13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 87Abstract DirectoryThursday, May 20, 2010Oral Abstract Session 232Neurologic & Ophthalmic <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Wilson ABC17 2:00 PMAAV-Mediated <strong>Gene</strong> <strong>Therapy</strong> Restores Visual Function and Behaviorto a Mouse Model <strong>of</strong> Leber Congential Amaurosis-1 (LCA1)Shannon E. Boye, Sanford L. Boye, Jijing Pang, Renee C. Ryals, Andy W. Neeley, DrewEverhart, Yumiko Umino, Joseph C. Besharse, Robert Barlow, William W. Hauswirth.18 2:15 PMAdeno-Associated Virus Vector Expressing an Amyloid beta 1-42Intrabody Rescues White Matter Pathology during Early Alzheimer’sDiseaseMaya K. Desai, Michael A. Mastrangelo, Deborah A. Ryan, Kelly L. Sudol, Wade C.Narrow, William J. Bowers.19 2:30 PMBrain <strong>Gene</strong> <strong>Therapy</strong> for Metachromatic Leucodystrophy: TowardsClinical TrialCaroline Sevin, Dolan Sondhi, Françoise Piguet, Marie-Anne Colle, Sylvie Raoul, ThomasRoujeau, Monique Piraud, Ornella Ahouansou, Celine Bouquet, Marie-Thérèse Vanier,Philippe Moullier, Yan Cherel, Neil R. Hackett, Michel Zerah, Patrick Aubourg, Ronald G.Crystal, Nathalie Cartier.20 2:45 PMNeonatal Intracerebroventricular Infusion <strong>of</strong> AAV2/8 Vector Leadsto High Levels <strong>of</strong> Human a-L-Iduronidase Expression in the Brainand Complete Neurophenotypic Correction in a Murine Model <strong>of</strong>Mucopolysaccharidosis Type IDaniel A. Wolf, Andrew W. Lenander, Zhenhong Nan, Lalitha R. Belur, Debra Swanson,Chester B. Whitley, Walter C. Low, R. Scott McIvor.21 3:00 PMCircuit-Specific Expression <strong>of</strong> Channelrhodopsin Restores VisualFunction in Blind rd1, rd16, and rho -/- MiceAlan Horsager, Jian Wen Liu, Edward S. Boyden, A. Cyrus Arman, Kimberly A. Silka,Mehdi Doroudchi, Shannon E. Boye, Benjamin C. Matteo, Alapakkam P. Sampath,William W. Hauswirth.22 3:15 PMCombination <strong>Therapy</strong> for the Treatment <strong>of</strong> Infantile Neuronal CeroidLip<strong>of</strong>uscinosis (INCL)Shannon L. Macauley-Rambach, Marie S. Roberts, Mark S. Sands.23 3:30 PMrAAV Delivery <strong>of</strong> a-Synuclein-Specific Single Chain AntibodiesAttenuates Dopamine Loss in a Mouse Model <strong>of</strong> Parkinson’s DiseaseKathleen Maguire-Zeuss, William J. Bowers, Wade Narrow, Eric Yehling, Howard J.Feder<strong>of</strong>f.24 3:45 PMPatterns <strong>of</strong> Motor Function Recovery in a Murine Model <strong>of</strong> SevereMenkes Disease Rescued by Brain-Directed AAV5 <strong>Gene</strong> <strong>Therapy</strong> PlusCopperAnthony Donsante, Stephen G. Kaler.Oral Abstract Session 233Cancer – Immunotherapy T <strong>Cell</strong> Studies2:00 PM - 4:00 PMRoom: Thurgood Marshall W25 2:00 PMAdoptive Transfer <strong>of</strong> Peripheral Blood Lymphocytes <strong>Gene</strong>ticallyModified To Express a T <strong>Cell</strong> Receptor Recognizing CarcinoembryonicAntigen into Patients with Metastatic Colorectal Cancer InducedInflammatory Colitis without Mediating Anti-Tumor EffectsMaria R. Parkhurst, James C. Yang, Russell C. Langan, Steven A. Feldman, Mark E.Dudley, Paul F. Robbins, Steven A. Rosenberg.26 2:15 PMAdenovirus Mediated Expression <strong>of</strong> Flt3L and HSV1-TK within theBrain Tumor Microenvironment Elicits Higher Therapeutic EfficacyThan Dendritic <strong>Cell</strong>s’ VaccinationYohei Mineharu, Gwendalyn D. King, Marianela Candolfi, Weidong Xiong, Hikmat Assi,Kader Yagiz, Kurt M. Kroeger, Patricia Lin, B. Serguei, Pedro R. Lowenstein, Maria G.Castro.27 2:30 PMPharmacokinetics <strong>of</strong> Anti-PSMA <strong>Gene</strong>-Modified T <strong>Cell</strong>s Infused afterNon-Myeloablative Conditioning in Prostate CancerAnthony J. Bais, Qiangzhong Ma, Hannah Boss, Esther L. Beaudoin, Richard P. Junghans.28 2:45 PMBinary Control <strong>of</strong> Tumor-Specific Cytotoxic T Lymphocytes byTransgenic IL7 and IL7r ExpressionJuan F. Vera, Ceidy Sanchez, Sujatha Muralidharan, Helen E. Heslop, Cliona M. Rooney,Gianpietro Dotti, Malcolm K. Brenner.29 3:00 PMMulti-Tumor-Antigen-Specific Cytotoxic T Lymphocytes for CancerImmunotherapyUlrike Gerdemann, Juan F. Vera, Anne S. Christin, Russell C. Cruz, Helen E. Heslop,Malcolm K. Brenner, Catherine M. Bollard, Cliona M. Rooney, Ann M. Leen.30 3:15 PMSimultaneous Comparison <strong>of</strong> T <strong>Cell</strong>s Expressing 1 st or 2 nd <strong>Gene</strong>rationCARs in Human Subjects with B-<strong>Cell</strong> Malignancies: Contribution <strong>of</strong>Costimulatory EndodomainsB. Savoldo, C. Ramos, C. Bollard, E. Liu, M. Mims, M. Keating, A. Gee, Z. Mei, C.Rooney, H. Heslop, M. Brenner, G. Dotti.Thursday, May 20 th
88<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, 2010Oral Abstract Session 233 — continuedCancer – Immunotherapy T <strong>Cell</strong> Studies2:00 PM - 4:00 PMRoom: Thurgood Marshall W31 3:30 PMProlonged Regression <strong>of</strong> Lymphoma and Elimination <strong>of</strong> CD19 + <strong>Cell</strong>sin a Patient That Received Anti-CD19 Chimeric Antigen Receptor-Transduced T <strong>Cell</strong>sJ. N. Kochenderfer, M. E. Dudley, R. A. Morgan, I. Maric, M. Raffeld, M. Stetler-Stevenson, J. E. Janik, W. H. Wilson, C. M. Yuan, S. A. Rosenberg.32 3:45 PMMarked Regression <strong>of</strong> Adenopathy Following Infusion <strong>of</strong> AutologousT <strong>Cell</strong>s <strong>Gene</strong>tically Targeted to the CD19 Antigen in a Patient withBulky CLLRenier J. Brentjens, Isabelle Riviere, Mark Frattini, Xiuyan Wang, Clare Taylor, MalgorzataOlszewska, Oriana Borquez-Ojeda, Shirley Bartido, Jolanta Stefanski, Raymond Yeh,Michel Sadelain.37 3:00 PMInsect <strong>Cell</strong> Carriers for Systemic Delivery <strong>of</strong> Oncolytic VirusesDominic G. Roy, Anthony T. Power, Fabrice Le Boeuf, John C. Bell.38 3:15 PMClinical Translational Development <strong>of</strong> rVSV(MD51)-M3:Biodistribution and Pharm/Tox Studies in Rats and MonkeysMarcia Meseck, Nelson Humala, Boxun Xie, Swan Thung, Susan Morgello, GreggGoldschlager, Savio Woo.39 3:30 PMA High-Throughput Pharmaco-Viral Approach Identifies OncolyticNovel Virus SensitizersJean-Simon Diallo, Fabrice Le Boeuf, Frances Lai, Julie Cox, Markus Vaha-Koskela,Hesham Abdelbary, Heather MacTavish, Katherine Waite, Theresa Falls, Jenny Wang,Ryan Brown, Jan E. Blanchard, Eric D. Brown, David H. Kirn, John Hiscott, Harry Atkins,Brian D. Lichty, John C. Bell.40 3:45 PMIdentification <strong>of</strong> Adenovirus Modulator <strong>Gene</strong>s in Human Cancer <strong>Cell</strong>sby Whole Genome RNAi Library ScreeningChristie Vermeulen, Nikki Tol, Ida van der Meulen-Muileman, Winald R. Gerritsen, VictorW. van Beusechem.Oral Abstract Session 234Cancer-Oncolytic Viruses I2:00 PM - 4:00 PMRoom: Marriott Ballroom Salon 133 2:00 PMDemonstration <strong>of</strong> Delivery and Antitumoral Activity <strong>of</strong> JX-594, aTargeted Multi-Mechanistic Oncolytic Poxvirus, Following a SingleIntravenous Infusion in Patients with Refractory Metastatic CancersJames Burke, Joe Stephenson, Laura Chow, Derek Jonker, Andrew Haas, Jorge Nieva,Kara Sabourin, Adina Pelusio, Caroline Breitbach, Taeho Hwang, Anne Moon, KelleyParato, Manijeh Daneshmand, John C. Bell, David H. Kirn.34 2:15 PMHighly Attenuated Vaccinia Virus with microRNA-Regulated Oncolysisfor Cancer VirotherapyMina Hikichi, Minoru Kidokoro, Hisatoshi Shida, Hideaki Tahara, Takafumi Nakamura.35 2:30 PMA Phase 1 Trial <strong>of</strong> Intravesical (IVE) CG0070 in Patients withSuperficial Bladder Cancer after BCG FailureJames M. Burke, Don Lamm, James Mckiernan, John Nemunaitis, Joe Stephenson, MaxMeng, James Arsenau, Junko Aimi, Daniel Maslyar.36 2:45 PMA Phase 1 Trial <strong>of</strong> the Novel Infectivity-Enhanced BicistronicAdenovirus Ad5.SSTR/TK.RGD in Recurrent Gynecologic CancerKenneth H. Kim, Kellie Schneider, Minghui Wang, Janis O’Malley, Wen Wan, MeredithA. Preuss, Raymond D. Harris, Rosemarie Aurigemma, <strong>Gene</strong> P. Siegal, Kurt Zinn, DavidT. Curiel, Ronald D. Alvarez.Oral Abstract Session 235Immunologic & Host Responses in <strong>Gene</strong> & <strong>Cell</strong> <strong>Therapy</strong>2:00 PM - 4:00 PMRoom: Virginia Suite41 2:00 PMRepeated Autologous Infusions <strong>of</strong> <strong>Cell</strong>s Modified with VSV-G-Pseudotyped Lentiviral Vector VRX496 Can Induce Anti-VSV-GHumoral Response in Humans without Clinical Consequence or Effecton Modified <strong>Cell</strong>s Persistence: Findings from Lexgenleucel-T Phase 2Clinical TrialFranck Lemiale, Lindsay Shovlin, Kris Andre, Gerard McGarrity, Tessio Rebello, LaurentHumeau.42 2:15 PMIntensive Pharmacological Immunosuppression Allows for RepetitiveLiver <strong>Gene</strong> Transfer with Recombinant Adenovirus in NonhumanPrimatesAntonio Fontanellas, Sandra Hervás-Stubbs, Itsaso Mauleón, Juan Dubrot, UxuaMancheño, María Collantes, Ana Sampedro, Carmen Unzu, Carlos Alfaro, Asis Palazón,Rafael Enríquez de Salamanca, Cristian Smerdou, Alberto Benito, Jesús Prieto, IvánPeñuelas, Ignacio Melero.43 2:30 PMInduction <strong>of</strong> Regulatory T <strong>Cell</strong>s as an Approach To Modulate CTLResponses Against AAV Capsid EpitopesDaniel J. Hui, Gary Pien, Etiena Basner-Tschakarjan, Federico Mingozzi, Jonathan D.Finn, Shangzhen Zhou, William Martin, Anne S. De Groot, Katherine A. High.EXHIBITOR PROSPECTUSfinal program
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