular Therapy) Tuesday November 4th 2014
Meng Xu, Hua Jin, Cheng-Xiong Xu, Bo Sun, Zhi-Gang Song, Wen-Zhi Bi, Yan Wang

23) AAV2 Delivery of Flt23k Intraceptors Inhibits Murine Choroidal Neovascularization (Molecular Therapy) Tuesday November 4th 2014
Xiaohui Zhang, Subrata K Das, Samuel F Passi, Hironori Uehara, Austin Bohner, Marcus Chen, Michelle Tiem, Bonnie Archer, Balamurali K Ambati

24) A Functional Variant of Elafin With Improved Anti-inflammatory Activity for Pulmonary Inflammation (Molecular Therapy) Tuesday November 4th 2014


25) Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I (Molecular Therapy) Tuesday November 4th 2014


26) A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines (Molecular Therapy) Tuesday November 4th 2014


27) Graft Versus Leukemia Response Without Graft-versus-host Disease Elicited By Adoptively Transferred Multivirus-specific T-cells (Molecular Therapy) Tuesday October 21st 2014
Jan J Melenhorst, Paul Castillo, Patrick J Hanley, Michael D Keller, Robert A Krance, Judith Margolin, Ann M Leen, Helen E Heslop, A John Barrett, Cliona M Rooney, Catherine M Bollard

28) Cyclosporin A and Rapamycin Relieve Distinct Lentiviral Restriction Blocks in Hematopoietic Stem and Progenitor Cells (Molecular Therapy) Tuesday October 21st 2014
Carolina Petrillo, Daniela Cesana, Francesco Piras, Sara Bartolaccini, Luigi Naldini, Eugenio Montini, Anna Kajaste-Rudnitski

29) CD133-targeted Gene Transfer Into Long-term Repopulating Hematopoietic Stem Cells (Molecular Therapy) Tuesday October 14th 2014
Christian Brendel, Benjamin Goebel, Abriss Daniela, Martijn Brugman, Sabrina Kneissl, Joachim Schw, Kerstin B Kaufmann, Uta M, Hana Kunkel, Linping Chen-Wichmann, Tobias Abel, Hubert Serve, Leonid Bystrykh, Christian J Buchholz, Manuel Grez

30) Fractalkine Over Expression Suppresses (Molecular Therapy) Tuesday October 14th 2014
Kevin R Nash, Peter Moran, Dylan J Finneran, Charles Hudson, Jesse Robinson, Dave Morgan, Paula C Bickford

31) Use of miRNA Response Sequences to Block Off-target Replication and Increase the Safety of an Unattenuated, Glioblastoma-targeted Oncolytic HSV (Molecular Therapy) Tuesday October 14th 2014
Lucia Mazzacurati, Marco Marzulli, Bonnie Reinhart, Yoshitaka Miyagawa, Hiroaki Uchida, William F Goins, Aofei Li, Balveen Kaur, Michael Caligiuri, Timothy Cripe, Nino Chiocca, Nduka Amankulor, Justus B Cohen, Joseph C Glorioso, Paola Grandi

32) Oral Delivery of ACE2/Ang-(1 (Molecular Therapy) Tuesday October 14th 2014
Pollob K Shil, Kwang-Chul Kwon, Ping Zhu, Amrisha Verma, Henry Daniell, Qiuhong Li

33) The Expression Pattern of Systemically Injected AAV9 in the Developing Mouse Retina Is Determined by Age (Molecular Therapy) Tuesday October 14th 2014
Leah C Byrne, Yvonne J Lin, Trevor Lee, David V Schaffer, John G Flannery

34) EZH2 Modulates Angiogenesis In Vitro and in a Mouse Model of Limb Ischemia (Molecular Therapy) Wednesday October 8th 2014
Tijana Miti, Andrea Caporali, Ilaria Floris, Marco Meloni, Micol Marchetti, Raul Urrutia, Gianni D Angelini, Costanza Emanueli

35) AAV8-Mediated In Vivo Overexpression of miR-155 Enhances the Protective Capacity of Genetically Attenuated Malarial Parasites (Molecular Therapy) Wednesday October 8th 2014
Franziska Hentzschel, Christiane Hammerschmidt-Kamper, Kathleen B, Kirsten Heiss, Bettina Knapp, Julia M Sattler, Lars Kaderali, Mirco Castoldi, Julia G Bindman, Yann Malato, Holger Willenbring, Ann-Kristin Mueller, Dirk Grimm

36) Antitumor Activity of miR-1280 in Melanoma by Regulation of Src (Molecular Therapy) Wednesday October 8th 2014
Vera Sun, Wen B Zhou, Mehdi Nosrati, Shahana Majid, Suresh Thummala, David de Semir, Vladimir Bezrookove, Sebastien de Feraudy, Liane Chun, Dirk Schadendorf, Robert Debs, Mohammed Kashani-Sabet, Altaf A Dar

37) Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy (Molecular Therapy) Wednesday October 8th 2014
Melvin Y Rincon, Shilpita Sarcar, Dina Danso-Abeam, Marleen Keyaerts, Janka Matrai, Ermira Samara-Kuko, Abel Acosta-Sanchez, Takis Athanasopoulos, George Dickson, Tony Lahoutte, Pieter De Bleser, Thierry VandenDriessche, Marinee K Chuah

38) In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides (Molecular Therapy) Tuesday September 30th 2014
Amber L Southwell, Niels H Skotte, Holly B Kordasiewicz, Michael E, Andrew T Watt, Jeffrey B Carroll, Crystal N Doty, Erika B Villanueva, Eugenia Petoukhov, Kuljeet Vaid, Yuanyun Xie, Susan M Freier, Eric E Swayze, Punit P Seth, Clarence Frank Bennett, Michael R Hayden

39) Combining Viral Vectored and Protein-in-adjuvant Vaccines Against the Blood-stage Malaria Antigen AMA1: Report on a Phase 1a Clinical Trial (Molecular Therapy) Tuesday September 30th 2014
Susanne H Hodgson, Prateek Choudhary, Sean C Elias, Kathryn H Milne, Thomas W Rampling, Sumi Biswas, Ian D Poulton, Kazutoyo Miura, Alexander D Douglas, Daniel GW Alanine, Joseph J Illingworth, Simone C de Cassan, Daming Zhu, Alfredo Nicosia, Carole A Long, Sarah Moyle, Eleanor Berrie, Alison M Lawrie, Yimin Wu, Ruth D Ellis, Adrian V S Hill, Simon J Draper

40) CTLA-4 and PD-L1 Checkpoint Blockade Enhances Oncolytic Measles Virus Therapy (Molecular Therapy) Tuesday September 30th 2014
Christine E Engeland, Christian Grossardt, R, Sascha Bossow, Diana Lutz, Johanna K Kaufmann, Ivan Shevchenko, Viktor Umansky, Dirk M Nettelbeck, Wilko Weichert, Dirk J, Christof von Kalle, Guy Ungerechts

41) Neovascularization Capacity of Mesenchymal Stromal Cells From Critical Limb Ischemia Patients Is Equivalent to Healthy Controls (Molecular Therapy) Tuesday September 30th 2014
Hendrik Gremmels, Martin Teraa, Paul HA Quax, Krista den Ouden, Joost O Fledderus, Marianne C Verhaar

42) Lentiviral Vector-based Insertional Mutagenesis Identifies Genes Involved in the Resistance to Targeted Anticancer Therapies (Molecular Therapy) Tuesday September 30th 2014
Marco Ranzani, Stefano Annunziato, Andrea Calabria, Stefano Brasca, Fabrizio Benedicenti, Pierangela Gallina, Luigi Naldini, Eugenio Montini

43) Mesenchymal Stem/Stromal Cells Protect the Ocular Surface by Suppressing Inflammation in an Experimental Dry Eye (Molecular Therapy) Tuesday September 23rd 2014
Min Joung Lee, Ah Young Ko, Jung Hwa Ko, Hyun Ju Lee, Mee Kum Kim, Won Ryang Wee, Sang In Khwarg, Joo Youn Oh

44) Treatment of CD33-directed Chimeric Antigen Receptor-modified T Cells in One Patient With Relapsed and Refractory Acute Myeloid Leukemia (Molecular Therapy) Tuesday September 23rd 2014
Quan-shun Wang, Yao Wang, Hai-yan Lv, Qing-wang Han, Hui Fan, Bo Guo, Li-li Wang, Wei-dong Han

45) Systemic Administration of Platelets Incorporating Inactivated Sendai Virus Eradicates Melanoma in Mice (Molecular Therapy) Tuesday September 16th 2014
Tomoyuki Nishikawa, Li Yu Tung, Yasufumi Kaneda

46) Interventional Magnetic Resonance Imaging-guided Cell Transplantation Into the Brain With Radially Branched Deployment (Molecular Therapy) Tuesday September 16th 2014
Matthew T Silvestrini, Dali Yin, Alastair J Martin, Valerie G Coppes, Preeti Mann, Paul S Larson, Philip A Starr, Xianmin Zeng, Nalin Gupta, S S Panter, Tejal A Desai, Daniel A Lim

47) Engager T Cells: A New Class of Antigen-specific T Cells That Redirect Bystander T Cells (Molecular Therapy) Tuesday September 16th 2014
Kota Iwahori, Sunitha Kakarla, Mireya P Velasquez, Feng Yu, Zongzhen Yi, Claudia Gerken, Xiao-Tong Song, Stephen Gottschalk

48) Efficient Drug Delivery and Induction of Apoptosis in Colorectal Tumors Using a Death Receptor 5-Targeted Nanomedicine (Molecular Therapy) Tuesday September 9th 2014
Daniela Schmid, Francois Fay, Donna M Small, Jakub Jaworski, Joel S Riley, Diana Tegazzini, Cathy Fenning, David S Jones, Patrick G Johnston, Daniel B Longley, Christopher J Scott

49) Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients (Molecular Therapy) Tuesday September 9th 2014
Caroline Le Guiner, Marie Montus, Laurent Servais, Yan Cherel, Virginie Francois, Jean-Laurent Thibaud, Claire Wary, B, Thibaut Larcher, Lydie Guigand, Maeva Dutilleul, Claire Domenger, Marine Allais, Maud Beuvin, Am, Johanne Le Duff, Marie Devaux, Nicolas Jaulin, Micka, Virginie Latournerie, Philippe Veron, Sylvie Boutin, Christian Leborgne, Diana Desgue, Jack-Yves Deschamps, Sophie Moullec, Yves Fromes, Adeline Vulin, Richard H Smith, Nicolas Laroudie, Fr, Christel Rivi, St, Thanh-Hoa Le, Nicolas Delaunay, Mehdi Gasmi, Robert M Kotin, Gis, Oumeya Adjali, Carole Masurier, Jean-Yves Hogrel, Pierre Carlier, Philippe Moullier, Thomas Voit

50) Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase (Molecular Therapy) Tuesday September 9th 2014
Salim S El-Amouri, Mei Dai, Jing-Fen Han, Roscoe O Brady, Dao Pan

51) Targeting Channelrhodopsin-2 to ON-bipolar Cells With Vitreally Administered AAV Restores ON and OFF Visual Responses in Blind Mice (Molecular Therapy) Tuesday September 9th 2014
Emilie Mac, Romain Caplette, Olivier Marre, Abhishek Sengupta, Antoine Chaffiol, Peggy Barbe, M, Ernst Bamberg, Jose-Alain Sahel, Serge Picaud, Jens Duebel, Deniz Dalkara

52) Cardiac I-1c Overexpression With Reengineered AAV Improves Cardiac Function in Swine Ischemic Heart Failure (Molecular Therapy) Tuesday September 2nd 2014
Kiyotake Ishikawa, Kenneth M Fish, Lisa Tilemann, Kleopatra Rapti, Jaume Aguero, Carlos G Santos-Gallego, Ahyoung Lee, Ioannis Karakikes, Chaoqin Xie, Fadi G Akar, Yuichi J Shimada, Judith K Gwathmey, Aravind Asokan, Scott McPhee, Jade Samulski, Richard Jude Samulski, Daniel C Sigg, Thomas Weber, Evangelia G Kranias, Roger J Hajjar

53) The Profile of Tumor Antigens Which Can be Targeted by Immunotherapy Depends Upon the Tumor (Molecular Therapy) Tuesday September 2nd 2014
Vanesa Alonso-Camino, Karishma Rajani, Timothy Kottke, Diana Rommelfanger-Konkol, Shane Zaidi, Jill Thompson, Jose Pulido, Elizabeth Ilett, Oliver Donnelly, Peter Selby, Hardev Pandha, Alan Melcher, Kevin Harrington, Rosa Maria Diaz, Richard Vile

54) A Chaperone Enhances Blood (Molecular Therapy) Tuesday September 2nd 2014
Giancarlo Parenti, Simona Fecarotta, Giancarlo la Marca, Barbara Rossi, Serena Ascione, Maria Alice Donati, Lucia Ovidia Morandi, Sabrina Ravaglia, Anna Pichiecchio, Daniela Ombrone, Michele Sacchini, Maria Barbara Pasanisi, Paola De Filippi, Cesare Danesino, Roberto Della Casa, Alfonso Romano, Carmine Mollica, Margherita Rosa, Teresa Agovino, Edoardo Nusco, Caterina Porto, Generoso Andria

55) Co-Expression of Tumor Antigen and Interleukin-2 From an Adenoviral Vector Augments the Efficiency of Therapeutic Tumor Vaccination (Molecular Therapy) Tuesday August 26th 2014
Benjamin Anderschou Holbech Jensen, Maria Abildgaard Steffensen, Karen N, Jan Pravsgaard Christensen, Allan Randrup Thomsen, Peter Johannes Holst

56) Aminoglycosides Restore Full-length Type VII Collagen by Overcoming Premature Termination Codons: Therapeutic Implications for Dystrophic Epidermolysis Bullosa (Molecular Therapy) Tuesday August 26th 2014
Jon Cogan, Jacqueline Weinstein, Xinyi Wang, Yingping Hou, Sabrina Martin, Andrew P South, David T Woodley, Mei Chen

57) RNAi-mediated TCR Knockdown Prevents Autoimmunity in Mice Caused by Mixed TCR Dimers Following TCR Gene Transfer (Molecular Therapy) Tuesday August 26th 2014
Mario Bunse, Gavin M Bendle, Carsten Linnemann, Laura Bies, Stephan Schulz, Ton N Schumacher, Wolfgang Uckert

58) Mesenchymal Stem Cells Improve Motor Functions and Decrease Neurodegeneration in Ataxic Mice (Molecular Therapy) Tuesday August 26th 2014
Jonathan Jones, Alicia Estirado, Carolina Redondo, Jesus Pacheco-Torres, Maria-Salom, Jos, Salvador Martinez

59) Baculovirus-Mediated miRNA Regulation to Suppress Hepatocellular Carcinoma Tumorigenicity and Metastasis (Molecular Therapy) Tuesday August 19th 2014
Chiu-Ling Chen, Jaw-Ching Wu, Guan-Yu Chen, Pei-Hsiang Yuan, Yen-Wen Tseng, Kuei-Chang Li, Shiaw-Min Hwang, Yu-Chen Hu

60) Chaperone Nanobodies Protect Gelsolin Against MT1-MMP Degradation and Alleviate Amyloid Burden in the Gelsolin Amyloidosis Mouse Model (Molecular Therapy) Tuesday August 19th 2014
Wouter Van Overbeke, Adriaan Verhelle, Inge Everaert, Olivier Zwaenepoel, Jo, Claude Cuvelier, Wim Derave, Jan Gettemans

61) Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants (Molecular Therapy) Tuesday August 19th 2014
Damien Marsic, Lakshmanan Govindasamy, Seth Currlin, David M Markusic, Yu-Shan Tseng, Roland W Herzog, Mavis Agbandje-McKenna, Sergei Zolotukhin

62) Muscle and Heart Function Restoration in a Limb Girdle Muscular Dystrophy 2I (LGMD2I) Mouse Model by Systemic FKRP Gene Delivery (Molecular Therapy) Tuesday August 19th 2014
Chunping Qiao, Chi-Hsien Wang, Chunxia Zhao, Peijuan Lu, Hiroyuki Awano, Bin Xiao, Jianbin Li, Zhenhua Yuan, Yi Dai, Carrie Bette Martin, Juan Li, Qilong Lu, Xiao Xiao

63) Effects of Vector Backbone and Pseudotype on Lentiviral Vector-mediated Gene Transfer: Studies in Infant ADA-Deficient Mice and Rhesus Monkeys (Molecular Therapy) Tuesday August 19th 2014
Denise Carbonaro Sarracino, Alice F Tarantal, C Chang I. Lee, Michele Martinez, Xiangyang Jin, Xiaoyan Wang, Cinnamon L Hardee, Sabine Geiger, Christoph A Kahl, Donald B Kohn

64) ESCRT-mediated Uptake and Degradation of Brain-targeted (Molecular Therapy) Tuesday August 12th 2014
Brian Spencer, Sharareh Emadi, Paula Desplats, Simona Eleuteri, Sarah Michael, Kori Kosberg, Jay Shen, Edward Rockenstein, Christina Patrick, Anthony Adame, Tania Gonzalez, Michael Sierks, Eliezer Masliah

65) Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy (Molecular Therapy) Tuesday August 12th 2014
Mario Di Matteo, Emira Samara-Kuko, Natalie J Ward, Simon N Waddingon, John H McVey, Marinee KL Chuah, Thierry VandenDriessche

66) Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates (Molecular Therapy) Tuesday August 5th 2014
Marinee K Chuah, Inge Petrus, Pieter De Bleser, Caroline Le Guiner, Gwladys Gernoux, Oumeya Adjali, Nisha Nair, Jessica Willems, Hanneke Evens, Melvin Y Rincon, Janka Matrai, Mario Di Matteo, Ermira Samara-Kuko, Bing Yan, Abel Acosta-Sanchez, Amine Meliani, Ghislaine Cherel, V, Olivier Christophe, Philippe Moullier, Federico Mingozzi, Thierry VandenDriessche

67) Anti-microRNA-378a Enhances Wound Healing Process by Upregulating Integrin Beta-3 and Vimentin (Molecular Therapy) Tuesday August 5th 2014
Haoran Li, Leslie Chang, William W Du, Shaan Gupta, Azam Khorshidi, Michael Sefton, Burton B Yang

68) Cytokine Conditioning Enhances Systemic Delivery and Therapy of an Oncolytic Virus (Molecular Therapy) Tuesday August 5th 2014
Elizabeth Ilett, Timothy Kottke, Oliver Donnelly, Jill Thompson, Candice Willmon, Rosa Diaz, Shane Zaidi, Matt Coffey, Peter Selby, Kevin Harrington, Hardev Pandha, Alan Melcher, Richard Vile

69) A Transgenic Probiotic Secreting a Parasite Immunomodulator for Site-Directed Treatment of Gut Inflammation (Molecular Therapy) Tuesday August 5th 2014
Rose A Whelan, Sebastian Rausch, Friederike Ebner, Dorothee G, Jan F Richter, Nina A Hering, J, Anja A K, Ahmed Keles, Pawel Janczyk, Karsten N, Lothar H Wieler, Susanne Hartmann

70) Translating the Immunogenicity of Prime-boost Immunization With ChAd63 and MVA ME-TRAP From Malaria Naive to Malaria-endemic Populations (Molecular Therapy) Tuesday July 29th 2014
Domtila Kimani, Ya Jankey Jagne, Momodou Cox, Eva Kimani, Carly M Bliss, Evelyn Gitau, Caroline Ogwang, Muhammed O Afolabi, Georgina Bowyer, Katharine A Collins, Nick Edwards, Susanne H Hodgson, Christopher J A Duncan, Alexandra J Spencer, Miguel G Knight, Abdoulie Drammeh, Nicholas A Anagnostou, Eleanor Berrie, Sarah Moyle, Sarah C Gilbert, Peninah Soipei, Joseph Okebe, Stefano Colloca, Riccardo Cortese, Nicola K Viebig, Rachel Roberts, Alison M Lawrie, Alfredo Nicosia, Egeruan B Imoukhuede, Philip Bejon, Roma Chilengi, Kalifa Bojang, Katie L Flanagan, Adrian V S Hill, Britta C Urban, Katie J Ewer

71) Characterisation of a Novel Fc Conjugate of Macrophage Colony-stimulating Factor (Molecular Therapy) Tuesday July 29th 2014
Deborah J Gow, Kristin A Sauter, Clare Pridans, Lindsey Moffat, Anuj Sehgal, Ben M Stutchfield, Sobia Raza, Philippa M Beard, Yi Ting Tsai, Graeme Bainbridge, Pamela L Boner, Greg Fici, David Garcia-Tapia, Roger A Martin, Theodore Oliphant, John A Shelly, Raksha Tiwari, Thomas L Wilson, Lee B Smith, Neil A Mabbott, David A Hume

72) Targeted Inhibition of Prostate Cancer Metastases with an RNA Aptamer to Prostate-specific Membrane Antigen (Molecular Therapy) Tuesday July 29th 2014
Justin P Dassie, Luiza I Hernandez, Gregory S Thomas, Matthew E Long, William M Rockey, Craig A Howell, Yani Chen, Frank J Hernandez, Xiu Ying Liu, Mary E Wilson, Lee-Ann Allen, Daniel A Vaena, David K Meyerholz, Paloma H Giangrande

73) MicroRNA-103-1 Selectively Downregulates Brain NCX1 and Its Inhibition by Anti-miRNA Ameliorates Stroke Damage and Neurological Deficits (Molecular Therapy) Tuesday July 22nd 2014
Antonio Vinciguerra, Luigi Formisano, Pierpaolo Cerullo, Natascia Guida, Ornella Cuomo, Alba Esposito, Gianfranco Di Renzo, Lucio Annunziato, Giuseppe Pignataro

74) Induction of Androgen Formation in the Male by a TAT-VDAC1 Fusion Peptide Blocking 14-3-3 (Molecular Therapy) Tuesday July 22nd 2014
Yasaman Aghazadeh, Daniel B Martinez-Arguelles, Jinjiang Fan, Martine Culty, Vassilios Papadopoulos

75) Gene Transfer Properties and Structural Modeling of Human Stem Cell-derived AAV (Molecular Therapy) Wednesday July 16th 2014
Laura J Smith, Taihra Ul-Hasan, Sarah K Carvaines, Kim Van Vliet, Ethel Yang, Kamehameha K Wong, Mavis Agbandje-McKenna, Saswati Chatterjee

76) Epicardial Placement of Mesenchymal Stromal Cell-sheets for the Treatment of Ischemic Cardiomyopathy; In Vivo Proof-of-concept Study (Molecular Therapy) Wednesday July 16th 2014
Nobuko Tano, Takuya Narita, Masahiro Kaneko, Chiho Ikebe, Steven R Coppen, Niall G Campbell, Manabu Shiraishi, Yasunori Shintani, Ken Suzuki

77) Serum-stabilized Naked Caspase-3 siRNA Protects Autotransplant Kidneys in a Porcine Model (Molecular Therapy) Wednesday July 16th 2014
Cheng Yang, Tian Zhao, Zitong Zhao, Yichen Jia, Long Li, Yufang Zhang, Mangen Song, Ruiming Rong, Ming Xu, Michael L Nicholson, Tongyu Zhu, Bin Yang

78) Myocyte-mediated Arginase Expression Controls Hyperargininemia but not Hyperammonemia in Arginase-deficient Mice (Molecular Therapy) Wednesday July 16th 2014
Chuhong Hu, Jennifer Kasten, Hana Park, Ragini Bhargava, Denise S Tai, Wayne W Grody, Quynh G Nguyen, Stephen D Hauschka, Stephen D Cederbaum, Gerald S Lipshutz

79) Gene Therapy in Patient-specific Stem Cell Lines and a Preclinical Model of Retinitis Pigmentosa With Membrane Frizzled-related Protein Defects (Molecular Therapy) Tuesday July 8th 2014
Yao Li, Wen-Hsuan Wu, Chun-Wei Hsu, Huy V Nguyen, Yi-Ting Tsai, Lawrence Chan, Takayuki Nagasaki, Irene H Maumenee, Lawrence A Yannuzzi, Quan V Hoang, Haiqing Hua, Dieter Egli, Stephen H Tsang

80) Lentiviral Arrays for Live-cell Dynamic Monitoring of Gene and Pathway Activity During Stem Cell Differentiation (Molecular Therapy) Tuesday July 8th 2014
Roshan M Padmashali, Panagiotis Mistriotis, Mao-shih Liang, Stelios T Andreadis

81) KITENIN-targeting MicroRNA-124 Suppresses Colorectal Cancer Cell Motility and Tumorigenesis (Molecular Therapy) Tuesday July 8th 2014
So-Yeon Park, Hangun Kim, Somy Yoon, Jeong A Bae, Seok-Yong Choi, Young Do Jung, Kyung Keun Kim

82) Nonallele Specific Silencing of Ataxin-7 Improves Disease Phenotypes in a Mouse Model of SCA7 (Molecular Therapy) Tuesday July 8th 2014
Pavitra S Ramachandran, Ryan L Boudreau, Kellie A Schaefer, Albert R La Spada, Beverly L Davidson

83) Role of Cysteine-rich 61 Protein (CCN1) in Macrophage-mediated Oncolytic Herpes Simplex Virus Clearance (Molecular Therapy) Tuesday July 1st 2014
Amy Haseley Thorne, Walter H Meisen, Luke Russell, Ji Young Yoo, Chelsea M Bolyard, Justin D Lathia, Jeremy Rich, Vinay K Puduvalli, Hsiaoyin Mao, Jianhua Yu, Michael A Caligiuri, Susheela Tridandapani, Balveen Kaur

84) Angiopoietin-like 4 Stimulates STAT3-mediated iNOS Expression and Enhances Angiogenesis to Accelerate Wound Healing in Diabetic Mice (Molecular Therapy) Tuesday July 1st 2014
Han Chung Chong, Jeremy Soon Kiat Chan, Chi Qin Goh, Natalia V Gounko, Baiwen Luo, Xiaoling Wang, Selin Foo, Marcus Thien Chong Wong, Cleo Choong, Sander Kersten, Nguan Soon Tan

85) Cytotoxic and Regulatory Properties of Circulating V (Molecular Therapy) Tuesday July 1st 2014
Gabrielle M Siegers, Lawrence S Lamb

86) Blockade of ActRIIB Signaling Triggers Muscle Fatigability and Metabolic Myopathy (Molecular Therapy) Tuesday July 1st 2014
Karima Relizani, Etienne Mouisel, Benoit Giannesini, Christophe Hourd, Ketan Patel, Susanne Morales Gonzalez, Kristina J, Alban Vignaud, France Pi, Dominique Fortin, Luis Garcia, St, Olli Ritvos, David Bendahan, Arnaud Ferry, Ren, Markus Schuelke, Helge Amthor

87) Epigenetic Manipulation Restores Functions of Defective CD8+ T Cells From Chronic Viral Infection (Molecular Therapy) Tuesday July 1st 2014
Fuqin Zhang, Xiaohui Zhou, Joanna R DiSpirito, Chuan Wang, Ying Wang, Hao Shen

88) MiR-148a- and miR-216a-regulated Oncolytic Adenoviruses Targeting Pancreatic Tumors Attenuate Tissue Damage Without Perturbation of miRNA Activity (Molecular Therapy) Tuesday July 1st 2014
Xavier Bofill-De Ros, Meritxell Gironella, Cristina Fillat

89) Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10 (Molecular Therapy) Tuesday June 24th 2014
Bin Yang, Shaoyong Li, Hongyan Wang, Yansu Guo, Dominic J Gessler, Chunyan Cao, Qin Su, Joshua Kramer, Li Zhong, Seemin Seher Ahmed, Hongwei Zhang, Ran He, Ronald C Desrosiers, Robert Brown, Zuoshang Xu, Guangping Gao

90) Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis (Molecular Therapy) Tuesday June 24th 2014
Maria Chiriaco, Giada Farinelli, Valentina Capo, Erika Zonari, Samantha Scaramuzza, Gigliola Di Matteo, Lucia Sergi Sergi, Maddalena Migliavacca, Raisa Jofra Hernandez, Ferdinando Bombelli, Ezio Giorda, Anna Kajaste-Rudnitski, Didier Trono, Manuel Grez, Paolo Rossi, Andrea Finocchi, Luigi Naldini, Bernhard Gentner, Alessandro Aiuti

91) Overcoming the Cystic Fibrosis Sputum Barrier to Leading Adeno-associated Virus Gene Therapy Vectors (Molecular Therapy) Tuesday June 24th 2014
Benjamin S Schuster, Anthony J Kim, Joshua C Kays, Mia M Kanzawa, William B Guggino, Michael P Boyle, Steven M Rowe, Nicholas Muzyczka, Jung Soo Suk, Justin Hanes

92) Therapeutic Delivery of miR-200c Enhances Radiosensitivity in Lung Cancer (Molecular Therapy) Tuesday June 17th 2014
Maria Angelica Cortez, David Valdecanas, Xiaochun Zhang, Yanai Zhan, Vikas Bhardwaj, George A Calin, Ritsuko Komaki, Dipak K Giri, Caio C Quini, Tatiana Wolfe, Heidi J Peltier, Andreas G Bader, John V Heymach, Raymond E Meyn, James W Welsh

93) Characterizing Human Stem Cell (Molecular Therapy) Tuesday June 17th 2014
Gareth T Young, Alex Gutteridge, Heather DE Fox, Anna L Wilbrey, Lishuang Cao, Lily T Cho, Adam R Brown, Caroline L Benn, Laura R Kammonen, Julia H Friedman, Magda Bictash, Paul Whiting, James G Bilsland, Edward B Stevens

94) ID Proteins Regulate Diverse Aspects of Cancer Progression and Provide Novel Therapeutic Opportunities (Molecular Therapy) Tuesday June 10th 2014
Radhika Nair, Wee Siang Teo, Vivek Mittal, Alexander Swarbrick

95) Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice (Molecular Therapy) Tuesday June 10th 2014
Li-Kai Tsai, Chien-Lin Chen, Chen-Hung Ting, Sue Lin-Chao, Wuh-Liang Hwu, James C Dodge, Marco A Passini, Seng H Cheng

96) Inflammation Converts Human Mesoangioblasts Into Targets of Alloreactive Immune Responses: Implications for Allogeneic Cell Therapy of DMD (Molecular Therapy) Wednesday June 4th 2014
Maddalena Noviello, Francesco Saverio Tedesco, Attilio Bondanza, Rossana Tonlorenzi, Maria Rosaria Carbone, Mattia Francesco Maria Gerli, Sarah Marktel, Sara Napolitano, Maria Pia Cicalese, Fabio Ciceri, Giuseppe Peretti, Giulio Cossu, Chiara Bonini

97) Minimally Invasive Approach to the Repair of Injured Skeletal Muscle With a Shape-memory Scaffold (Molecular Therapy) Wednesday June 4th 2014
Lin Wang, Lan Cao, Janet Shansky, Zheng Wang, David Mooney, Herman Vandenburgh

98) Restoration of the Majority of the Visual Spectrum by Using Modified Volvox Channelrhodopsin-1 (Molecular Therapy) Wednesday June 4th 2014
Hiroshi Tomita, Eriko Sugano, Namie Murayama, Taku Ozaki, Fumiaki Nishiyama, Kitako Tabata, Maki Takahashi, Takehiko Saito, Makoto Tamai

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