TABLE OF CONTENTS
Volume 17, Issue 7 (July 2009)
In This Issue |
Research Highlights |
Commentary |
Review |
Original Articles |
Corrigenda
Editorial
An Integrated Funding Mechanism for Clinical Trials in Human Gene Therapy FREE
Arthur Nienhuis and Theodore Friedmann
Mol Ther 17: 1117-1118; doi:10.1038/mt.2009.129
In This Issue
In This Issue FREE
Mol Ther 17: 1120; doi:10.1038/mt.2009.131
See also: Original Article by Daadi et al. See also: Original Article by Yeghiazarians et al. See also: Original Article by Wang et al. See also: Original Article by Duque et al. See also: Original Article by Vink et al. See also: Original Article by Staunstrup et al.Research Highlights
Research Highlights
Research Highlights
Mol Ther 17: 1121; doi:10.1038/mt.2009.132
Commentary
A Case for Immunosuppression for Myoblast Transplantation in Duchenne Muscular Dystrophy
Jacques P Tremblay, Daniel Skuk, Benjamino Palmieri and David M Rothstein
Mol Ther 17: 1122-1124; doi:10.1038/mt.2009.125
Review
Acquired and Multigenic Disease
Herpes Simplex Virus Oncolytic Therapy for Pediatric Malignancies
Gregory K Friedman, Joseph G Pressey, Alyssa T Reddy, James M Markert and G Yancey Gillespie
Mol Ther 17: 1125-1135; advance online publication, April 14, 2009; doi:10.1038/mt.2009.73
Original Articles
Monogenic Disease
Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System
Elena L Aronovich, Jason B Bell, Shaukat A Khan, Lalitha R Belur, Roland Gunther, Brenda Koniar, Patricia A Schachern, Josh B Parker, Cathy S Carlson, Chester B Whitley, R Scott McIvor, Pankaj Gupta and Perry B Hackett
Mol Ther 17: 1136-1144; advance online publication, April 21, 2009; doi:10.1038/mt.2009.87
Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A
Christopher B Doering, Gabriela Denning, Kerry Dooriss, Bagirath Gangadharan, Jennifer M Johnston, Keith W Kerstann, David A McCarty and H Trent Spencer
Mol Ther 17: 1145-1154; advance online publication, March 3, 2009; doi:10.1038/mt.2009.35
Short-term Correction of Arginase Deficiency in a Neonatal Murine Model With a Helper-dependent Adenoviral Vector
Chia-Ling Gau, Robin A Rosenblatt, Vincenzo Cerullo, Fides D Lay, Adrienne C Dow, Justin Livesay, Nicola Brunetti-Pierri, Brendan Lee, Stephen D Cederbaum, Wayne W Grody and Gerald S Lipshutz
Mol Ther 17: 1155-1163; advance online publication, April 14, 2009; doi:10.1038/mt.2009.65
Acquired and Multigenic Disease
Oncolytic Rat Parvovirus H-1PV, a Candidate for the Treatment of Human Lymphoma: In Vitro and In Vivo Studies
Assia L Angelova, Marc Aprahamian, Ginette Balboni, Henri-Jacques Delecluse, Regina Feederle, Irina Kiprianova, Svitlana P Grekova, Angel S Galabov, Mathias Witzens-Harig, Anthony D Ho, Jean Rommelaere and Zahari Raykov
Mol Ther 17: 1164-1172; advance online publication, April 14, 2009; doi:10.1038/mt.2009.78
HSV-mediated Transfer of Artemin Overcomes Myelin Inhibition to Improve Outcome After Spinal Cord Injury
Zhigang Zhou, Xiangmin Peng, David J Fink and Marina Mata
Mol Ther 17: 1173-1179; advance online publication, March 17, 2009; doi:10.1038/mt.2009.52
Intratracheal Gene Transfer of Adrenomedullin Using Polyplex Nanomicelles Attenuates Monocrotaline-induced Pulmonary Hypertension in Rats
Mariko Harada-Shiba, Itaru Takamisawa, Kanjiro Miyata, Takehiko Ishii, Nobuhiro Nishiyama, Keiji Itaka, Kenji Kangawa, Fumiki Yoshihara, Yujiro Asada, Kinta Hatakeyama, Noriya Nagaya and Kazunori Kataoka
Mol Ther 17: 1180-1186; advance online publication, March 31, 2009; doi:10.1038/mt.2009.63
Vector Engineering and Delivery
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
Sandra Duque, Béatrice Joussemet, Christel Riviere, Thibaut Marais, Laurence Dubreil, Anne-Marie Douar, John Fyfe, Philippe Moullier, Marie-Anne Colle and Martine Barkats
Mol Ther 17: 1187-1196; advance online publication, April 14, 2009; doi:10.1038/mt.2009.71
Sleeping Beauty Transposition From Nonintegrating Lentivirus
Conrad A Vink, H Bobby Gaspar, Richard Gabriel, Manfred Schmidt, R Scott McIvor, Adrian J Thrasher and Waseem Qasim
Mol Ther 17: 1197-1204; advance online publication, May 5, 2009; doi:10.1038/mt.2009.94
Vector Toxicology, Immunogenicity and Safety
Hybrid Lentivirus-transposon Vectors With a Random Integration Profile in Human Cells
Nicklas H Staunstrup, Brian Moldt, Lajos Mátés, Palle Villesen, Maria Jakobsen, Zoltán Ivics, Zsuzsanna Izsvák and Jacob Giehm Mikkelsen
Mol Ther 17: 1205-1214; advance online publication, February 24, 2009; doi:10.1038/mt.2009.10
A Preclinical Animal Model to Assess the Effect of Pre-existing Immunity on AAV-mediated Gene Transfer
Hua Li, Shih-Wen Lin, Wynetta Giles-Davis, Yan Li, Dongming Zhou, Zhi Quan Xiang, Katherine A High and Hildegund CJ Ertl
Mol Ther 17: 1215-1224; advance online publication, April 14, 2009; doi:10.1038/mt.2009.79
Oligonucleotide Therapy
The Effect of Chemical Modification and Nanoparticle Formulation on Stability and Biodistribution of siRNA in Mice
Shan Gao, Frederik Dagnaes-Hansen, Ebbe Juel Bech Nielsen, Jesper Wengel, Flemming Besenbacher, Kenneth Alan Howard and Jørgen Kjems
Mol Ther 17: 1225-1233; advance online publication, April 28, 2009; doi:10.1038/mt.2009.91
Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD Therapy
Aurélie Goyenvalle, Arran Babbs, Gert-Jan B van Ommen, Luis Garcia and Kay E Davies
Mol Ther 17: 1234-1240; advance online publication, May 19, 2009; doi:10.1038/mt.2009.113
Cell Therapy
Autologous Bone Marrow Stromal Cells Genetically Engineered to Secrete an IGF-I Receptor Decoy Prevent the Growth of Liver Metastases
Ni Wang, Lucia Fallavollita, Long Nguyen, Julia Burnier, Moutih Rafei, Jacques Galipeau, Shoshana Yakar and Pnina Brodt
Mol Ther 17: 1241-1249; advance online publication, April 14, 2009; doi:10.1038/mt.2009.82
Injection of Bone Marrow Cell Extract Into Infarcted Hearts Results in Functional Improvement Comparable to Intact Cell Therapy
Yerem Yeghiazarians, Yan Zhang, Megha Prasad, Henry Shih, Shereen A Saini, Junya Takagawa, Richard E Sievers, Maelene L Wong, Neel K Kapasi, Rachel Mirsky, Juha Koskenvuo, Petros Minasi, Jianqin Ye, Mohan N Viswanathan, Franca S Angeli, Andrew J Boyle, Matthew L Springer and William Grossman
Mol Ther 17: 1250-1256; advance online publication, April 21, 2009; doi:10.1038/mt.2009.85
Engraftment of Bone Marrow–derived Stem Cells to the Lung in a Model of Acute Respiratory Infection by Pseudomonas aeruginosa
Joanna Rejman, Carla Colombo and Massimo Conese
Mol Ther 17: 1257-1265; advance online publication, May 5, 2009; doi:10.1038/mt.2009.96
Hepatocyte Transplantation Improves Phenotype and Extends Survival in a Murine Model of Intermediate Maple Syrup Urine Disease
Kristen J Skvorak, Harbhajan S Paul, Kenneth Dorko, Fabio Marongiu, Ewa Ellis, Donald Chace, Carolyn Ferguson, K Michael Gibson, Gregg E Homanics and Stephen C Strom
Mol Ther 17: 1266-1273; advance online publication, May 12, 2009; doi:10.1038/mt.2009.99
Therapeutic Immunity by Adoptive Tumor-primed CD4+ T-cell Transfer in Combination With In Vivo GITR Ligation
Zuqiang Liu, Shenghe Tian, Louis D Falo Jr, Shimon Sakaguchi and Zhaoyang You
Mol Ther 17: 1274-1281; advance online publication, May 5, 2009; doi:10.1038/mt.2009.100
Molecular and Magnetic Resonance Imaging of Human Embryonic Stem Cell–Derived Neural Stem Cell Grafts in Ischemic Rat Brain
Marcel M Daadi, Zongjin Li, Ahmet Arac, Brad A Grueter, Marc Sofilos, Robert C Malenka, Joseph C Wu and Gary K Steinberg
Mol Ther 17: 1282-1291; advance online publication, May 12, 2009; doi:10.1038/mt.2009.104
Clinical Trials
A Phase I/II Clinical Trial in Localized Prostate Cancer of an Adenovirus Expressing Nitroreductase with CB1984
Prashant Patel, J Graham Young, Vivien Mautner, Daniel Ashdown, Sarah Bonney, Robert G Pineda, Stuart I Collins, Peter F Searle, Diana Hull, Elizabeth Peers, John Chester, D Michael Wallace, Alan Doherty, Hing Leung, Lawrence S Young and Nicholas D James
Mol Ther 17: 1292-1299; advance online publication, April 14, 2009; doi:10.1038/mt.2009.80
Corrigenda
Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models" FREE
Francesco Marangoni, Marita Bosticardo, Sabine Charrier, Elena Draghici, Michela Locci, Samantha Scaramuzza, Cristina Panaroni, Maurilio Ponzoni, Francesca Sanvito, Claudio Doglioni, Marie Liabeuf, Bernard Gjata, Marie Montus, Katherine Siminovitch, Alessandro Aiuti, Luigi Naldini, Loïc Dupré, Maria Grazia Roncarolo, Anne Galy and Anna Villa
Mol Ther 17: 1300; doi:10.1038/mt.2009.61
Corrigendum to "Clustering and Internalization of Integrin 
v
3 With a Tetrameric RGD-synthetic Peptide" FREE
Mol Ther 17: 1301; doi:10.1038/mt.2009.109
Corrigendum to "A Phase I/II Clinical Trial in Localized Prostate Cancer of an Adenovirus Expressing Nitroreductase With CB1984" FREE
Mol Ther 17: 1302; doi:10.1038/mt.2009.110
