TABLE OF CONTENTS
|  |  |  | Volume 20, Issue 1 (January 2012) |  | In this issue
Editorial
In This Issue
Research Highlights
Letters to the Editor
Commentaries
Review
Original Articles
Corrigendum
Also new
   AOP
| |  |  |  | | Advertisement |  | |  | | | Editorial | Top |  | Mathematical Modeling in Immunotherapy of Cancer: Personalizing Clinical TrialsZvia Agur and Stanimir Vuk-Pavlović Mol Ther 2012 20: 1-2; 10.1038/mt.2011.272 Full Text |  | In This Issue | Top |  | In This IssueMol Ther 2012 20: 4; 10.1038/mt.2011.278 Full Text |  | Research Highlights | Top |  | Research HighlightsMol Ther 2012 20: 5; 10.1038/mt.2011.279 Full Text |  | Letters to the Editor | Top |  | Is Serum IL-17A a Useful Systemic Biomarker in Patients With Langerhans Cell Histiocytosis?Polyzois Makras, Stergios A Polyzos, Athanasios D Anastasilakis, Evangelos Terpos, Athanasios Papatheodorou and Gregory A Kaltsas Mol Ther 2012 20: 6-7; 10.1038/mt.2011.239 Full Text |  | Commentaries | Top |  | Antisense Oligonucleotides Shed New Light on the Pathogenesis and Treatment of Spinal Muscular AtrophyJiing-Kuan Yee and Ren-Jang Lin Mol Ther 2012 20: 8-10; 10.1038/mt.2011.275 Full Text |  |  |  | Multifunctional Theranostic Nanoparticles for Brain TumorsYuhua Wang and Leaf Huang Mol Ther 2012 20: 10-11; 10.1038/mt.2011.274 Full Text |  |  |  | Live and Let Die: A New Suicide Gene Therapy Moves to the ClinicRichard A Morgan Mol Ther 2012 20: 11-13; advance online publication, January 1, 2011; 10.1038/mt.2011.273 Full Text |  | Review | Top |  | Mesenchymal Stem/Stromal Cells (MSCs): Role as Guardians of InflammationDarwin J Prockop and Joo Youn Oh Mol Ther 2012 20: 14-20; advance online publication, October 18, 2011; 10.1038/mt.2011.211 Abstract | Full Text |  | Original Articles | Top |  | Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe MiceKai Qiu, Darin J Falk, Paul J Reier, Barry J Byrne and David D Fuller Mol Ther 2012 20: 21-27; advance online publication, October 18, 2011; 10.1038/mt.2011.214 Abstract | Full Text |  |  |  | ACE2 and Ang-(1-7) Confer Protection Against Development of Diabetic RetinopathyAmrisha Verma, Zhiying Shan, Bo Lei, Lihui Yuan, Xuan Liu, Takahiko Nakagawa, Maria B Grant, Alfred S Lewin, William W Hauswirth, Mohan K Raizada and Qiuhong Li Mol Ther 2012 20: 28-36; advance online publication, July 26, 2011; 10.1038/mt.2011.155 Abstract | Full Text |  |  |  | Bevacizumab With Angiostatin-armed oHSV Increases Antiangiogenesis and Decreases Bevacizumab-induced Invasion in U87 GliomaWei Zhang, Giulia Fulci, Jason S Buhrman, Anat O Stemmer-Rachamimov, John W Chen, Gregory R Wojtkiewicz, Ralph Weissleder, Samuel D Rabkin and Robert L Martuza Mol Ther 2012 20: 37-45; advance online publication, September 13, 2011; 10.1038/mt.2011.187 Abstract | Full Text |  |  |  | 2A Peptide-based, Lentivirus-mediated Anti-death Receptor 5 Chimeric Antibody Expression Prevents Tumor Growth in Nude MiceMeng Li, Yaomin Wu, Yuhe Qiu, Zhenyu Yao, Shilian Liu, Yanxin Liu, Juan Shi and Dexian Zheng Mol Ther 2012 20: 46-53; advance online publication, September 20, 2011; 10.1038/mt.2011.197 Abstract | Full Text |  |  |  | Adenovirus i-Leader Truncation Bioselected Against Cancer-associated Fibroblasts to Overcome Tumor Stromal BarriersCristina Puig-Saus, Alena Gros, Ramon Alemany and Manel Cascalló Mol Ther 2012 20: 54-62; advance online publication, August 23, 2011; 10.1038/mt.2011.159 Abstract | Full Text |  |  |  | Optimization of hCFTR Lung Expression in Mice Using DNA NanoparticlesLinas Padegimas, Tomasz H Kowalczyk, Sam Adams, Chris R Gedeon, Sharon M Oette, Karla Dines, Susannah L Hyatt, Ozge Sesenoglu-Laird, Olena Tyr, Robert C Moen and Mark J Cooper Mol Ther 2012 20: 63-72; advance online publication, September 27, 2011; 10.1038/mt.2011.196 Abstract | Full Text |  |  |  | Neutralizing Antibodies Against AAV Serotypes 1, 2, 6, and 9 in Sera of Commonly Used Animal ModelsKleopatra Rapti, Vedell Louis-Jeune, Erik Kohlbrenner, Kiyotake Ishikawa, Dennis Ladage, Sergei Zolotukhin, Roger J Hajjar and Thomas Weber Mol Ther 2012 20: 73-83; advance online publication, September 13, 2011; 10.1038/mt.2011.177 Abstract | Full Text |  |  |  | Evaluation of Residual Promoter Activity in γ-Retroviral Self-inactivating (SIN) VectorsWenqin Xu, Jill L Russ and Maribeth V Eiden Mol Ther 2012 20: 84-90; advance online publication, October 18, 2011; 10.1038/mt.2011.204 Abstract | Full Text |  |  |  | Delivery of siRNA to the Mouse Lung via a Functionalized LipopolyamineKevin J Polach, Majed Matar, Jennifer Rice, Gregory Slobodkin, Jeff Sparks, Richard Congo, Angela Rea-Ramsey, Diane McClure, Elaine Brunhoeber, Monika Krampert, Andrea Schuster, Kerstin Jahn-Hofmann, Matthias John, Hans-Peter Vornlocher, Jason G Fewell, Khursheed Anwer and Anke Geick Mol Ther 2012 20: 91-100; advance online publication, October 11, 2011; 10.1038/mt.2011.210 Abstract | Full Text |  |  |  | Short-interfering RNAs Induce Retinal Degeneration via TLR3 and IRF3Mark E Kleinman, Hiroki Kaneko, Won Gil Cho, Sami Dridi, Benjamin J Fowler, Alexander D Blandford, Romulo JC Albuquerque, Yoshio Hirano, Hiroko Terasaki, Mineo Kondo, Takashi Fujita, Balamurali K Ambati, Valeria Tarallo, Bradley D Gelfand, Sasha Bogdanovich, Judit Z Baffi and Jayakrishna Ambati Mol Ther 2012 20: 101-108; advance online publication, October 11, 2011; 10.1038/mt.2011.212 Abstract | Full Text |  |  |  | Targeted Gene Modification of Hematopoietic Progenitor Cells in Mice Following Systemic Administration of a PNA-peptide ConjugateFaye A Rogers, Sharon S Lin, Denise C Hegan, Diane S Krause and Peter M Glazer Mol Ther 2012 20: 109-118; advance online publication, August 9, 2011; 10.1038/mt.2011.163 Abstract | Full Text |  |  |  | Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular AtrophyErkan Y Osman, Pei-Fen Yen and Christian L Lorson Mol Ther 2012 20: 119-126; advance online publication, October 25, 2011; 10.1038/mt.2011.232 Abstract | Full Text |  |  |  | Efficient Generation of Functional Hepatocytes From Human Embryonic Stem Cells and Induced Pluripotent Stem Cells by HNF4α TransductionKazuo Takayama, Mitsuru Inamura, Kenji Kawabata, Kazufumi Katayama, Maiko Higuchi, Katsuhisa Tashiro, Aki Nonaka, Fuminori Sakurai, Takao Hayakawa, Miho Kusuda Furue and Hiroyuki Mizuguchi Mol Ther 2012 20: 127-137; advance online publication, November 8, 2011; 10.1038/mt.2011.234 Abstract | Full Text |  |  |  | Human Skeletal Muscle Cells With a Slow Adhesion Rate After Isolation and an Enhanced Stress Resistance Improve Function of Ischemic HeartsMasaho Okada, Thomas R Payne, Lauren Drowley, Ron J Jankowski, Nobuo Momoi, Sarah Beckman, William CW Chen, Bradley B Keller, Kimimasa Tobita and Johnny Huard Mol Ther 2012 20: 138-145; advance online publication, November 8, 2011; 10.1038/mt.2011.229 Abstract | Full Text |  |  |  | Slowing Down Differentiation of Engrafted Human Myoblasts Into Immunodeficient Mice Correlates With Increased Proliferation and MigrationIngo Riederer, Elisa Negroni, Maximilien Bencze, Annie Wolff, Ahmed Aamiri, James P Di Santo, Suse D. Silva-Barbosa, Gillian Butler-Browne, Wilson Savino and Vincent Mouly Mol Ther 2012 20: 146-154; advance online publication, September 20, 2011; 10.1038/mt.2011.193 Abstract | Full Text |  |  |  | N-cadherin Determines Individual Variations in the Therapeutic Efficacy of Human Umbilical Cord Blood-derived Mesenchymal Stem Cells in a Rat Model of Myocardial InfarctionEun Ju Lee, Eue-Keun Choi, Soo Kyoung Kang, Gi-Hwan Kim, Ju Young Park, Hyun-Jae Kang, Sae-Won Lee, Keum-Hyun Kim, Jin Sook Kwon, Ki Hong Lee, Youngkeun Ahn, Ho-Jae Lee, Hyun-Jai Cho, Soo Jin Choi, Won Il Oh, Young-Bae Park and Hyo-Soo Kim Mol Ther 2012 20: 155-167; advance online publication, November 8, 2011; 10.1038/mt.2011.202 Abstract | Full Text |  |  |  | Long-term Engraftment of Multipotent Mesenchymal Stromal Cells That Differentiate to Form Myogenic Cells in Dogs With Duchenne Muscular DystrophyYuko Nitahara-Kasahara, Hiromi Hayashita-Kinoh, Sachiko Ohshima-Hosoyama, Hironori Okada, Michiko Wada-Maeda, Akinori Nakamura, Takashi Okada and Shin'ichi Takeda Mol Ther 2012 20: 168-177; advance online publication, September 20, 2011; 10.1038/mt.2011.181 Abstract | Full Text |  |  |  | Aire Controls Mesenchymal Stem Cell-mediated Suppression in Chronic ColitisBiju Parekkadan, Anne L Fletcher, Matthew Li, Melissa Y Tjota, Angelique Bellemare-Pelletier, Jack M Milwid, Je-Wook Lee, Martin L Yarmush and Shannon J Turley Mol Ther 2012 20: 178-186; advance online publication, September 27, 2011; 10.1038/mt.2011.192 Abstract | Full Text |  |  |  | Human MSC Suppression Correlates With Cytokine Induction of Indoleamine 2,3-Dioxygenase and Bystander M2 Macrophage DifferentiationMoïra François, Raphaëlle Romieu-Mourez, Mengyang Li and Jacques Galipeau Mol Ther 2012 20: 187-195; advance online publication, September 20, 2011; 10.1038/mt.2011.189 Abstract | Full Text |  |  |  | Distinct Strategies Are Required to Suppress Antigen-specific Responses to Genetically Modified Keratinocytes and FibroblastsSoosan Ghazizadeh, Li T Huang and Weibing Zhang Mol Ther 2012 20: 196-203; advance online publication, October 11, 2011; 10.1038/mt.2011.205 Abstract | Full Text |  |  |  | Normal Collagen and Bone Production by Gene-targeted Human Osteogenesis Imperfecta iPSCsDavid R Deyle, Iram F Khan, Gaoying Ren, Pei-Rong Wang, Jordan Kho, Ulrike Schwarze and David W Russell Mol Ther 2012 20: 204-213; advance online publication, October 25, 2011; 10.1038/mt.2011.209 Abstract | Full Text |  |  |  | Safety and Tolerability of the Easy Vax™ Clinical Epidermal Electroporation System in Healthy AdultsSamer S El-Kamary, Melissa Billington, Stephen Deitz, Elaina Colby, Howard Rhinehart, Yukun Wu, William Blackwelder, Robert Edelman, Albert Lee and Alan King Mol Ther 2012 20: 214-220; advance online publication, November 8, 2011; 10.1038/mt.2011.235 Abstract | Full Text |  |  |  | Verapamil Results in Increased Blood Levels of Oncolytic Adenovirus in Treatment of Patients With Advanced CancerAnniina Koski, Mari Raki, Petri Nokisalmi, Ilkka Liikanen, Lotta Kangasniemi, Timo Joensuu, Anna Kanerva, Sari Pesonen, Ramon Alemany and Akseli Hemminki Mol Ther 2012 20: 221-229; advance online publication, November 1, 2011; 10.1038/mt.2011.230 Abstract | Full Text |  |  |  | Hematopoietic Stem Cell Mobilization for Gene Therapy of Adult Patients With Severe β-Thalassemia: Results of Clinical Trials Using G-CSF or Plerixafor in Splenectomized and Nonsplenectomized SubjectsEvangelia Yannaki, Thalia Papayannopoulou, Erica Jonlin, Fani Zervou, Garyfalia Karponi, Angeliki Xagorari, Pamela Becker, Nikoleta Psatha, Ioannis Batsis, Panayotis Kaloyannidis, Varvara Tahynopoulou, Varnavas Constantinou, Asimina Bouinta, Konstantia Kotta, Aglaia Athanassiadou, Achilles Anagnostopoulos, Athanasios Fassas and George Stamatoyannopoulos Mol Ther 2012 20: 230-238; advance online publication, September 27, 2011; 10.1038/mt.2011.195 Abstract | Full Text |  | Corrigendum | Top |  | Corrigendum: Treatment of Brain Inflammatory Diseases by Delivering Exosome Encapsulated Anti-Inflammatory Drugs From the Nasal Region to the BrainMol Ther 2012 20: 239; 10.1038/mt.2011.222 Full Text |  |  |  | Dedicated to the treatment of genetic and acquired diseases? 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