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1) Strand and Cell Type-specific Function of microRNA-126 in Angiogenesis (Molecular Therapy) Tuesday June 21st 2016
Qinbo Zhou, Chastain Anderson, Jakub Hanus, Fangkun Zhao, Jing Ma, Akihiko Yoshimura, Shusheng Wang

2) Mesenchymal Stem Cells Deliver Exogenous MicroRNA-let7c via Exosomes to Attenuate Renal Fibrosis (Molecular Therapy) Tuesday June 21st 2016
Bo Wang, Kevin Yao, Brooke M Huuskes, Hsin-Hui Shen, Junli Zhuang, Catherine Godson, Eoin P Brennan, Jennifer L Wilkinson-Berka, Andrea F Wise, Sharon D Ricardo

3) Complete Spectrum of CRISPR/Cas9-induced Mutations on HBV cccDNA (Molecular Therapy) Tuesday June 21st 2016
Christoph Seeger, Ji A Sohn

4) Translational Implications for Off-the-shelf Immune Cells Expressing Chimeric Antigen Receptors (Molecular Therapy) Tuesday June 14th 2016
Hiroki Torikai, Laurence JN Cooper

5) Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency (Molecular Therapy) Tuesday June 14th 2016
Maria Garcia-Gomez, Andrea Calabria, Maria Garcia-Bravo, Fabrizio Benedicenti, Penelope Kosinski, Sergio L, Collin Hill, Mar, Miguel A Mart, Israel Orman, Joan-LLuis Vives-Corrons, Charles Kung, Axel Schambach, Shengfang Jin, Juan A Bueren, Eugenio Montini, Susana Navarro, Jose C Segovia

6) Enhanced ADCC and NK Cell Activation of an Anticarcinoma Bispecific Antibody by Genetic Insertion of a Modified IL-15 Cross-linker (Molecular Therapy) Tuesday June 14th 2016
Joerg U Schmohl, Martin Felices, Elizabeth Taras, Jeff S Miller, Daniel A Vallera

7) Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform (Molecular Therapy) Tuesday June 14th 2016
Juwita H, Shahabuddin S Hoseini, Julia D Suerth, Dirk Hoffmann, Marcel Maluski, Jessica Herbst, Holger Maul, Arnab Ghosh, Britta Eiz-Vesper, Qinggong Yuan, Michael Ott, Michael Heuser, Axel Schambach, Martin G Sauer

8) In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy (Molecular Therapy) Tuesday June 7th 2016
Sourav R Choudhury, Zachary Fitzpatrick, Anne F Harris, Stacy A Maitland, Jennifer S Ferreira, Yuanfan Zhang, Shan Ma, Rohit B Sharma, Heather L Gray-Edwards, Jacob A Johnson, Aime K Johnson, Laura C Alonso, Claudio Punzo, Kathryn R Wagner, Casey A Maguire, Robert M Kotin, Douglas R Martin, Miguel Sena-Esteves

9) Rapid and Efficient Stable Gene Transfer to Mesenchymal Stromal Cells Using a Modified Foamy Virus Vector (Molecular Therapy) Tuesday June 7th 2016
Nathan Paul Sweeney, Cathy Regan, Jiahui Liu, Antonio Galleu, Francesco Dazzi, Dirk Lindemann, Charles Anthony Rupar, Myra Olga McClure

10) Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy (Molecular Therapy) Tuesday June 7th 2016
Olivier Bornert, Tobias K, Jeroen Bremer, Peter C van den Akker, Anna MG Pasmooij, Alexander Nystr

11) Development of Third-generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-cells (Molecular Therapy) Tuesday May 24th 2016
Olivier Humbert, Don W. Gisch, Martin E. Wohlfahrt, Amie B. Adams, Phil D. Greenberg, Tom M. Schmitt, Grant D. Trobridge, Hans-Peter Kiem

12) Development of Novel Small Hairpin RNAs That do not Require Processing by Dicer or AGO2 (Molecular Therapy) Tuesday May 24th 2016
Shin-ichiro Ohno, Karen Itano, Yuichirou Harada, Koutaro Asada, Keiki Oikawa, Mikie Kashiwazako, Hikaru Okuyama, Katsuyoshi Kumagai, Masakatsu Takanashi, Katsuko Sudo, Norihiko Ikeda, Masahiko Kuroda

13) Vastatin, an Endogenous Antiangiogenesis Polypeptide That Is Lost in Hepatocellular Carcinoma, Effectively Inhibits Tumor Metastasis (Molecular Therapy) Tuesday May 17th 2016
Zan Shen, Chen Yao, Zifeng Wang, Lu Yue, Zheping Fang, Hong Yao, Feng Lin, Hui Zhao, Yuan-Jue Sun, Xiu-wu Bian, Wenqi Jiang, Xiaomei Wang, Yi Li, Gang Lu, Wai Sang Poon, Hsiang-Fu Kung, Marie Chia-mi Lin

14) Engineering Periodic shRNA for Enhanced Silencing Efficacy (Molecular Therapy) Tuesday May 10th 2016
Connie Wu, Kevin E Shopsowitz, Paula T Hammond

15) CRNDE Promotes Malignant Progression of Glioma by Attenuating miR-384/PIWIL4/STAT3 Axis (Molecular Therapy) Tuesday May 10th 2016
Jian Zheng, Xiaobai Liu, Ping Wang, Yixue Xue, Jun Ma, Chengbin Qu, Yunhui Liu

16) Allospecific Tregs Expanded After Anergization Remain Suppressive in Inflammatory Conditions but Lack Expression of Gut-homing Molecules (Molecular Therapy) Tuesday May 3rd 2016
Eleni Kotsiou, John G Gribben, Jeff K Davies

17) Randomized Phase 2 Trial of the Oncolytic Virus Pelareorep (Reolysin) in Upfront Treatment of Metastatic Pancreatic Adenocarcinoma (Molecular Therapy) Tuesday May 3rd 2016
Anne M Noonan, Matthew R Farren, Susan M Geyer, Ying Huang, Sanaa Tahiri, Daniel Ahn, Sameh Mikhail, Kristen K Ciombor, Shubham Pant, Santiago Aparo, Jennifer Sexton, John L Marshall, Thomas A Mace, Christina S Wu, Bassel El-Rayes, Cynthia D Timmers, James Zwiebel, Gregory B Lesinski, Miguel A Villalona-Calero, Tanios S Bekaii-Saab

18) Predictive and Prognostic Clinical Variables in Cancer Patients Treated With Adenoviral Oncolytic Immunotherapy (Molecular Therapy) Tuesday May 3rd 2016
Kristian Taipale, Ilkka Liikanen, Anniina Koski, Raita Heiskanen, Anna Kanerva, Otto Hemminki, Minna Oksanen, Susanna Gr, Kari Hemminki, Timo Joensuu, Akseli Hemminki

19) rAAV Gene Therapy in a Canavan (Molecular Therapy) Tuesday May 3rd 2016
Seemin Seher Ahmed, Stefan A Schattgen, Ashley E Frakes, Elif M Sikoglu, Qin Su, Jia Li, Thomas G Hampton, Andrew R Denninger, Daniel A Kirschner, Brian Kaspar, Reuben Matalon, Guangping Gao

20) Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries (Molecular Therapy) Tuesday April 26th 2016
Jakob K, Timo Sieber, Stefan Michelfelder, Lars Lunding, Elmar Spies, Agnes Hunger, Malik Alawi, Kleopatra Rapti, Daniela Indenbirken, Oliver J M, Renata Pasqualini, Wadih Arap, J, Martin Trepel

21) Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid (Molecular Therapy) Tuesday April 19th 2016
Koen Vercauteren, Brad E Hoffman, Irene Zolotukhin, Geoffrey D Keeler, Jing W Xiao, Etiena Basner-Tschakarjan, Katherine A High, Hildegund CJ Ertl, Charles M Rice, Arun Srivastava, Ype P de Jong, Roland W Herzog

22) GD2-specific CAR T Cells Undergo Potent Activation and Deletion Following Antigen Encounter but can be Protected From Activation-induced Cell Death by PD-1 Blockade (Molecular Therapy) Tuesday April 19th 2016
Tessa Gargett, Wenbo Yu, Gianpietro Dotti, Eric S Yvon, Susan N Christo, John D Hayball, Ian D Lewis, Malcolm K Brenner, Michael P Brown

23) Protein Kinase C- (Molecular Therapy) Tuesday April 12th 2016
Daniela Castanotto, Min Lin, Claudia Kowolik, Troels Koch, Bo Rode Hansen, Henrik Oerum, Cy A Stein

24) Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes (Molecular Therapy) Tuesday April 12th 2016
Sheng Zhou, Soghra Fatima, Zhijun Ma, Yong-Dong Wang, Taihe Lu, Laura J Janke, Yang Du, Brian P Sorrentino

25) Targeted Delivery of C/EBP (Molecular Therapy) Tuesday April 12th 2016
Sorah Yoon, Kai-Wen Huang, Vikash Reebye, Paul Mintz, Yu-Wen Tien, Hong-Shiee Lai, P, Isabella Reccia, Piotr Swiderski, Brian Armstrong, Agnieszka Jozwiak, Duncan Spalding, Long Jiao, Nagy Habib, John J Rossi

26) A Role for the Long Noncoding RNA SENCR in Commitment and Function of Endothelial Cells (Molecular Therapy) Tuesday April 5th 2016
Mounia Boulberdaa, Elizabeth Scott, Margaret Ballantyne, Raquel Garcia, Betty Descamps, Gianni D Angelini, Mairi Brittan, Amanda Hunter, Martin McBride, John McClure, Joseph M Miano, Costanza Emanueli, Nicholas L Mills, Joanne C Mountford, Andrew H Baker

27) Stable, Nonviral Expression of Mutated Tumor Neoantigen-specific T-cell Receptors Using the Sleeping Beauty Transposon/Transposase System (Molecular Therapy) Tuesday April 5th 2016
Drew C Deniger, Anna Pasetto, Eric Tran, Maria R Parkhurst, Cyrille J Cohen, Paul F Robbins, Laurence JN Cooper, Steven A Rosenberg

28) Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients (Molecular Therapy) Tuesday April 5th 2016
Irene Gil-Farina, Raffaele Fronza, Christine Kaeppel, Esperanza Lopez-Franco, Valerie Ferreira, Delia D, Alberto Benito, Jesus Prieto, Harald Petry, Gloria Gonzalez-Aseguinolaza, Manfred Schmidt

29) CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease (Molecular Therapy) Tuesday April 5th 2016
John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard, Seng H Cheng

30) Inhibiting Notch Activity in Breast Cancer Stem Cells by Glucose Functionalized Nanoparticles Carrying (Molecular Therapy) Tuesday March 29th 2016
Veronika Mamaeva, Rasmus Niemi, Michaela Beck, Ezgi, Diti Desai, Sebastian Landor, Tove Gronroos, Pauliina Kronqvist, Ina K N Pettersen, Emmet McCormack, Jessica M Rosenholm, Mika Linden, Cecilia Sahlgren

31) Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics (Molecular Therapy) Tuesday March 22nd 2016
Masayuki Matsui, Thazha P Prakash, David R Corey

32) Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington (Molecular Therapy) Wednesday March 16th 2016
Kari Pollock, Heather Dahlenburg, Haley Nelson, Kyle D Fink, Whitney Cary, Kyle Hendrix, Geralyn Annett, Audrey Torrest, Peter Deng, Joshua Gutierrez, Catherine Nacey, Karen Pepper, Stefanos Kalomoiris, Johnathon D Anderson, Jeannine McGee, William Gruenloh, Brian Fury, Gerhard Bauer, Alexandria Duffy, Theresa Tempkin, Vicki Wheelock, Jan A Nolta

33) Gene Transfer of Brain-derived Neurotrophic Factor (BDNF) Prevents Neurodegeneration Triggered by FXN Deficiency (Molecular Therapy) Wednesday March 16th 2016
Yurika Katsu-Jim, Frida Lor, Juan Carlos Corona, Javier D

34) Chop Deficiency Protects Mice Against Bleomycin-induced Pulmonary Fibrosis by Attenuating M2 Macrophage Production (Molecular Therapy) Wednesday March 16th 2016
Yingying Yao, Yi Wang, Zhijun Zhang, Long He, Jianghui Zhu, Meng Zhang, Xiaoyu He, Zhenshun Cheng, Qilin Ao, Yong Cao, Ping Yang, Yunchao Su, Jianping Zhao, Shu Zhang, Qilin Yu, Qin Ning, Xudong Xiang, Weining Xiong, Cong-Yi Wang, Yongjian Xu

35) Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy (Molecular Therapy) Wednesday March 16th 2016
Soeren Turan, Alfonso P Farruggio, Waracharee Srifa, John W Day, Michele P Calos

36) Progress With Developing Use of Gene Editing To Cure Chronic Infection With Hepatitis B Virus (Molecular Therapy) Wednesday March 16th 2016
Abdullah Ely, Buhle Moyo, Patrick Arbuthnot

37) Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent (Molecular Therapy) Tuesday March 8th 2016
Monir Shababi, Zhihua Feng, Eric Villalon, Christine M Sibigtroth, Erkan Y Osman, Madeline R Miller, Patricka A Williams-Simon, Abby Lombardi, Thalia H Sass, Arleigh K Atkinson, Michael L Garcia, Chien-Ping Ko, Christian L Lorson

38) In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA (Molecular Therapy) Tuesday March 8th 2016
Dustin J Landau, Elizabeth Drake Brooks, Pablo Perez-Pinera, Hiruni Amarasekara, Adam Mefferd, Songtao Li, Andrew Bird, Charles A Gersbach, Dwight D Koeberl

39) Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy (Molecular Therapy) Tuesday March 8th 2016
Eirini P Papapetrou, Axel Schambach

40) Human Mesenchymal Stem Cell Delivery System Modulates Ischemic Cardiac Remodeling With an Increase of Coronary Artery Blood Flow (Molecular Therapy) Tuesday March 1st 2016
Young Sook Lee, Wan Seok Joo, Hyun Soo Kim, Sung Wan Kim

41) H7N9 Live Attenuated Influenza Vaccine Is Highly Immunogenic, Prevents Virus Replication, and Protects Against Severe Bronchopneumonia in Ferrets (Molecular Therapy) Tuesday March 1st 2016
J, Irina Isakova-Sivak, Harry van Dijken, Sanne Spijkers, Justin Mouthaan, Rineke de Jong, Tatiana Smolonogina, Paul Roholl, Larisa Rudenko

42) Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector (Molecular Therapy) Tuesday March 1st 2016
Wei Huang, Travis McMurphy, Xianglan Liu, Chuansong Wang, Lei Cao

43) AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6 (Molecular Therapy) Tuesday March 1st 2016
Virginie Pichard, Nathalie Provost, Alexandra Mendes-Madeira, Lyse Libeau, Philippe Hulin, Kizito-Tshitoko Tshilenge, Marine Biget, Baptiste Ameline, Jack-Yves Deschamps, Michel Weber, Guyl, Marie-Anne Colle, Philippe Moullier, Fabienne Rolling

44) Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing (Molecular Therapy) Tuesday February 16th 2016
Ciaran M Lee, Thomas J Cradick, Eli J Fine, Gang Bao

45) Genome-editing Technologies for Gene and Cell Therapy (Molecular Therapy) Tuesday February 16th 2016
Morgan L Maeder, Charles A Gersbach

46) Salient Features of Endonuclease Platforms for Therapeutic Genome Editing (Molecular Therapy) Tuesday February 16th 2016
Michael T Certo, Richard A Morgan

47) CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus Escape (Molecular Therapy) Tuesday February 16th 2016
Gang Wang, Na Zhao, Ben Berkhout, Atze T Das

48) Induced Apoptosis Investigation in Wild-type and FLT3-ITD Acute Myeloid Leukemia Cells by Nanochannel Electroporation and Single-cell qRT-PCR (Molecular Therapy) Tuesday February 16th 2016
Keliang Gao, Xiaomeng Huang, Chi-Ling Chiang, Xinmei Wang, Lingqian Chang, Pouyan Boukany, Guido Marcucci, Robert Lee, Ly James Lee

49) Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 Promoter (Molecular Therapy) Tuesday February 16th 2016
Haiyan Ji, Zhengtao Jiang, Panpan Lu, Li Ma, Chuan Li, Hanyu Pan, Zheng Fu, Xiying Qu, Pengfei Wang, Junxiao Deng, Xinyi Yang, Jianhua Wang, Huanzhang Zhu

50) The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells (Molecular Therapy) Tuesday February 16th 2016
Ciaran M Lee, Thomas J Cradick, Gang Bao

51) PTH Induces Systemically Administered Mesenchymal Stem Cells to Migrate to and Regenerate Spine Injuries (Molecular Therapy) Tuesday February 9th 2016
Dmitriy Sheyn, Galina Shapiro, Wafa Tawackoli, Douk Soo Jun, Youngdo Koh, Kyu Bok Kang, Susan Su, Xiaoyu Da, Shiran Ben-David, Maxim Bez, Eran Yalon, Ben Antebi, Pablo Avalos, Tomer Stern, Elazar Zelzer, Edward M Schwarz, Zulma Gazit, Gadi Pelled, Hyun M Bae, Dan Gazit

52) Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I (Molecular Therapy) Tuesday February 9th 2016
Cristina Martin-Higueras, Sergio Luis-Lima, Eduardo Salido

53) Actin-resistant DNAse I Expression From Oncolytic Adenovirus Enadenotucirev Enhances Its Intratumoral Spread and Reduces Tumor Growth (Molecular Therapy) Tuesday February 9th 2016
Alison Tedcastle, Sam Illingworth, Alice Brown, Leonard W Seymour, Kerry D Fisher

54) Smooth Muscle Cell (Molecular Therapy) Tuesday February 9th 2016
William H Thiel, Carla L Esposito, David D Dickey, Justin P Dassie, Matthew E Long, Joshua Adam, Jennifer Streeter, Brandon Schickling, Maysam Takapoo, Katie S Flenker, Julia Klesney-Tait, Vittorio de Franciscis, Francis J Miller, Paloma H Giangrande

55) Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T Cells (Molecular Therapy) Tuesday February 9th 2016
Andreas Gogol-D, Ismahen Ammar, Saumyashree Gupta, Mario Bunse, Csaba Miskey, Wei Chen, Wolfgang Uckert, Thomas F Schulz, Zsuzsanna Izsv, Zolt

56) MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy (Molecular Therapy) Tuesday February 9th 2016
Naemeh Pourshafie, Philip R Lee, Ke-lian Chen, George G Harmison, Laura C Bott, Masahisa Katsuno, Gen Sobue, Barrington G Burnett, Kenneth H Fischbeck, Carlo Rinaldi

57) Development and Validation of a Small Single-domain Antibody That Effectively Inhibits Matrix Metalloproteinase 8 (Molecular Therapy) Tuesday February 9th 2016
Delphine Demeestere, Eline Dejonckheere, Sophie Steeland, Paco Hulpiau, Jurgen Haustraete, Nick Devoogdt, Rielana Wichert, Christoph Becker-Pauly, Elien Van Wonterghem, Sylviane Dewaele, Griet Van Imschoot, Jeroen Aerts, Lutgarde Arckens, Yvan Saeys, Claude Libert, Roosmarijn E Vandenbroucke

58) Novel Conserved-region T-cell Mosaic Vaccine With High Global HIV-1 Coverage Is Recognized by Protective Responses in Untreated Infection (Molecular Therapy) Tuesday February 9th 2016
Beatrice Ondondo, Hayato Murakoshi, Genevieve Clutton, Sultan Abdul-Jawad, Edmund G-T Wee, Hiroyuki Gatanaga, Shinichi Oka, Andrew J McMichael, Masafumi Takiguchi, Bette Korber, Tom

59) Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1 (Molecular Therapy) Tuesday February 9th 2016
Chaitali Dutta, Nicole Avitahl-Curtis, Natalie Pursell, Marita Larsson Cohen, Benjamin Holmes, Rohan Diwanji, Wei Zhou, Luciano Apponi, Martin Koser, Bo Ying, Dongyu Chen, Xue Shui, Utsav Saxena, Wendy A Cyr, Anee Shah, Naim Nazef, Weimin Wang, Marc Abrams, Henryk Dudek, Eduardo Salido, Bob D Brown, Chengjung Lai

60) Current Progress in Therapeutic Gene Editing for Monogenic Diseases (Molecular Therapy) Tuesday February 9th 2016
Versha Prakash, Marc Moore, Rafael J Y

61) SSRP1 Contributes to the Malignancy of Hepatocellular Carcinoma and Is Negatively Regulated by miR-497 (Molecular Therapy) Tuesday February 9th 2016
Qianshan Ding, Ke He, Tao Luo, Yunchao Deng, Hanning Wang, Hao Liu, Jinqian Zhang, Kaiyun Chen, Jinfeng Xiao, Xiaopeng Duan, Rui Huang, Zhenglin Xia, Wenjie Zhou, Jinliang He, Honggang Yu, Xingyuan Jiao, Guoan Xiang

62) Modulation of Protein (Molecular Therapy) Tuesday February 2nd 2016
Ioanna Petta, Sam Lievens, Claude Libert, Jan Tavernier, Karolien De Bosscher

63) Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human Cells (Molecular Therapy) Tuesday February 2nd 2016
Jaichandran Sivalingam, Dimitar Kenanov, Hao Han, Ajit Johnson Nirmal, Wai Har Ng, Sze Sing Lee, Jeyakumar Masilamani, Toan Thang Phan, Sebastian Maurer-Stroh, Oi Lian Kon

64) Serial Activation of the Inducible Caspase 9 Safety Switch After Human Stem Cell Transplantation (Molecular Therapy) Tuesday February 2nd 2016
Xiaoou Zhou, Swati Naik, Olga Dakhova, Gianpietro Dotti, Helen E. Heslop, Malcolm K. Brenner

65) Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia (Molecular Therapy) Tuesday January 26th 2016
Tytteli A K Turunen, Jere Kurkipuro, Tommi Heikura, Taina Vuorio, Elisa Hyt, Zsuzsanna Izsv, Seppo Yl

66) Relationship Between the Physicochemical Properties of Lipid Nanoparticles and the Quality of siRNA Delivery to Liver Cells (Molecular Therapy) Tuesday January 26th 2016
Yusuke Sato, Hiroto Hatakeyama, Mamoru Hyodo, Hideyoshi Harashima

67) Re-expression of Selected Epigenetically Silenced Candidate Tumor Suppressor Genes in Cervical Cancer by TET2-directed Demethylation (Molecular Therapy) Tuesday January 26th 2016
Christian Huisman, Monique G P van der Wijst, Matthijs Schokker, Pilar Blancafort, Martijn M Terpstra, Klaas Kok, Ate GJ van der Zee, Ed Schuuring, G Bea A Wisman, Marianne G Rots

68) Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector (Molecular Therapy) Tuesday January 26th 2016
Sourav R Choudhury, Anne F Harris, Damien J Cabral, Allison M Keeler, Ellen Sapp, Jennifer S Ferreira, Heather L Gray-Edwards, Jacob A Johnson, Aime K Johnson, Qin Su, Lorelei Stoica, Marian DiFiglia, Neil Aronin, Douglas R Martin, Guangping Gao, Miguel Sena-Esteves

69) Development of Nonviral Vectors Targeting the Brain as a Therapeutic Approach For Parkinson (Molecular Therapy) Tuesday January 26th 2016
Hayate Javed, Sindhu A Menon, Karima M Al-Mansoori, Abdelmojib Al-Wandi, Nour K Majbour, Mustafa T Ardah, Shiji Varghese, Nishant N Vaikath, M Emdadul Haque, Mimoun Azzouz, Omar MA El-Agnaf

70) SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically (Molecular Therapy) Tuesday January 19th 2016
Tianming Li, Qidi Zheng, Jiahui An, Mengying Wu, Haiyan Li, Xin Gui, Hu Pu, Dongdong Lu

71) Genetic Immunization With In Vivo Dendritic Cell-targeting Liposomal DNA Vaccine Carrier Induces Long-lasting Antitumor Immune Response (Molecular Therapy) Tuesday January 19th 2016
Arup Garu, Gopikrishna Moku, Suresh Kumar Gulla, Arabinda Chaudhuri

72) In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa (Molecular Therapy) Tuesday January 19th 2016
Benjamin Bakondi, Wenjian Lv, Bin Lu, Melissa K Jones, Yuchun Tsai, Kevin J Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J Breunig, Clive N Svendsen, Shaomei Wang

73) Combination wt-p53 and MicroRNA-125b Transfection in a Genetically Engineered Lung Cancer Model Using Dual CD44/EGFR-targeting Nanoparticles (Molecular Therapy) Tuesday January 19th 2016
Meghna Talekar, Malav Trivedi, Parin Shah, Qijun Ouyang, Adwait Oka, Srujan Gandham, Mansoor M Amiji

74) Development of an Interleukin-1 (Molecular Therapy) Tuesday January 19th 2016
Claudia Cavelti-Weder, Katharina Timper, Eleonora Seelig, Cornelia Keller, Martin Osranek, Ute L, Gunther Spohn, Patrik Maurer, Philipp M, Gary T Jennings, Joerg Willers, Philippe Saudan, Marc Y Donath, Martin F Bachmann

75) Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome (Molecular Therapy) Tuesday January 12th 2016
Maximilian M, Ciaran M Lee, Giedrius Gasiunas, Timothy H Davis, Thomas J Cradick, Virginijus Siksnys, Gang Bao, Toni Cathomen, Claudio Mussolino

76) CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice (Molecular Therapy) Tuesday January 5th 2016
Li Xu, Ki Ho Park, Lixia Zhao, Jing Xu, Mona El Refaey, Yandi Gao, Hua Zhu, Jianjie Ma, Renzhi Han

77) Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases (Molecular Therapy) Tuesday January 5th 2016
Mark J Osborn, Beau R Webber, Friederike Knipping, Cara-lin Lonetree, Nicole Tennis, Anthony P DeFeo, Amber N McElroy, Colby G Starker, Catherine Lee, Sarah Merkel, Troy C Lund, Karen S Kelly-Spratt, Michael C Jensen, Daniel F Voytas, Christof von Kalle, Manfred Schmidt, Richard Gabriel, Keli L Hippen, Jeffrey S Miller, Andrew M Scharenberg, Jakub Tolar, Bruce R Blazar

78) Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator Complex (Molecular Therapy) Tuesday January 5th 2016
Sheena M Saayman, Daniel C Lazar, Tristan A Scott, Jonathan R Hart, Mayumi Takahashi, John C Burnett, Vicente Planelles, Kevin V Morris, Marc S Weinberg

79) Increased Valency of Conserved-mosaic Vaccines Enhances the Breadth and Depth of Epitope Recognition (Molecular Therapy) Tuesday January 5th 2016
Sultan Abdul-Jawad, Beatrice Ondondo, Andy van Hateren, Andrew Gardner, Tim Elliott, Bette Korber, Tom

80) A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs (Molecular Therapy) Tuesday January 5th 2016
Nicolina Cristina Sorrentino, Veronica Maffia, Sandra Strollo, Vincenzo Cacace, Noemi Romagnoli, Anna Manfredi, Domenico Ventrella, Francesco Dondi, Francesca Barone, Massimo Giunti, Anne-Renee Graham, Yan Huang, Susan L Kalled, Alberto Auricchio, Maria Laura Bacci, Enrico Maria Surace, Alessandro Fraldi

81) CRISPR-mediated Activation of Latent HIV-1 Expression (Molecular Therapy) Tuesday January 5th 2016
Prajit Limsirichai, Thomas Gaj, David V Schaffer

82) Variable Virulence and Efficacy of BCG Vaccine Strains in Mice and Correlation With Genome Polymorphisms (Molecular Therapy) Tuesday January 5th 2016
Lu Zhang, Huan-wei Ru, Fu-zeng Chen, Chun-yan Jin, Rui-feng Sun, Xiao-yong Fan, Ming Guo, Jun-tao Mai, Wen-xi Xu, Qing-xia Lin, Jun Liu

83) Clonal Dynamics In Vivo of Virus Integration Sites of T Cells Expressing a Safety Switch (Molecular Therapy) Tuesday January 5th 2016
Edmund C Chang, Hao Liu, John A West, Xiaoou Zhou, Olga Dakhova, David A Wheeler, Helen E Heslop, Malcolm K Brenner, Gianpietro Dotti

84) Perfusion Pressure Is a Critical Determinant of the Intratumoral Extravasation of Oncolytic Viruses (Molecular Therapy) Tuesday January 5th 2016
Amber Miller, Rebecca Nace, Camilo Ayala-Breton C, Michael Steele, Kent Bailey, Kah Whye Peng, Stephen J Russell

85) A Recombinant Bispecific CD20 (Molecular Therapy) Tuesday December 15th 2015
Kristina Nalivaiko, Martin Hofmann, Karina Kober, Nadine Teichweyde, Peter H Krammer, Hans-Georg Rammensee, Ludger Grosse-Hovest, Gundram Jung

86) Absence of WASp Enhances Hematopoietic and Megakaryocytic Differentiation in a Human Embryonic Stem Cell Model (Molecular Therapy) Tuesday December 8th 2015
Miguel G Toscano, Pilar Mu, Almudena S, Mari, Karim Benabdellah, Per Anderson, Ver, Pedro J Real, Olaf Neth, Agueda Molinos-Quintana, Philip D Gregory, Michael C Holmes, Francisco Martin

87) An RNA Molecule Derived From Sendai Virus DI Particles Induces Antitumor Immunity and Cancer Cell-selective Apoptosis (Molecular Therapy) Tuesday December 8th 2015
Li-Wen Liu, Tomoyuki Nishikawa, Yasufumi Kaneda

88) AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility (Molecular Therapy) Tuesday December 1st 2015
Stephen C Kolwicz, Guy L Odom, Sarah G Nowakowski, Farid Moussavi-Harami, Xiaolan Chen, Hans Reinecke, Stephen D Hauschka, Charles E Murry, Gregory G Mahairas, Michael Regnier

89) Myocardial Delivery of Lipidoid Nanoparticle Carrying modRNA Induces Rapid and Transient Expression (Molecular Therapy) Tuesday December 1st 2015
Irene C Turnbull, Ahmed A Eltoukhy, Kenneth M Fish, Mathieu Nonnenmacher, Kiyotake Ishikawa, Jiqiu Chen, Roger J Hajjar, Daniel G Anderson, Kevin D Costa

90) Characterization and Functional Analysis of scFv-based Chimeric Antigen Receptors to Redirect T Cells to IL13R (Molecular Therapy) Tuesday December 1st 2015
Giedre Krenciute, Simone Krebs, David Torres, Meng-Fen Wu, Hao Liu, Gianpietro Dotti, Xiao-Nan Li, Maciej S Lesniak, Irina V Balyasnikova, Stephen Gottschalk

91) CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy (Molecular Therapy) Tuesday December 1st 2015
Charis L Himeda, Takako I Jones, Peter L Jones

92) Development of Novel Adenoviral Vectors to Overcome Challenges Observed With HAdV-5 (Molecular Therapy) Tuesday November 24th 2015
Julio Alonso-Padilla, Tibor Papp, Gy, M, Menzo Havenga, Angelique Lemckert, Bal, Andrew H Baker

93) Virus-mediated EpoR76E Therapy Slows Optic Nerve Axonopathy in Experimental Glaucoma (Molecular Therapy) Tuesday November 24th 2015
Wesley S Bond, Jessica Hines-Beard, YPaul L GoldenMerry, Mara Davis, Alma Farooque, Rebecca M Sappington, David J Calkins, Tonia S Rex

94) Glucocorticoid Cell Priming Enhances Transfection Outcomes in Adult Human Mesenchymal Stem Cells (Molecular Therapy) Tuesday November 17th 2015
Abby M Kelly, Sarah A Plautz, Janos Zempleni, Angela K Pannier

95) Nanoparticle Delivered VEGF-A siRNA Enhances Photodynamic Therapy for Head and Neck Cancer Treatment (Molecular Therapy) Tuesday November 3rd 2015
Rumwald Leo G Lecaros, Leaf Huang, Tsai-Chia Lee, Yih-Chih Hsu

96) Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector (Molecular Therapy) Tuesday November 3rd 2015
Joshua C Grieger, Stephen M Soltys, Richard Jude Samulski

97) Grapefruit-derived Nanovectors Delivering Therapeutic miR17 Through an Intranasal Route Inhibit Brain Tumor Progression (Molecular Therapy) Tuesday November 3rd 2015
Xiaoying Zhuang, Yun Teng, Abhilash Samykutty, Jingyao Mu, Zhongbin Deng, Lifeng Zhang, Pengxiao Cao, Yuan Rong, Jun Yan, Donald Miller, Huang-Ge Zhang

98) Silent IL2RG Gene Editing in Human Pluripotent Stem Cells (Molecular Therapy) Tuesday November 3rd 2015
Li B Li, Chao Ma, Geneve Awong, Marion Kennedy, German Gornalusse, Gordon Keller, Dan S Kaufman, David W Russell

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Mitch Andre Garcia's Chem Feeds 2008-present

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