TABLE OF CONTENTS

Volume 16, Issue 3 (March 2008)

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Editorial

NIH Recombinant DNA Advisory Committee Continues to Ponder Adverse Event Associated With AAV Gene Therapy Trial FREE

David A Williams

Mol Ther 16: 427-428; doi:10.1038/mt.2008.2

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In This Issue

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Research Highlights

Research Highlights FREE

Mol Ther 16: 431; doi:10.1038/mt.2008.21

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Commentary

En Route to Ethical Recommendations for Gene Transfer Clinical Trials FREE

Nancy MP King and Odile Cohen-Haguenauer

Mol Ther 16: 432-438; doi:10.1038/mt.2008.13

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Reviews

Vector Toxicology, Immunogenicity and Safety

The MDS1–EVI1 Gene Complex as a Retrovirus Integration Site: Impact on Behavior of Hematopoietic Cells and Implications for Gene Therapy FREE

Jean-Yves Métais and Cynthia E Dunbar

Mol Ther 16: 439-449; advance online publication, January 29, 2008; doi:10.1038/sj.mt.6300372

Cell Therapy

Genetic Modification of Neural Stem Cells FREE

Rahul Jandial, Ilyas Singec, Christopher P Ames and Evan Y Snyder

Mol Ther 16: 450-457; advance online publication, February 5, 2008; doi:10.1038/sj.mt.6300402

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Original Articles

Monogenic Disease

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer FREE

Jeannette Bennicelli, John Fraser Wright, Andras Komaromy, Jonathan B Jacobs, Bernd Hauck, Olga Zelenaia, Federico Mingozzi, Daniel Hui, Daniel Chung, Tonia S Rex, Zhangyong Wei, Guang Qu, Shangzhen Zhou, Caroline Zeiss, Valder R Arruda, Gregory M Acland, Lou F Dell'Osso, Katherine A High, Albert M Maguire and Jean Bennett

Mol Ther 16: 458-465; advance online publication, January 22, 2008; doi:10.1038/sj.mt.6300389

Acquired and Multigenic Disease

Freeze-dried Tendon Allografts as Tissue-engineering Scaffolds for Gdf5 Gene Delivery FREE

Patrick Basile, Tulin Dadali, Justin Jacobson, Sys Hasslund, Michael Ulrich-Vinther, Kjeld Søballe, Yasuhiko Nishio, M Hicham Drissi, Howard N Langstein, David J Mitten, Regis J O'Keefe, Edward M Schwarz and Hani A Awad

Mol Ther 16: 466-473; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300395

Protection Against Aminoglycoside-induced Ototoxicity by Regulated AAV Vector–mediated GDNF Gene Transfer Into the Cochlea FREE

Yuhe Liu, Takashi Okada, Kuniko Shimazaki, Kianoush Sheykholeslami, Tatsuya Nomoto, Shin-Ichi Muramatsu, Hiroaki Mizukami, Akihiro Kume, Shuifang Xiao, Keiichi Ichimura and Keiya Ozawa

Mol Ther 16: 474-480; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300379

CNS Delivery of Vectored Prion-specific Single-chain Antibodies Delays Disease Onset FREE

Charles A Wuertzer, Mark A Sullivan, Xing Qiu and Howard J Federoff

Mol Ther 16: 481-486; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300387

Delta-24-RGD in Combination With RAD001 Induces Enhanced Anti-glioma Effect via Autophagic Cell Death FREE

Marta M Alonso, Hong Jiang, Tomohisa Yokoyama, Jing Xu, Nebiyou B Bekele, Frederick F Lang, Seiji Kondo, Candelaria Gomez-Manzano and Juan Fueyo

Mol Ther 16: 487-493; advance online publication, February 5, 2008; doi:10.1038/sj.mt.6300400

Vector Engineering and Delivery

Adeno-associated Virus of a Single-polarity DNA Genome Is Capable of Transduction In Vivo  FREE

Xiaohuai Zhou, Xinghua Zeng, Zhenghong Fan, Chengwen Li, Thomas McCown, R Jude Samulski and Xiao Xiao

Mol Ther 16: 494-499; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300397

Inducible Packaging Cells for Large-scale Production of Lentiviral Vectors in Serum-free Suspension Culture FREE

Sophie Broussau, Nadine Jabbour, Guillaume Lachapelle, Yves Durocher, Rosanne Tom, Julia Transfiguracion, Rénald Gilbert and Bernard Massie

Mol Ther 16: 500-507; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300383

Receptor-targeted Nanocomplexes optimized for Gene Transfer to Primary Vascular Cells and Explant Cultures of Rabbit Aorta FREE

Scott A Irvine, Qing-Hai Meng, Faiza Afzal, Jimmy Ho, John B Wong, Helen C Hailes, Alethea B Tabor, Jean R McEwan and Stephen L Hart

Mol Ther 16: 508-515; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300381

Development of Peptide-targeted Lipoplexes to CXCR4-expressing Rat Glioma Cells and Rat Proliferating Endothelial Cells FREE

Wouter HP Driessen, Nobutaka Fujii, Hirokazu Tamamura and Sean M Sullivan

Mol Ther 16: 516-524; advance online publication, January 15, 2008; doi:10.1038/sj.mt.6300388

Vector Toxicology, Immunogenicity and Safety

Globin Lentiviral Vector Insertions Can Perturb the Expression of Endogenous Genes in beta-thalassemic Hematopoietic Cells FREE

Phillip W Hargrove, Steven Kepes, Hideki Hanawa, John C Obenauer, Deiqing Pei, Cheng Cheng, John T Gray, Geoffrey Neale and Derek A Persons

Mol Ther 16: 525-533; advance online publication, January 15, 2008; doi:10.1038/sj.mt.6300394

A Rapid and Quantitative Assay for Measuring Neighboring Gene Activation by Vector Proviruses FREE

Paul C Hendrie, Yunwen Huo, Raisa B Stolitenko and David W Russell

Mol Ther 16: 534-540; advance online publication, January 22, 2008; doi:10.1038/sj.mt.6300398

Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles FREE

Stéphanie Lorain, David-Alexandre Gross, Aurélie Goyenvalle, Olivier Danos, Jean Davoust and Luis Garcia

Mol Ther 16: 541-547; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300377

Silencing of Episomal Transgene Expression in Liver by Plasmid Bacterial Backbone DNA Is Independent of CpG Methylation FREE

Zhi-Ying Chen, Efren Riu, Chen-Yi He, Hui Xu and Mark A Kay

Mol Ther 16: 548-556; advance online publication, February 5, 2008; doi:10.1038/sj.mt.6300399

Oligonucleotide Therapy

Lentiviral Vector Design for Multiple shRNA Expression and Durable HIV-1 Inhibition FREE

Olivier ter Brake, Karen 't Hooft, Ying Poi Liu, Mireille Centlivre, Karin Jasmijn von Eije and Ben Berkhout

Mol Ther 16: 557-564; advance online publication, January 15, 2008; doi:10.1038/sj.mt.6300382

Promoter-targeted siRNAs Induce Gene Silencing of Simian Immunodeficiency Virus (SIV) Infection In Vitro  FREE

Heidi GW Lim, Kazuo Suzuki, David A Cooper and Anthony D Kelleher

Mol Ther 16: 565-570; advance online publication, January 29, 2008; doi:10.1038/sj.mt.6300380

Cell Therapy

Targeted Migration of Mesenchymal Stem Cells Modified With CXCR4 Gene to Infarcted Myocardium Improves Cardiac Performance FREE

Zhaokang Cheng, Lailiang Ou, Xin Zhou, Fei Li, Xiaohua Jia, Yinguo Zhang, Xiaolei Liu, Yuming Li, Christopher A Ward, Luis G Melo and Deling Kong

Mol Ther 16: 571-579; advance online publication, February 5, 2008; doi:10.1038/sj.mt.6300374

Sleeping Beauty Transposon-mediated Engineering of Human Primary T Cells for Therapy of CD19+ Lymphoid Malignancies FREE

Xin Huang, Hongfeng Guo, Johnthomas Kang, Suet Choi, Tom C Zhou, Syam Tammana, Christopher J Lees, Zhong-Ze Li, Michael Milone, Bruce L Levine, Jakub Tolar, Carl H June, R Scott McIvor, John E Wagner, Bruce R Blazar and Xianzheng Zhou

Mol Ther 16: 580-589; advance online publication, January 29, 2008; doi:10.1038/sj.mt.6300404

Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency FREE

Susannah I Thornhill, Axel Schambach, Steven J Howe, Meera Ulaganathan, Elke Grassman, David Williams, Bernhard Schiedlmeier, Neil J Sebire, H Bobby Gaspar, Christine Kinnon, Christopher Baum and Adrian J Thrasher

Mol Ther 16: 590-598; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300393

Engineering Human Tumor-specific Cytotoxic T Cells to Function in a Hypoxic Environment FREE

Hongsung Kim, Guangyong Peng, John M Hicks, Heidi L Weiss, Erwin G Van Meir, Malcolm K Brenner and Patricia Yotnda

Mol Ther 16: 599-606; advance online publication, January 29, 2008; doi:10.1038/sj.mt.6300391

Vaccine Technology

Tumor Protection Following Vaccination With Low Doses of Lentivirally Transduced DCs Expressing the Self-antigen erbB2 FREE

Miriam E Mossoba, Jagdeep S Walia, Vanessa I Rasaiah, Nicole Buxhoeveden, Renee Head, Chuyan Ying, Jason E Foley, Jonathan L Bramson, Daniel H Fowler and Jeffrey A Medin

Mol Ther 16: 607-617; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300390

Clinical Trials

A Phase I Trial of Ad.hIFN-beta Gene Therapy for Glioma FREE

E Antonio Chiocca, Katie M Smith, Byron McKinney, Cheryl A Palmer, Steven Rosenfeld, Kevin Lillehei, Allan Hamilton, Betty K DeMasters, Kevin Judy and David Kirn

Mol Ther 16: 618-626; advance online publication, January 8, 2008; doi:10.1038/sj.mt.6300396

A Phase I Trial of Intratumoral Administration of Reovirus in Patients With Histologically Confirmed Recurrent Malignant Gliomas FREE

Peter Forsyth, Gloria Roldán, David George, Carla Wallace, Cheryl Ann Palmer, Don Morris, Gregory Cairncross, Maureen Vallee Matthews, James Markert, Yancey Gillespie, Matt Coffey, Brad Thompson and Mark Hamilton

Mol Ther 16: 627-632; advance online publication, February 5, 2008; doi:10.1038/sj.mt.6300403

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Corrigendum

Corrigendum to "Prolonged Susceptibility to Antibody-mediated Neutralization for Adeno-associated Vectors Targeted to the Liver" FREE

Samuel L Murphy, Hojun Li, Shangzhen Zhou, Alexander Schlachterman and Katherine A High

Mol Ther 16: 633; doi:10.1038/sj.mt.6300386

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