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1) Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders (Molecular Therapy) Tuesday May 19th 2015
Giancarlo Parenti, Generoso Andria, Kenneth J Valenzano

2) Experimental Curative Fluorescence-guided Surgery of Highly Invasive Glioblastoma Multiforme Selectively Labeled With a Killer-reporter Adenovirus (Molecular Therapy) Tuesday May 19th 2015
Shuya Yano, Shinji Miwa, Hiroyuki Kishimoto, Makoto Toneri, Yukihiko Hiroshima, Mako Yamamoto, Michael Bouvet, Yasuo Urata, Hiroshi Tazawa, Shunsuke Kagawa, Toshiyoshi Fujiwara, Robert M Hoffman

3) Inhibition of Dexamethasone-induced Fatty Liver Development by Reducing miR-17-5p Levels (Molecular Therapy) Tuesday May 19th 2015
William W Du, Fengqiong Liu, Sze Wan Shan, Xindi Cindy Ma, Shaan Gupta, Tianru Jin, David Spaner, Sergey N Krylov, Yaou Zhang, Wenhua Ling, Burton B Yang

4) Monocyte/Macrophage-derived IGF-1 Orchestrates Murine Skeletal Muscle Regeneration and Modulates Autocrine Polarization (Molecular Therapy) Tuesday May 19th 2015
Joanne Tonkin, Lieve Temmerman, Robert D Sampson, Enrique Gallego-Colon, Laura Barberi, Daniel Bilbao, Michael D Schneider, Antonio Musar, Nadia Rosenthal

5) Mesenchymal Stromal Cells Derived From Crohn (Molecular Therapy) Tuesday May 19th 2015
Raghavan Chinnadurai, Ian B Copland, Spencer Ng, Marco Garcia, Mahadev Prasad, Dalia Arafat, Greg Gibson, Subra Kugathasan, Jacques Galipeau

6) Engineering T Cells to Functionally Cure HIV-1 Infection (Molecular Therapy) Tuesday May 19th 2015
Rachel S Leibman, James L Riley

7) The Cancer Genome Atlas Analysis Predicts MicroRNA for Targeting Cancer Growth and Vascularization in Glioblastoma (Molecular Therapy) Tuesday May 19th 2015
Hon-Kit Andus Wong, Rachid El Fatimy, Courtney Onodera, Zhiyun Wei, Ming Yi, Athul Mohan, Sindhuja Gowrisankaran, Priya Karmali, Eric Marcusson, Hiroaki Wakimoto, Robert Stephens, Erik J Uhlmann, Jun S Song, Bakhos Tannous, Anna M Krichevsky

8) [18F]FHBG PET/CT Imaging of CD34-TK75 Transduced Donor T Cells in Relapsed Allogeneic Stem Cell Transplant Patients: Safety and Feasibility (Molecular Therapy) Tuesday May 12th 2015
Linda G Eissenberg, Michael P Rettig, Julie K Ritchey, Julie L Prior, Sally W Schwarz, Jennifer Frye, Brian S White, Robert S Fulton, Armin Ghobadi, Matthew L Cooper, Daniel R Couriel, Muhammad Esa Seegulam, David Piwnica-Worms, Farrokh Dehdashti, Kenneth Cornetta, John F DiPersio

9) Case Report of a Fatal Serious Adverse Event Upon Administration of T Cells Transduced With a MART-1-specific T-cell Receptor (Molecular Therapy) Tuesday May 12th 2015
Joost H van den Berg, Raquel Gomez-Eerland, Bart van de Wiel, Lenie Hulshoff, Daan van den Broek, Adriaan Bins, Hanno L Tan, Jane V Harper, Namir J Hassan, Bent K Jakobsen, Annelies Jorritsma, Christian U Blank, Ton N M Schumacher, John B A G Haanen

10) Barrier Function of the Repaired Skin Is Disrupted Following Arrest of Dicer in Keratinocytes (Molecular Therapy) Tuesday May 12th 2015
Subhadip Ghatak, Yuk Cheung Chan, Savita Khanna, Jaideep Banerjee, Jessica Weist, Sashwati Roy, Chandan K Sen

11) Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors (Molecular Therapy) Tuesday May 5th 2015
Maxime Ferrand, Sylvie Da Rocha, Guillaume Corre, Anne Galy, Florence Boisgerault

12) Phase 1 Trial of Bi-shRNA STMN1 BIV in Refractory Cancer (Molecular Therapy) Tuesday April 28th 2015
Minal Barve, Zhaohui Wang, Padmasini Kumar, Christopher M Jay, Xiuquan Luo, Cynthia Bedell, Robert G Mennel, Gladice Wallraven, Francis Charles Brunicardi, Neil Senzer, John Nemunaitis, Donald D Rao

13) Pilot Trial of FANG Immunotherapy in Ewing (Molecular Therapy) Tuesday April 28th 2015
Maurizio Ghisoli, Minal Barve, Reva Schneider, Robert Mennel, Carl Lenarsky, Gladice Wallraven, Beena O Pappen, John LaNoue, Padmasini Kumar, Derek Nemunaitis, Alyssa Roth, James Nemunaitis, Sam Whiting, Neil Senzer, Frederick A Fletcher, John Nemunaitis

14) Blocking the Adhesion Cascade at the Premetastatic Niche for Prevention of Breast Cancer Metastasis (Molecular Therapy) Tuesday April 28th 2015
Shin-Ae Kang, Nafis Hasan, Aman P Mann, Wei Zheng, Lichao Zhao, Lynsie Morris, Weizhu Zhu, Yan D Zhao, K Stephen Suh, William C Dooley, David Volk, David G Gorenstein, Massimo Cristofanilli, Hallgeir Rui, Takemi Tanaka

15) High Local Concentrations of Intradermal MSCs Restore Skin Integrity and Facilitate Wound Healing in Dystrophic Epidermolysis Bullosa (Molecular Therapy) Tuesday April 28th 2015
Tobias K, Markus Mezger, Ingrid Hausser, Rupert Handgretinger, Leena Bruckner-Tuderman, Alexander Nystr

16) Action of Obestatin in Skeletal Muscle Repair: Stem Cell Expansion, Muscle Growth, and Microenvironment Remodeling (Molecular Therapy) Tuesday April 21st 2015
Ux, Ic, Jessica Gonz, Daniel Beiroa, Viviana Moresi, Carlos S Mosteiro, Wei Lin, Juan E Vi, Jos, Tom, Felipe F Casanueva, Rub, Rosal, Jean-Marc Renaud, Sergio Adamo, Yolanda Pazos, Jes

17) Polarized Macrophages Have Distinct Roles in the Differentiation and Migration of Embryonic Spinal-cord-derived Neural Stem Cells After Grafting to Injured Sites of Spinal Cord (Molecular Therapy) Tuesday April 21st 2015
Kun Zhang, Jingjing Zheng, Ganlan Bian, Ling Liu, Qian Xue, Fangfang Liu, Caiyong Yu, Haifeng Zhang, Bing Song, Sookja K Chung, Gong Ju, Jian Wang

18) From Marrow to Matrix: Novel Gene and Cell Therapies for Epidermolysis Bullosa (Molecular Therapy) Tuesday April 21st 2015
Beau R Webber, Jakub Tolar

19) Complement Inhibition Prevents Oncolytic Vaccinia Virus Neutralization in Immune Humans and Cynomolgus Macaques (Molecular Therapy) Tuesday April 21st 2015
Laura Evgin, Sergio A Acuna, Christiano Tanese de Souza, Monique Marguerie, Chantal G Lemay, Carolina S Ilkow, C Scott Findlay, Theresa Falls, Kelley A Parato, David Hanwell, Alyssa Goldstein, Roberto Lopez, Sandra Lafrance, Caroline J Breitbach, David Kirn, Harold Atkins, Rebecca C Auer, Joshua M Thurman, Gregory L Stahl, John D Lambris, John C Bell, J Andrea McCart

20) Intraspinal Delivery of Polyethylene Glycol-coated Gold Nanoparticles Promotes Functional Recovery After Spinal Cord Injury (Molecular Therapy) Tuesday April 21st 2015
Florentia Papastefanaki, Igor Jakovcevski, Nafsika Poulia, Nevena Djogo, Florian Schulz, Tamara Martinovic, Darko Ciric, Gabrielle Loers, Tobias Vossmeyer, Horst Weller, Melitta Schachner, Rebecca Matsas

21) Excision of Expanded GAA Repeats Alleviates the Molecular Phenotype of Friedreich (Molecular Therapy) Tuesday April 14th 2015
Yanjie Li, Urszula Polak, Angela D Bhalla, Natalia Rozwadowska, Jill Sergesketter Butler, David R Lynch, Sharon Y R Dent, Marek Napierala

22) Modular Three-component Delivery System Facilitates HLA Class I Antigen Presentation and CD8+ T-cell Activation Against Tumors (Molecular Therapy) Tuesday April 14th 2015
Benjamin J Umlauf, Chin-Ying Chung, Kathlynn C Brown

23) Mesenchymal Stem Cell-derived Extracellular Vesicles: Toward Cell-free Therapeutic Applications (Molecular Therapy) Tuesday April 14th 2015
Sweta Rani, Aideen E Ryan, Matthew D Griffin, Thomas Ritter

24) Long Noncoding RNA Arid2-IR Is a Novel Therapeutic Target for Renal Inflammation (Molecular Therapy) Tuesday March 31st 2015
Qin Zhou, Xiao R Huang, Jianwen Yu, Xueqing Yu, Hui Y Lan

25) Gene Therapy With the Caspase Activation and Recruitment Domain Reduces the Ocular Inflammatory Response (Molecular Therapy) Tuesday March 17th 2015
Cristhian J Ildefonso, Henrique Jaime, Manas R Biswal, Shannon E Boye, Qiuhong Li, William W Hauswirth, Alfred S Lewin

26) BRAF- and MEK-Targeted Small Molecule Inhibitors Exert Enhanced Antimelanoma Effects in Combination With Oncolytic Reovirus Through ER Stress (Molecular Therapy) Tuesday March 10th 2015
Victoria Roulstone, Malin Pedersen, Joan Kyula, David Mansfield, Aadil A Khan, Grainne McEntee, Michelle Wilkinson, Eleni Karapanagiotou, Matt Coffey, Richard Marais, Adel Jebar, Fiona Errington-Mais, Alan Melcher, Richard Vile, Hardev Pandha, Martin McLaughlin, Kevin J Harrington

27) Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice (Molecular Therapy) Tuesday March 10th 2015
Maria Dahl, Alexander Doyle, Karin Olsson, Jan-Eric M, Andr, Mina Mirzaian, Johannes M Aerts, Mats Ehinger, Michael Rothe, Ute Modlich, Axel Schambach, Stefan Karlsson

28) T-cell Subsets in Peripheral Blood and Tumors of Patients Treated With Oncolytic Adenoviruses (Molecular Therapy) Tuesday March 10th 2015
Taipale Kristian, Liikanen Ilkka, Juhila Juuso, Karioja-Kallio Aila, Oksanen Minna, Turkki Riku, Linder Nina, Lundin Johan, Ristim, Kanerva Anna, Koski Anniina, Joensuu Timo, V, Hemminki Akseli

29) Host-based Prophylaxis Successfully Targets Liver Stage Malaria Parasites (Molecular Therapy) Tuesday March 10th 2015
Alyse N Douglass, Heather S Kain, Marian Abdullahi, Nadia Arang, Laura S Austin, Sebastian A Mikolajczak, Zachary P Billman, Jen C C Hume, Sean C Murphy, Stefan H I Kappe, Alexis Kaushansky

30) Lentivirus-mediated Gene Transfer in Hematopoietic Stem Cells Is Impaired in SHIV-infected, ART-treated Nonhuman Primates (Molecular Therapy) Tuesday March 10th 2015
Patrick M Younan, Christopher W Peterson, Patricia Polacino, John P Kowalski, Willimark Obenza, Hannah W Miller, Brian P Milless, Phil Gafken, Stephen C DeRosa, Shiu-Lok Hu, Hans-Peter Kiem

31) Intraosseous Delivery of Lentiviral Vectors Targeting Factor VIII Expression in Platelets Corrects Murine Hemophilia A (Molecular Therapy) Tuesday March 10th 2015
Xuefeng Wang, Simon C Shin, Andy F J Chiang, Iram Khan, Dao Pan, David J Rawlings, Carol H Miao

32) Disruption of KATP Channel Expression in Skeletal Muscle by Targeted Oligonucleotide Delivery Promotes Activity-linked Thermogenesis (Molecular Therapy) Tuesday March 10th 2015
Siva Rama Krishna Koganti, Zhiyong Zhu, Ekaterina Subbotina, Zhan Gao, Ana Sierra, Manuel Proenza, Liping Yang, Alexey Alekseev, Denice Hodgson-Zingman, Leonid Zingman

33) Intracellular Delivery System for Antibody (Molecular Therapy) Tuesday March 10th 2015
Geoffrey Y Berguig, Anthony J Convertine, Shani Frayo, Hanna B Kern, Erik Procko, Debashish Roy, Selvi Srinivasan, Daciana H Margineantu, Garrett Booth, Maria Corinna Palanca-Wessels, David Baker, David Hockenbery, Oliver W Press, Patrick S Stayton

34) Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression (Molecular Therapy) Tuesday March 10th 2015
Maria Grazia Biferi, Carmine Nicoletti, Germana Falcone, Eleonora M R Puggioni, Nunzia Passaro, Alessia Mazzola, Deborah Pajalunga, Germana Zaccagnini, Emanuele Rizzuto, Alberto Auricchio, Lorena Zentilin, Gabriele De Luca, Mauro Giacca, Fabio Martelli, Antonio Musio, Antonio Musar, Marco Crescenzi

35) cAMP and EPAC Signaling Functionally Replace OCT4 During Induced Pluripotent Stem Cell Reprogramming (Molecular Therapy) Tuesday March 10th 2015
Ashley L Fritz, Maroof M Adil, Sunnie R Mao, David V Schaffer

36) Overcoming Insulin Insufficiency by Forced Follistatin Expression in (Molecular Therapy) Tuesday March 10th 2015
Chunxia Zhao, Chunping Qiao, Ru-Hang Tang, Jiangang Jiang, Jianbin Li, Carrie Bette Martin, Karen Bulaklak, Juan Li, Dao Wen Wang, Xiao Xiao

37) 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

38) 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

39) 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

40) 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

41) 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

42) 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

43) 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

44) 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

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

46) 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

47) 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

48) 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

49) 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

50) 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

51) 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

52) 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

53) 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

54) 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

55) 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

56) 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

57) 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

58) 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

59) 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

60) 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

61) 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

62) 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

63) 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

64) 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

65) 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

66) 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

67) 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

68) 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

69) 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

70) 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

71) 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

72) 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

73) 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

74) 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

75) 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

76) 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

77) 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

78) 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

79) 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

80) 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

81) 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

82) 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

83) 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

84) 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

85) 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

86) 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

87) 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

88) 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

89) 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


90) 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

91) 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

92) 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

93) 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

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


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


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


97) 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

98) 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

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Mitch Andre Garcia's Chem Feeds 2008-present

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