13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010101Abstract DirectoryThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthOligonucleotide & RNAi Therapeutics I252Preclinical Safety and Toxicity Assessment <strong>of</strong> Stathmin 1 BifunctionalshRNA Formulation Following a Single Intravenous Injection in RatsAnagha P. Phadke, Chris M. Jay, Donald D. Rao, Neil Senzer, Phillip B. Maples, JohnNemunaitis, Alex W. Tong.253Application <strong>of</strong> siRNA-Containing Nanoparticles for In Vitro and InVivo <strong>Gene</strong> Silencing in Tumor by Controlling Intracellular TraffickingHiroto Hatakeyama, Erika Ito, Hidetaka Akita, Motoi Oishi, Yukio Nagasaki, ShirohFutaki, Hideyoshi Harashima.254The Novel Comb Shaped Amphiphilic Polyallylamine for PotentialOral Delivery <strong>of</strong> siRNA to Gastrointestinal Cancers: PhysicochemicalCharacterizations and In Vitro EvaluationsJianfeng Guo, Colin J. Thompson, Woei Ping Cheng, Caitriona O’Driscoll.255Optimalization <strong>of</strong> Polymerase II sh/miRNA Expression forTherapeutic ApplicationsPiotr Maczuga, Tita Ritsema, Annemart Koorneef, Harald Petry, Sander van Deventer,Pavlina Konstantinova.256The Effect <strong>of</strong> Helper Lipids on Cationic Lipid-Based siRNA DeliverySystemGuoqin Niu, Hartmut Derendorf, Jeffrey A. Hughes.257c-Cbl shRNA-Expressing Adenovirus Functions as a Sensitizer <strong>of</strong>TRAIL-Induced ApoptosisSeeun Oh, So Y. Kim, Bo K. Sun, Dongho Kim, Suhung Lee, Yong J. Lee, Joo-Hang Kim,Jae J. Song.258ncRNAs Originating from the Dystrophin <strong>Gene</strong> as Biomarker forAssessing Antisense <strong>Therapy</strong>Matteo Bovolenta, Annarita Armaroli, Marcella Neri, Simona Brioschi, Marina Fabris,Chiara Scotton, S<strong>of</strong>ia Falzarano, Paola Rimessi, Emanuele Valli, Giovanni Perini, LucianoMerlini, Francesca Gualandi, Alessandra Ferlini.259Efficient In Vivo Delivery <strong>of</strong> Cholesterol-Conjugated siRNA to theLiver Using Endogenous Chylomicron RemnantKazutaka Nishina, Kie Yoshida, Yoshitaka Uno, Hiroya Kuwahara, Wenying Piao,Hidehiro Mizusawa, Takanori Yokota.Stem <strong>Cell</strong> Biology260Controlled Nonviral <strong>Gene</strong> Delivery and Expression Using StableNeural Stem <strong>Cell</strong> Line Transfected with a Hypoxia-Inducible <strong>Gene</strong>Expression SystemYoon Ha, MengLu Liu, KeungNyun Kim, DoHeum Yoon.261Non-Viral Carriers Deliver mRNA Encoding Marker Proteins in HeLacells and mRNA Encoding CXCR4 in Mesenchymal Stem <strong>Cell</strong>sJoanna Rejman, Geertrui Tavernier, Joseph Demeester, Stefaan S. D. Desmedt.262<strong>Gene</strong>tic Modification <strong>of</strong> Neural Stem <strong>Cell</strong>sRavi K. Chandra.263APOA-1 Is a Novel Marker <strong>of</strong> Erythroid <strong>Cell</strong> Maturation fromHematopoietic Stem <strong>Cell</strong>s in Mice and HumansTomoko Inoue, Daisuke Sugiyama, Ryo Kurita, Tatsuo Oikawa, Kasem Kulkeaw, HirotakaKawano, Yoshie Miura, Michiyo Okada, Youko Suehiro, Atsushi Takahashi, TomotoshiMarumoto, Hiroyuki Inoue, Norio Komatsu, Kenzaburo Tani.264A Stem <strong>Cell</strong> <strong>Gene</strong> <strong>Therapy</strong> Approach for Enhancing the Efficacy <strong>of</strong>Breast Cancer <strong>Therapy</strong> by Trastuzumab (Herceptin)Ines Beyer, Jonas Persson, ZongYi Li, Ying Liu, Roma Yumul, Andre Lieber.265Ovarian Cancer Stem <strong>Cell</strong>s Are in an Epithelial/Mesenchymal HybridState and Have Multipotential Capacity for DifferentiationRobert Strauss, ZongYi Li, Ying Liu, Nicole Urban, Charles Drescher, Jiri Bartek, AndreLieber.266SB-T-1214 Dramatically Downregulates Stem <strong>Cell</strong>-Related <strong>Gene</strong>Expression Pr<strong>of</strong>iles in Metastatic Colon Cancer <strong>Cell</strong>sYan Leyfman, Yuan Wang, Iwao Ojima, Galina Botchkina.267Identifying <strong>Gene</strong>s and Signaling Pathways Regulating the QuiescentState in Muscle Stem (Satellite) <strong>Cell</strong>sKristy Boyle, Jeffrey S. Chamberlain.268Pathway Analysis <strong>of</strong> Multipotent Mesenchymal Stromal <strong>Cell</strong>s UsingBacMam ToolsLucas Chase, Thomas Machleidt, Mahendra S. Rao, Uma Lakshmipathy.269The Placental Microvasculature Is a Rich Source <strong>of</strong> HighlyProliferative Endothelial Colony Forming <strong>Cell</strong>s Capable <strong>of</strong> DevelopingFunctioning Vessels In VivoBrian M. Rapp, Reza Saadatzadeh, Janak Bhasvar, Richard Ofstein, Michael P. Murphy.270microRNA Function during Human Mesenchymal Stem <strong>Cell</strong> AdipogenicDifferentiationAnnette Enright, Vladimir Benes, Georgina Shaw, Frank Barry, Linda Howard.Thursday, May 20 th
102<strong>American</strong> <strong>Society</strong> <strong>of</strong> <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>Abstract DirectoryThursday, May 20 thThursday, May 20, 2010Poster Session I — continued6:15 PM - 8:15 PMRoom: Exhibit Hall C & B-SouthStem <strong>Cell</strong> Biology271A Novel Fluorescence/Fluorine MRI Dual-Mode Reagent for <strong>Cell</strong>Tracking and Determination <strong>of</strong> <strong>Cell</strong> FateAnthony G. Balducci, Brooke Helfer, Amy K. Wesa, Jelena Janjic, Eric Ahrens.272The Stromal Vascular Fraction <strong>of</strong> Human Term Placenta Is a RichSource <strong>of</strong> Pluripotent Mesenchymal Stem <strong>Cell</strong>sMohammad R. Saadatzadeh, Janak Bhavsar, Brian M. Rapp, Peter J. Moreno, MichaelP. Murphy, Michael P. Murphy.273The HSV-2 Protein, ICP10PK, Potentiates the NeuroprotectiveActivity <strong>of</strong> Stem <strong>Cell</strong>sKyle A. Wilson, Jennifer M. Laing, Aurelian Laure.<strong>Cell</strong> Processing and Vector Production I274Process Improvements for cGMP Manufacture <strong>of</strong> Lexgenleucel-T forHIV TreatmentReuben Cohen, Yelena Skripchenko, Tony Encinas, Gerard J. McGarrity, Shuyuan Zhang.275TAG Vaccine Clinical Trial ManufacturingPhillip B. Maples, Padmasini Kumar, Yang Yu, Beena O. Pappen, Joseph Kuhn, JohnNemunaitis.276Development and GMP Validation <strong>of</strong> Large Scale LentiviralTransduction <strong>of</strong> CD34+ <strong>Cell</strong>s for Metacromatic Leukodystrophy andWiskott-Aldrich Syndrome <strong>Gene</strong> <strong>Therapy</strong> Clinical TrialsGiuliana Vallanti, Chiara Pozza, Francesca Rossetti, Letizia Martelli, Giuseppina Marano,Alessandra Gatti, Lucia Turchetto, Paola Massariello, Monica Salomoni, Claudia Benati,Sergio Fracchia, Alessandro Lorini, Marco Dieci, Raul Ziliotto, Alessandra Biffi, AlessandroAiuti, Claudio Bordignon, Luigi Naldini, Marina Radrizzani.277Large Scale rVSV Viral Vector Production in a cGMP FacilityLara J. Ausubel, Marcia Meseck, Ivy Derecho, Patricia Lopez, Christine Knoblauch, RossMcMahon, Jonathan Anderson, Nina Dunphy, Valerie Quezada, Mathew Luo, David Hsu,Savio Woo, Larry Couture.278Challenges in the Manufacturing <strong>of</strong> Retroviral <strong>Gene</strong> <strong>Therapy</strong> VectorsAna F. Rodrigues, Ana I. Amaral, Manuel J. T. Carrondo, Paula M. Alves, Ana S.Coroadinha.279Production <strong>of</strong> cGMP Grade Lentiviral VectorLara J. Ausubel, Christine Knoblauch, Patricia Lopez, Ross McMahon, Jonathan Anderson,Valerie Quezada, Nina Dunphy, Ivy Derecho, Mathew Luo, David Hsu, Larry Couture.280Design and Operation <strong>of</strong> a New State-<strong>of</strong>-the-Art Multi-Product cGMPCleanroom Facility for Vector Production and <strong>Cell</strong> ManipulationJohannes C. M. van der Loo, William P. Swaney, Kathryn Mangopoulos, MicheleBeuerlein, Diana L. Nordling, Lilith R. Reeves, Punam Malik.281Highly Efficient Manufacturing <strong>of</strong> Clinical Grade Lentiviral Vector in aNew, State <strong>of</strong> the Art Academic Good Manufacturing Practice (GMP)FacilityGerhard Bauer, Joseph Anderson, Matt Lindsey, Jan Nolta.282The Role <strong>of</strong> JNK in Lentivirus <strong>Gene</strong> TransferMeng Horng Lee, Roshan Padmashali, Stelios T. Andreadis.Late Breaking Science I922HSV-mediated Transfer <strong>of</strong> the Sox11 <strong>Gene</strong> and Peripheral NerveRegenerationXiaotang Jing, Shaohua Huang, Joseph C. Glorioso, Kathryn M. Albers923Real-Time In Vivo Imaging <strong>of</strong> Neural Stem <strong>Cell</strong>s for Targeted <strong>Therapy</strong><strong>of</strong> MedulloblastomaMargarita Gutova, Vazgen Kandalyyan, Ira Harutyunyan, Joseph Najbauer, Rex A.Moats, Gregory M. Shackleford, Michael E. Barish and Karen S. Aboody924Clinical Trial Entry <strong>of</strong> a Novel Prodrug Activator <strong>Gene</strong> TransferTechnology for the Treatment <strong>of</strong> CancerH. Gruber, C. Ibanez, A. daSilva, W. Gunzburg, D. Pertschuk, D. Gessner, N. Kasahara,C.R. Logg, O. Perez, D. Ostertag, J. Robbins, D.J. Jolly925Integration-Defective Lentiviral Vectors for Hemophilia: A SaferPlatform for Immune Tolerance Induction and Reduced GenotoxicityJanka Mátrai, Alessio Cantore, Cynthia C. Bartholomae, A Annoni, Abel Acosta-Sanchez, Ermira Samara-Kuko, Liesbeth De Waele, Ling Ma, Tim C. Nichols, Christ<strong>of</strong>von Kalle, Roncarolo, Marie-Gracia, Marinee K.L. Chuah, Manfred Schmidt, ThierryVandenDriessche, Luigi Naldini926Abstract Withdrawn927Targeting CD70 Positive Malignancies with T cells Expressinga CD27-Zeta Chimeric Antigen Receptor Reveals ImportantCostimulatory Role <strong>of</strong> CD27D. Shaffer, B Savoldo, G. Dotti, D Spencer, S. Kenney, C.M. Rooney, S. GottschalkEXHIBITOR PROSPECTUSfinal program
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