TABLE OF CONTENTS

Volume 15, Issue 9 (September 2007)

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Editorial

Humility and Clinical Trials FREE

James Wilson Associate Editor

Mol Ther 15: 1571-1572; doi:10.1038/sj.mt.6300279

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In This Issue

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Research Highlights

Research Highlights FREE

Mol Ther 15: 1575; doi:10.1038/sj.mt.6300285

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Commentary

A Decade of Accomplishments: Gene Therapy and the ASGT FREE

Theodore Friedmann

Mol Ther 15: 1576-1578; doi:10.1038/sj.mt.6300284

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Review

Vector Engineering and Delivery

Non-invasive Imaging in Gene Therapy FREE

Jani Kristian Räty, Timo Liimatainen, Minna Unelma Kaikkonen, Olli Gröhn, Kari Jumani Airenne and Seppo Ylä-Herttuala

Mol Ther 15: 1579-1586; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300233

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Original Articles

Monogenic Disease

Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse FREE

Sue Fletcher, Kaite Honeyman, Abbie M Fall, Penny L Harding, Russell D Johnsen, Joshua P Steinhaus, Hong M Moulton, Patrick L Iversen and Stephen D Wilton

Mol Ther 15: 1587-1592; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300245

Acquired and Multigenic Disease

DNAzyme-mediated Inhibition of Japanese Encephalitis Virus Replication in Mouse Brain FREE

Mohan Babu Appaiahgari and Sudhanshu Vrati

Mol Ther 15: 1593-1599; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300231

Replication-competent Adenovirus-mediated Suicide Gene Therapy with Radiation in a Preclinical Model of Pancreatic Cancer FREE

Svend O Freytag, Kenneth N Barton, Stephen L Brown, Vinod Narra, Yingshu Zhang, Don Tyson, Colleen Nall, Mei Lu, Munther Ajlouni, Benjamin Movsas and Jae Ho Kim

Mol Ther 15: 1600-1606; advance online publication, June 12, 2007; doi:10.1038/sj.mt.6300212

Systemic Toxicity–Efficacy Profile of ICOVIR-5, a Potent and Selective Oncolytic Adenovirus Based on the pRB Pathway FREE

Manel Cascallo, Marta M Alonso, Juan J Rojas, Anna Perez-Gimenez, Juan Fueyo and Ramon Alemany

Mol Ther 15: 1607-1615; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300239

Decorin Gene Transfer Promotes Muscle Cell Differentiation and Muscle Regeneration FREE

Yong Li, Juan Li, Jinghong Zhu, Bin Sun, Maria Branca, Ying Tang, William Foster, Xiao Xiao and Johnny Huard

Mol Ther 15: 1616-1622; advance online publication, July 3, 2007; doi:10.1038/sj.mt.6300250

Peripheral Transgene Expression of Plasma Gelsolin Reduces Amyloid in Transgenic Mouse Models of Alzheimer's Disease FREE

Aaron C Hirko, Edwin M Meyer, Michael A King and Jeffery A Hughes

Mol Ther 15: 1623-1629; advance online publication, July 3, 2007; doi:10.1038/sj.mt.6300253

Vector Engineering and Delivery

Functional Analysis of Various Promoters in Lentiviral Vectors at Different Stages of In Vitro Differentiation of Mouse Embryonic Stem Cells FREE

Sunghoi Hong, Dong-Youn Hwang, Soonsang Yoon, Ole Isacson, Ali Ramezani, Robert G Hawley and Kwang-Soo Kim

Mol Ther 15: 1630-1639; advance online publication, July 3, 2007; doi:10.1038/sj.mt.6300251

Improved Retinal Transduction In Vivo and Photoreceptor-specific Transgene Expression Using Adenovirus Vectors With Modified Penton Base FREE

Siobhan M Cashman, Laura McCullough and Rajendra Kumar-Singh

Mol Ther 15: 1640-1646; advance online publication, May 15, 2007; doi:10.1038/sj.mt.6300203

Development of Renal-targeted Vectors Through Combined In Vivo Phage Display and Capsid Engineering of Adenoviral Fibers From Serotype 19p FREE

Laura Denby, Lorraine M Work, Dan J Von Seggern, Eugene Wu, John H McVey, Stuart A Nicklin and Andrew H Baker

Mol Ther 15: 1647-1654; advance online publication, June 5, 2007; doi:10.1038/sj.mt.6300214

Bone Regeneration by Regulated In Vivo Gene Transfer Using Biocompatible Polyplex Nanomicelles FREE

Keiji Itaka, Shinsuke Ohba, Kanjiro Miyata, Hiroshi Kawaguchi, Kozo Nakamura, Tsuyoshi Takato, Ung-Il Chung and Kazunori Kataoka

Mol Ther 15: 1655-1662; advance online publication, June 5, 2007; doi:10.1038/sj.mt.6300218

Vector Toxicology, Immunogenicity and Safety

2'-O-methyl-modified RNAs Act as TLR7 Antagonists FREE

Marjorie Robbins, Adam Judge, Lisa Liang, Kevin McClintock, Ed Yaworski and Ian MacLachlan

Mol Ther 15: 1663-1669; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300240

Long-term Engraftment of Single Genetically Modified Human Epidermal Holoclones Enables Safety Pre-assessment of Cutaneous Gene Therapy FREE

Fernando Larcher, Elena Dellambra, Laura Rico, Sergio Bondanza, Rodolfo Murillas, Claudia Cattoglio, Fulvio Mavilio, José L Jorcano, Giovanna Zambruno and Marcela Del Rio

Mol Ther 15: 1670-1676; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300238

Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice FREE

Denise E Sabatino, Tippi C MacKenzie, William Peranteau, Shyrie Edmonson, Cesare Campagnoli, Yi-Lin Liu, Alan W Flake and Katherine A High

Mol Ther 15: 1677-1685; advance online publication, June 12, 2007; doi:10.1038/sj.mt.6300219

Targeted Inflammation During Oncolytic Virus Therapy Severely Compromises Tumor Blood Flow FREE

Caroline J Breitbach, Jennifer M Paterson, Chantal G Lemay, Theresa J Falls, Allison McGuire, Kelley A Parato, David F Stojdl, Manijeh Daneshmand, Kelly Speth, David Kirn, J Andrea McCart, Harold Atkins and John C Bell

Mol Ther 15: 1686-1693; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300215

Activation of CFTR-specific T Cells in Cystic Fibrosis Mice Following Gene Transfer FREE

Maria P Limberis, Joanita Figueredo, Roberto Calcedo and James M Wilson

Mol Ther 15: 1694-1700; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300210

Cell Therapy

Long-term Correction of beta-Thalassemia With Minimal Cellular Requirement and Transplantation Modalities FREE

Hady Felfly and Marie Trudel

Mol Ther 15: 1701-1709; advance online publication, May 22, 2007; doi:10.1038/sj.mt.6300204

Transplanted Hepatocytes Over-expressing FoxM1B Efficiently Repopulate Chronically Injured Mouse Liver Independent of Donor Age FREE

Nicolas Brezillon, Martine Lambert-Blot, Serban Morosan, Dominique Couton, Claudia Mitchell, Dina Kremsdorf, Robert H Costa, Helene Gilgenkrantz and Jacques-Emmanuel Guidotti

Mol Ther 15: 1710-1715; advance online publication, June 12, 2007; doi:10.1038/sj.mt.6300232

Vaccine Technology

Successful Immunization with a Single Injection of Non-integrating Lentiviral Vector FREE

Donatella RM Negri, Zuleika Michelini, Silvia Baroncelli, Massimo Spada, Silvia Vendetti, Viviana Buffa, Roberta Bona, Pasqualina Leone, Mary E Klotman and Andrea Cara

Mol Ther 15: 1716-1723; advance online publication, June 26, 2007; doi:10.1038/sj.mt.6300241

A New Multi-clade DNA Prime/Recombinant MVA Boost Vaccine Induces Broad and High Levels of HIV-1-specific CD8+ T-cell and Humoral Responses in Mice FREE

Andreas Bråve, Andreas Boberg, Lindvi Gudmundsdotter, Erik Rollman, Kristian Hallermalm, Karl Ljungberg, Pontus Blomberg, Richard Stout, Staffan Paulie, Eric Sandström, Gunnel Biberfeld, Patricia Earl, Bernard Moss, Josephine H. Cox and Britta Wahren

Mol Ther 15: 1724-1733; advance online publication, June 19, 2007; doi:10.1038/sj.mt.6300235

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Corrigenda

Corrigendum to "Intranasal Administration of the Growth-compromised HSV-2 Vector DeltaRR Prevents Kainate-induced Seizures and Neuronal Loss in Rats and Mice" FREE

Jennifer M Laing, Michael D Gober, Erin K Golembewski, Scott M Thompson, Kymberly A Gyure, Paul J Yarowsky and Laure Aurelian

Mol Ther 15: 1734; doi:10.1038/sj.mt.6300211

Corrigendum to "Cellular Dynamics of EGF Receptor–targeted Synthetic Viruses" FREE

Karla de Bruin, Nadia Ruthardt, Katharina von Gersdorff, Ralf Bausinger, Ernst Wagner, Manfred Ogris and Christoph Bräuchle

Mol Ther 15: 1735; doi:10.1038/sj.mt.6300247

Corrigendum to "Avian Influenza Virus Hemagglutinin Display on Baculovirus Envelope: Cytoplasmic Domain Affects Virus Properties and Vaccine Potential" FREE

Ding-Gang Yang, Yao-Chi Chung, Yiu-Kay Lai, Chia-Wei Lai, Hung-Jen Liu and Yu-Chen Hu

Mol Ther 15: 1736; doi:10.1038/sj.mt.6300236

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