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1) Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa (Molecular Therapy) Tuesday February 24th 2015
Adeline Berger, St, Charl, Melissa Desrosiers, C, Thomas Voit, Luis Garcia, Jos, Alexis-Pierre Bemelmans

2) Paracrine Factors of Human Fetal MSCs Inhibit Liver Cancer Growth Through Reduced Activation of IGF-1R/PI3K/Akt Signaling (Molecular Therapy) Tuesday February 24th 2015
Yulyana Yulyana, Ivy A W Ho, Kian Chuan Sia, Jennifer P Newman, Xin Yi Toh, Berwini B Endaya, Jerry K Y Chan, Massimiliano Gnecchi, Hung Huynh, Alexander Y F Chung, Kiat Hon Lim, Hui Sun Leong, Narayanan Gopalakrishna Iyer, Kam Man Hui, Paula Y P Lam

3) Chimeric Antigen Receptors With Mutated IgG4 Fc Spacer Avoid Fc Receptor Binding and Improve T Cell Persistence and Antitumor Efficacy (Molecular Therapy) Tuesday February 17th 2015
Mahesh Jonnalagadda, Armen Mardiros, Ryan Urak, Xiuli Wang, Lauren J Hoffman, Alyssa Bernanke, Wen-Chung Chang, William Bretzlaff, Renate Starr, Saul Priceman, Julie R Ostberg, Stephen J Forman, Christine E Brown

4) PET Imaging of Oncolytic VSV Expressing the Mutant HSV-1 Thymidine Kinase Transgene in a Preclinical HCC Rat Model (Molecular Therapy) Tuesday February 17th 2015
Kim A Mu, Jennifer Altomonte, Iina Laitinen, Sibylle Ziegler, Katja Steiger, Irene Esposito, Roland M Schmid, Oliver Ebert

5) Controlled Striatal DOPA Production From a Gene Delivery System in a Rodent Model of Parkinson (Molecular Therapy) Tuesday February 17th 2015
Erik Cederfj, Lauren Broom, Deniz Kirik

6) Cell Cycle Status of CD34+ Hemopoietic Stem Cells Determines Lentiviral Integration in Actively Transcribed and Development-related Genes (Molecular Therapy) Tuesday February 10th 2015
Eleni Papanikolaou, Anna Paruzynski, Ioannis Kasampalidis, Annette Deichmann, Evangelos Stamateris, Manfred Schmidt, Christof von Kalle, Nicholas P Anagnou

7) First-in-man Phase 1 Clinical Trial of Gene Therapy for Advanced Pancreatic Cancer: Safety, Biodistribution, and Preliminary Clinical Findings (Molecular Therapy) Tuesday February 10th 2015
Louis Buscail, Barbara Bournet, Fabienne Vernejoul, Gilles Cambois, Hubert Lulka, Na, Marl, Aline Meulle, Alix Vignolle-Vidoni, Laetitia Ligat, Nathalie Saint-Laurent, Fr, S, Marion Gayral, Fr, J, Odile Barbey, Fabian Gross, Rosine Guimbaud, Philippe Otal, Fr, G, Pierre Cordelier

8) The Clinical Relevance of the miR-197/CKS1B/STAT3-mediated PD-L1 Network in Chemoresistant Non-small-cell Lung Cancer (Molecular Therapy) Tuesday February 10th 2015
Yu Fujita, Shigehiro Yagishita, Keitaro Hagiwara, Yusuke Yoshioka, Nobuyoshi Kosaka, Fumitaka Takeshita, Tomohiro Fujiwara, Koji Tsuta, Hiroshi Nokihara, Tomohide Tamura, Hisao Asamura, Makoto Kawaishi, Kazuyoshi Kuwano, Takahiro Ochiya

9) High Capsid (Molecular Therapy) Tuesday February 10th 2015
Mathieu Nonnenmacher, Harm van Bakel, Roger J Hajjar, Thomas Weber

10) Enhancing Antitumor Efficacy of Chimeric Antigen Receptor T Cells Through Constitutive CD40L Expression (Molecular Therapy) Tuesday February 10th 2015
Kevin J Curran, Beatrijs A Seinstra, Yan Nikhamin, Raymond Yeh, Yelena Usachenko, Dayenne G van Leeuwen, Terence Purdon, Hollie J Pegram, Renier J Brentjens

11) Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy (Molecular Therapy) Tuesday February 10th 2015
Yi Gong, Dakai Mu, Shilpa Prabhakar, Ann Moser, Patricia Musolino, JiaQian Ren, Xandra O Breakefield, Casey A Maguire, Florian S Eichler

12) Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice (Molecular Therapy) Tuesday February 10th 2015
F Jason Duncan, Bartholomew J Naughton, Kimberly Zaraspe, Darren A Murrey, Aaron S Meadows, Kelly Reed Clark, David E Newsom, Peter White, Haiyan Fu, Douglas M McCarty

13) Hybrid Nonviral/Viral Vector Systems for Improved piggyBac DNA Transposon In Vivo Delivery (Molecular Therapy) Tuesday February 3rd 2015
Ashley L Cooney, Brajesh K Singh, Patrick L Sinn

14) Characterization of a Bispecific FLT3 X CD3 Antibody in an Improved, Recombinant Format for the Treatment of Leukemia (Molecular Therapy) Tuesday February 3rd 2015
Michael Durben, Dominik Schmiedel, Martin Hofmann, Fabian Vogt, Tina N, Elwira Pyz, Hans-J, Hans-Georg Rammensee, Helmut R Salih, Ludger Gro, Gundram Jung

15) Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid Mutants (Molecular Therapy) Tuesday February 3rd 2015
Matthew J Benskey, Nathan C Kuhn, James J Galligan, Joanna Garcia, Shannon E Boye, William W Hauswirth, Christian Mueller, Sanford L Boye, Fredric P Manfredsson

16) Mutated BRAF Emerges as a Major Effector of Recurrence in a Murine Melanoma Model After Treatment With Immunomodulatory Agents (Molecular Therapy) Tuesday January 27th 2015
Shane Zaidi, Miran Blanchard, Kevin Shim, Elizabeth Ilett, Karishma Rajani, Christopher Parrish, Nicolas Boisgerault, Tim Kottke, Jill Thompson, Esteban Celis, Jose Pulido, Peter Selby, Hardev Pandha, Alan Melcher, Kevin Harrington, Richard Vile

17) Enzyme Enhancers for the Treatment of Fabry and Pompe Disease (Molecular Therapy) Tuesday January 20th 2015
Jan Lukas, Anne-Marie Pockrandt, Susanne Seemann, Muhammad Sharif, Franziska Runge, Susann Pohlers, Chaonan Zheng, Anne Gl, Matthias Beller, Arndt Rolfs, Anne-Katrin Giese

18) Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency (Molecular Therapy) Tuesday January 20th 2015
Marlene Carmo, Kimberly A Risma, Paritha Arumugam, Swati Tiwari, Adrianne E Hontz, Claudia A Montiel-Equihua, Maria E Alonso-Ferrero, Michael P Blundell, Axel Schambach, Christopher Baum, Punam Malik, Adrian J Thrasher, Michael B Jordan, H Bobby Gaspar

19) Biological Ablation of Sentinel Lymph Node Metastasis in Submucosally Invaded Early Gastrointestinal Cancer (Molecular Therapy) Tuesday January 20th 2015
Satoru Kikuchi, Hiroyuki Kishimoto, Hiroshi Tazawa, Yuuri Hashimoto, Shinji Kuroda, Masahiko Nishizaki, Takeshi Nagasaka, Yasuhiro Shirakawa, Shunsuke Kagawa, Yasuo Urata, Robert M Hoffman, Toshiyoshi Fujiwara

20) Synthetic Consensus HIV-1 DNA Induces Potent Cellular Immune Responses and Synthesis of Granzyme B, Perforin in HIV Infected Individuals (Molecular Therapy) Tuesday January 20th 2015
Matthew P Morrow, Pablo Tebas, Jian Yan, Lorenzo Ramirez, Anna Slager, Kim Kraynyak, Malissa Diehl, Divya Shah, Amir Khan, Jessica Lee, Jean Boyer, J Joseph Kim, Niranjan Y Sardesai, David B Weiner, Mark L Bagarazzi

21) HDAC Inhibitors Target HDAC5, Upregulate MicroRNA-125a-5p, and Induce Apoptosis in Breast Cancer Cells (Molecular Therapy) Tuesday January 20th 2015
Tsung-Hua Hsieh, Chia-Yi Hsu, Cheng-Fang Tsai, Cheng-Yu Long, Chin-Hu Wu, Deng-Chyang Wu, Jau-Nan Lee, Wei-Chun Chang, Eing-Mei Tsai

22) Depletion of Bone Marrow CCSP-Expressing Cells Delays Airway Regeneration (Molecular Therapy) Tuesday January 13th 2015
Martha L Bustos, Marco Mura, David Hwang, Olga Ludkovski, Amy P Wong, Armand Keating, Thomas K Waddell

23) Cell-sheet Therapy With Omentopexy Promotes Arteriogenesis and Improves Coronary Circulation Physiology in Failing Heart (Molecular Therapy) Tuesday January 13th 2015
Satoshi Kainuma, Shigeru Miyagawa, Satsuki Fukushima, James Pearson, Yi Ching Chen, Atsuhiro Saito, Akima Harada, Motoko Shiozaki, Hiroko Iseoka, Tadashi Watabe, Hiroshi Watabe, Genki Horitsugi, Mana Ishibashi, Hayato Ikeda, Hirotsugu Tsuchimochi, Takashi Sonobe, Yutaka Fujii, Hisamichi Naito, Keiji Umetani, Tatsuya Shimizu, Teruo Okano, Eiji Kobayashi, Takashi Daimon, Takayoshi Ueno, Toru Kuratani, Koichi Toda, Nobuyuki Takakura, Jun Hatazawa, Mikiyasu Shirai, Yoshiki Sawa

24) A New Cell-penetrating Peptide That Blocks the Autoinhibitory XIP Domain of NCX1 and Enhances Antiporter Activity (Molecular Therapy) Tuesday January 13th 2015
Pasquale Molinaro, Anna Pannaccione, Maria Jos, Agnese Secondo, Ornella Cuomo, Rossana Sirabella, Maria Cantile, Roselia Ciccone, Antonella Scorziello, Gianfranco di Renzo, Lucio Annunziato

25) Sourcing of an Alternative Pericyte-Like Cell Type from Peripheral Blood in Clinically Relevant Numbers for Therapeutic Angiogenic Applications (Molecular Therapy) Tuesday January 13th 2015
Anna Blocki, Yingting Wang, Maria Koch, Anna Goralczyk, Sebastian Beyer, Nikita Agarwal, Michelle Lee, Shehzahdi Moonshi, Jean-Yves Dewavrin, Priscilla Peh, Herbert Schwarz, Kishore Bhakoo, Michael Raghunath

26) Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases (Molecular Therapy) Tuesday January 13th 2015
David G Ousterout, Ami M Kabadi, Pratiksha I Thakore, Pablo Perez-Pinera, Matthew T Brown, William H Majoros, Timothy E Reddy, Charles A Gersbach

27) Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates (Molecular Therapy) Tuesday January 13th 2015
Jagdeep S Walia, Naderah Altaleb, Alexander Bello, Christa Kruck, Matthew C LaFave, Gaurav K Varshney, Shawn M Burgess, Biswajit Chowdhury, David Hurlbut, Richard Hemming, Gary P Kobinger, Barbara Triggs-Raine

28) In Vivo AAV1 Transduction With hRheb(S16H) Protects Hippocampal Neurons by BDNF Production (Molecular Therapy) Tuesday January 13th 2015
Min-Tae Jeon, Jin Han Nam, Won-Ho Shin, Eunju Leem, Kyoung Hoon Jeong, Un Ju Jung, Young-Seuk Bae, Young-Ho Jin, Nikolai Kholodilov, Robert E Burke, Seok-Geun Lee, Byung Kwan Jin, Sang Ryong Kim

29) Phase 1 Study of Intratumoral Pexa-Vec (JX-594), an Oncolytic and Immunotherapeutic Vaccinia Virus, in Pediatric Cancer Patients (Molecular Therapy) Tuesday January 13th 2015
Timothy P Cripe, Minhtran C Ngo, James I Geller, Chrystal U Louis, Mark A Currier, John M Racadio, Alexander J Towbin, Cliona M Rooney, Adina Pelusio, Anne Moon, Tae-Ho Hwang, James M Burke, John C Bell, David H Kirn, Caroline J Breitbach

30) PTEN Depletion Decreases Disease Severity and Modestly Prolongs Survival in a Mouse Model of Spinal Muscular Atrophy (Molecular Therapy) Tuesday January 6th 2015
Daniel Little, Chiara F Valori, Chantal A Mutsaers, Ellen J Bennett, Matthew Wyles, Basil Sharrack, Pamela J Shaw, Thomas H Gillingwater, Mimoun Azzouz, Ke Ning

31) Functional Aspects of Intrahepatic Hepatitis B Virus-specific T Cells Induced by Therapeutic DNA Vaccination (Molecular Therapy) Tuesday January 6th 2015
Anette Brass, Lars Frelin, David R Milich, Matti S, Gustaf Ahl

32) Intrinsic Transgene Immunogenicity Gears CD8+ T-cell Priming After rAAV-Mediated Muscle Gene Transfer (Molecular Therapy) Tuesday January 6th 2015
Maxime Carpentier, St, Pascal Chappert, M, Romain Hardet, Dominique Urbain, C, Sahil Adriouch, Luis Garcia, Jean Davoust, David-Alexandre Gross

33) Transplantation of Glial Progenitors That Overexpress Glutamate Transporter GLT1 Preserves Diaphragm Function Following Cervical SCI (Molecular Therapy) Tuesday January 6th 2015
Ke Li, Elham Javed, Tamara J Hala, Daniel Sannie, Kathleen A Regan, Nicholas J Maragakis, Megan C Wright, David J Poulsen, Angelo C Lepore

34) Inhibitory/Suppressive Oligodeoxynucleotide Nanocapsules as Simple Oral Delivery Devices for Preventing Atopic Dermatitis in Mice (Molecular Therapy) Tuesday January 6th 2015
Yeqin Wang, Yoshinari Yamamoto, Suguru Shigemori, Takafumi Watanabe, Kazushi Oshiro, Xinyu Wang, Pengfei Wang, Takashi Sato, Shinichi Yonekura, Sachi Tanaka, Haruki Kitazawa, Takeshi Shimosato

35) 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

36) 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

37) 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

38) 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

39) 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

40) 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

41) 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

42) 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

43) 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

44) 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

45) 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

46) 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

47) 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

48) 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

49) 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

50) 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

51) 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

52) 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

53) 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


54) 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

55) 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

56) 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

57) 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

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


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


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


61) 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

62) 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

63) 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

64) 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

65) 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

66) 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

67) 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

68) 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

69) 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

70) 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

71) 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

72) 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

73) 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

74) 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

75) 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

76) 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

77) 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

78) 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

79) 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

80) 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

81) 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

82) 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

83) 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

84) 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

85) 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

86) 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

87) 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

88) 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

89) 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

90) 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

91) 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

92) 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

93) 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

94) 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

95) 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

96) 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

97) 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

98) 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

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