ADVANCE ONLINE PUBLICATION
The latest research papers, published online ahead of print. These online versions are definitive and may be cited using the digital object identifier (DOI).
About advance online publication
November 10 2009 |
November 3 2009 |
October 27 2009 |
October 20 2009 |
October 13 2009 |
October 6 2009 |
September 22 2009 |
September 15 2009 |
September 8 2009 |
September 1 2009 |
August 18 2009 |
August 11 2009 |
August 4 2009 |
July 28 2009 |
July 21 2009 |
July 14 2009
November 17 2009
Original Article
Pretranscriptional Regulation of Tgf-
1 by PI Polyamide Prevents Scarring and Accelerates Wound Healing of the Cornea After Exposure to Alkali
Min Chen, Hiroyuki Matsuda, Linghua Wang, Takayoshi Watanabe, Makoto T Kimura, Jun Igarashi, Xiaofei Wang, Tohru Sakimoto, Noboru Fukuda, Mitsuru Sawa and Hiroki Nagase
Mol Ther advance online publication, November 17, 2009; doi:10.1038/mt.2009.263
November 10 2009
Original Articles
Characterization of Genome Integrity for Oversized Recombinant AAV Vector
Biao Dong, Hiroyuki Nakai and Weidong Xiao
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.258
Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction
Jonathan D Finn, Daniel Hui, Harre D Downey, Danielle Dunn, Gary C Pien, Federico Mingozzi, Shangzhen Zhou and Katherine A High
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.257
Adipose-derived Stem Cells as Therapeutic Delivery Vehicles of an Oncolytic Virus for Glioblastoma
Darnell T Josiah, Dongqin Zhu, Fernanda Dreher, John Olson, Grant McFadden and Hannah Caldas
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.265
Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome 
8.2 kb
Yi Lai, Yongping Yue and Dongsheng Duan
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.256
Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery MTOpen
Louise R Rodino-Klapac, Chrystal L Montgomery, William G Bremer, Kimberly M Shontz, Vinod Malik, Nancy Davis, Spencer Sprinkle, Katherine J Campbell, Zarife Sahenk, K Reed Clark, Christopher M Walker, Jerry R Mendell and Louis G Chicoine
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.254
Effect of Genome Size on AAV Vector Packaging
Zhijian Wu, Hongyan Yang and Peter Colosi
Mol Ther advance online publication, November 10, 2009; doi:10.1038/mt.2009.255
November 3 2009
Original Articles
Eradication of Therapy-resistant Human Prostate Tumors Using an Ultrasound-guided Site-specific Cancer Terminator Virus Delivery Approach
Adelaide Greco, Altomare Di Benedetto, Candace M Howard, Sarah Kelly, Rounak Nande, Yulia Dementieva, Michele Miranda, Arturo Brunetti, Marco Salvatore, Luigi Claudio, Devanand Sarkar, Paul Dent, David T Curiel, Paul B Fisher and Pier P Claudio
Mol Ther advance online publication, November 3, 2009; doi:10.1038/mt.2009.252
Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression
En Kimura, Sheng Li, Paul Gregorevic, Brent M Fall and Jeffrey S Chamberlain
Mol Ther advance online publication, November 3, 2009; doi:10.1038/mt.2009.253
Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle
Alice Toromanoff, Oumeya Adjali, Thibaut Larcher, Marcelo Hill, Lydie Guigand, Pierre Chenuaud, Jack-Yves Deschamps, Olivier Gauthier, Gilles Blancho, Bernard Vanhove, Fabienne Rolling, Yan Chérel, Philippe Moullier, Ignacio Anegon and Caroline Le Guiner
Mol Ther advance online publication, November 3, 2009; doi:10.1038/mt.2009.251
The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques
Lili Wang, Roberto Calcedo, Huan Wang, Peter Bell, Rebecca Grant, Luk H Vandenberghe, Julio Sanmiguel, Hiroki Morizono, Mark L Batshaw and James M Wilson
Mol Ther advance online publication, November 3, 2009; doi:10.1038/mt.2009.245
October 27 2009
Original Articles
Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy MTOpen
Randy J Chandler and Charles P Venditti
Mol Ther advance online publication, October 27, 2009; doi:10.1038/mt.2009.247
Long-term Physiologically Regulated Expression of the Low-density Lipoprotein Receptor In Vivo Using Genomic DNA Mini-gene Constructs
Olivia C Hibbitt, Eileen McNeil, Michele MP Lufino, Len Seymour, Keith Channon and Richard Wade-Martins
Mol Ther advance online publication, October 27, 2009; doi:10.1038/mt.2009.249
Overexpression of Sonic Hedgehog in the Lung Mimics the Effect of Lung Injury and Compensatory Lung Growth on Pulmonary Sca-1 and CD34 Positive Cells
Anja Krause, Yaqin Xu, Ju Joh, Ralf Hubner, Austen Gess, Thomas Ilic and Stefan Worgall
Mol Ther advance online publication, October 27, 2009; doi:10.1038/mt.2009.229
Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models
Lili Wang, Huan Wang, Peter Bell, Robert J McCarter, Jianping He, Roberto Calcedo, Luk H Vandenberghe, Hiroki Morizono, Mark L Batshaw and James M Wilson
Mol Ther advance online publication, October 27, 2009; doi:10.1038/mt.2009.246
AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function
Makiko Yasuda, David F Bishop, Mary Fowkes, Seng H Cheng, Lin Gan and Robert J Desnick
Mol Ther advance online publication, October 27, 2009; doi:10.1038/mt.2009.250
October 20 2009
Review
Supplying Clotting Factors From Hematopoietic Stem Cell–derived Erythroid and Megakaryocytic Lineage Cells
Michel Sadelain, Alex Chang and Leszek Lisowski
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.238
Original Articles
Low-level shRNA Cytotoxicity Can Contribute to MYC-induced Hepatocellular Carcinoma in Adult Mice
Shelly Beer, David I Bellovin, Joyce S Lee, Kimberly Komatsubara, Lora S Wang, Huishan Koh, Kathleen Börner, Theresa A Storm, Corrine R Davis, Mark A Kay, Dean W Felsher and Dirk Grimm
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.222
Adipose Tissue–derived Mesenchymal Stem Cells Expressing Prodrug-converting Enzyme Inhibit Human Prostate Tumor Growth
Ilaria T Cavarretta, Veronika Altanerova, Miroslava Matuskova, Lucia Kucerova, Zoran Culig and Cestmir Altaner
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.237
Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping
Aurélie Goyenvalle, Arran Babbs, Dave Powell, Ryszard Kole, Sue Fletcher, Steve D Wilton and Kay E Davies
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.248
Enhanced Antitumor Efficacy of Vasculostatin (Vstat120) Expressing Oncolytic HSV-1
Jayson Hardcastle, Kazuhiko Kurozumi, Nina Dmitrieva, Martin P Sayers, Sarwat Ahmad, Peter Waterman, Ralph Weissleder, E Antonio Chiocca and Balveen Kaur
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.232
A Self-inactivating 
-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice
Jason A Metcalf, Xiucui Ma, Bruce Linders, Susan Wu, Axel Schambach, Kevin K Ohlemiller, Attila Kovacs, Mark Bigg, Li He, Douglas M Tollefsen and Katherine P Ponder
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.236
Gene Therapy of Mpl Deficiency: Challenging Balance Between Leukemia and Pancytopenia
Daniel C Wicke, Johann Meyer, Guntram Buesche, Dirk Heckl, Hans Kreipe, Zhixiong Li, Karl H Welte, Matthias Ballmaier, Christopher Baum and Ute Modlich
Mol Ther advance online publication, October 20, 2009; doi:10.1038/mt.2009.233
October 13 2009
Original Articles
Amphiregulin Promotes BAX Inhibition and Resistance to Gefitinib in Non-small-cell Lung Cancers
Benoît Busser, Lucie Sancey, Véronique Josserand, Carole Niang, Marie C Favrot, Jean-Luc Coll and Amandine Hurbin
Mol Ther advance online publication, October 13, 2009; doi:10.1038/mt.2009.226
Amphiregulin Promotes Resistance to Gefitinib in NonSmall Cell Lung Cancer Cells by Regulating Ku70 Acetylation
Benoît Busser, Lucie Sancey, Véronique Josserand, Carole Niang, Saadi Khochbin, Marie C Favrot, Jean-Luc Coll and Amandine Hurbin
Mol Ther advance online publication, October 13, 2009; doi:10.1038/mt.2009.227
Advanced Phase I/II Studies of Targeted Gene Delivery In Vivo: Intravenous Rexin-G for Gemcitabine-resistant Metastatic Pancreatic Cancer MTOpen
Sant P Chawla, Victoria S Chua, Lita Fernandez, Dorris Quon, William C Blackwelder, Erlinda M Gordon and Frederick L Hall
Mol Ther advance online publication, October 13, 2009; doi:10.1038/mt.2009.228
Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway
Terence R Flotte, Anne C Fischer, Jason Goetzmann, Christian Mueller, Liudmila Cebotaru, Ziying Yan, Lilli Wang, James M Wilson, William B Guggino and John F Engelhardt
Mol Ther advance online publication, October 13, 2009; doi:10.1038/mt.2009.230
Polysialic Acid Glycomimetic Promotes Functional Recovery and Plasticity After Spinal Cord Injury in Mice
Ali Mehanna, Igor Jakovcevski, Ay
e Acar, Meifang Xiao, Gabriele Loers, Geneviève Rougon, Andrey Irintchev and Melitta Schachner
Mol Ther advance online publication, October 13, 2009; doi:10.1038/mt.2009.235
October 6 2009
Original Articles
Engineered Newcastle Disease Virus as an Improved Oncolytic Agent Against Hepatocellular Carcinoma
Jennifer Altomonte, Sabrina Marozin, Roland M Schmid and Oliver Ebert
Mol Ther advance online publication, October 6, 2009; doi:10.1038/mt.2009.231
A Comparison of the Growth Responses Following Intramuscular GHRH Plasmid Administration Versus Daily Growth Hormone Injections in Young Pigs
Amir S Khan, Ruxandra Draghia-Akli, Roman J Shypailo, Kenneth I Ellis, Harry Mersmann and Marta L Fiorotto
Mol Ther advance online publication, October 6, 2009; doi:10.1038/mt.2009.224
PiggyBac Transposon-based Inducible Gene Expression In Vivo After Somatic Cell Gene Transfer
Sai K Saridey, Li Liu, Joseph E Doherty, Aparna Kaja, Daniel L Galvan, Bradley S Fletcher and Matthew H Wilson
Mol Ther advance online publication, October 6, 2009; doi:10.1038/mt.2009.234
Combinatorial Control of Suicide Gene Expression by Tissue-specific Promoter and microRNA Regulation for Cancer Therapy
Chunxiao Wu, Jiakai Lin, Michelle Hong, Yukti Choudhury, Poonam Balani, Doreen Leung, Lam H Dang, Ying Zhao, Jieming Zeng and Shu Wang
Mol Ther advance online publication, October 6, 2009; doi:10.1038/mt.2009.225
September 22 2009
Original Articles
Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates
Hemraj B Dodiya, Tomas Bjorklund, James Stansell III, Ronald J Mandel, Deniz Kirik and Jeffrey H Kordower
Mol Ther advance online publication, September 22, 2009; doi:10.1038/mt.2009.216
The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease
Richie Khanna, Rebecca Soska, Yi Lun, Jessie Feng, Michelle Frascella, Brandy Young, Nastry Brignol, Lee Pellegrino, Sheela A Sitaraman, Robert J Desnick, Elfrida R Benjamin, David J Lockhart and Kenneth J Valenzano
Mol Ther advance online publication, September 22, 2009; doi:10.1038/mt.2009.220
Abrogation of Local Cancer Recurrence After Radiofrequency Ablation by Dendritic Cell-based Hyperthermic Tumor Vaccine
Qiong Liu, Bo Zhai, Wen Yang, Le-Xing Yu, Wei Dong, Ya-Qin He, Lei Chen, Liang Tang, Yan Lin, Dan-Dan Huang, Hong-Ping Wu, Meng-Chao Wu, He-Xin Yan and Hong-Yang Wang
Mol Ther advance online publication, September 22, 2009; doi:10.1038/mt.2009.221
Noninvasive Imaging and Radiovirotherapy of Prostate Cancer Using an Oncolytic Measles Virus Expressing the Sodium Iodide Symporter
Pavlos Msaouel, Ianko D Iankov, Cory Allen, Ileana Aderca, Mark J Federspiel, Donald J Tindall, John C Morris, Michael Koutsilieris, Stephen J Russell and Evanthia Galanis
Mol Ther advance online publication, September 22, 2009; doi:10.1038/mt.2009.218
Chimeric Antigen Receptors Combining 4-1BB and CD28 Signaling Domains Augment PI3kinase/AKT/Bcl-XL Activation and CD8+ T Cell–mediated Tumor Eradication
Xiao-Song Zhong, Maiko Matsushita, Jason Plotkin, Isabelle Riviere and Michel Sadelain
Mol Ther advance online publication, September 22, 2009; doi:10.1038/mt.2009.210
September 15 2009
Original Articles
G-rich Oligonucleotides Inhibit HIF-1
and HIF-2 and Block Tumor Growth
Yongli Guan, Kavitha Ramasamy Reddy, Qiqing Zhu, Yifei Li, KangAe Lee, Priya Weerasinghe, Josef Prchal, Gregg L Semenza and Naijie Jing
Mol Ther advance online publication, September 15, 2009; doi:10.1038/mt.2009.219
Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme
Nestor W Meza, Maria E Alonso-Ferrero, Susana Navarro, Oscar Quintana-Bustamante, Antonio Valeri, Maria Garcia-Gomez, Juan A Bueren, Jose M Bautista and Jose C Segovia
Mol Ther advance online publication, September 15, 2009; doi:10.1038/mt.2009.200
Expanded Anticancer Therapeutic Window of Hexon-modified Oncolytic Adenovirus
Elena V Shashkova, Shannon M May, Konstantin Doronin and Michael A Barry
Mol Ther advance online publication, September 15, 2009; doi:10.1038/mt.2009.217
Suppression of FVIII Inhibitor Formation in Hemophilic Mice by Delivery of Transgene Modified Apoptotic Fibroblasts
Rui-Jun Su, Angela Epp, Yvette Latchman, Doug Bolgiano, Steven W Pipe and Neil C Josephson
Mol Ther advance online publication, September 15, 2009; doi:10.1038/mt.2009.209
September 8 2009
Original Articles
Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment
Marc T Abrams, Martin L Koser, Jessica Seitzer, Stephanie C Williams, Martha A DiPietro, Weimin Wang, Andrew W Shaw, Xianzhi Mao, Vasant Jadhav, Joseph P Davide, Paul A Burke, Alan B Sachs, Steven M Stirdivant and Laura Sepp-Lorenzino
Mol Ther advance online publication, September 8, 2009; doi:10.1038/mt.2009.208
Therapeutic and Tumor-specific Immunity Induced by Combination of Dendritic Cells and Oncolytic Adenovirus Expressing IL-12 and 4-1BBL MTOpen
Jing-Hua Huang, Song-Nan Zhang, Kyung-Ju Choi, Il-Kyu Choi, Joo-Hang Kim, Mingul Lee, Hoguen Kim and Chae-Ok Yun
Mol Ther advance online publication, September 8, 2009; doi:10.1038/mt.2009.205
Image-guided, Intravascular Hydrodynamic Gene Delivery to Skeletal Muscle in Pigs
Kenya Kamimura, Guisheng Zhang and Dexi Liu
Mol Ther advance online publication, September 8, 2009; doi:10.1038/mt.2009.206
IRES-based Vector Coexpressing FGF2 and Cyr61 Provides Synergistic and Safe Therapeutics of Lower Limb Ischemia MTOpen
Audrey Rayssac, Charles Neveu, Mélanie Pucelle, Loïc Van den Berghe, Leonel Prado-Lourenco, Jean-François Arnal, Xavier Chaufour and Anne-Catherine Prats
Mol Ther advance online publication, September 8, 2009; doi:10.1038/mt.2009.211
Systemic Delivery of Synthetic MicroRNA-16 Inhibits the Growth of Metastatic Prostate Tumors via Downregulation of Multiple Cell-cycle Genes
Fumitaka Takeshita, Lubna Patrawala, Mitsuhiko Osaki, Ryou-u Takahashi, Yusuke Yamamoto, Nobuyoshi Kosaka, Masaki Kawamata, Kevin Kelnar, Andreas G. Bader, David Brown and Takahiro Ochiya
Mol Ther advance online publication, September 8, 2009; doi:10.1038/mt.2009.207
September 1 2009
Original Articles
Activation of Transgene-specific T Cells Following Lentivirus-mediated Gene Delivery to Mouse Lung
Maria P Limberis, Christie L Bell, Jack Heath and James M Wilson
Mol Ther advance online publication, September 1, 2009; doi:10.1038/mt.2009.190
Induction of Tumor-specific Immune Response by Gene Transfer of Hsp70-cell-penetrating Peptide Fusion Protein to Tumors in Mice
Makiya Nishikawa, Takayuki Otsuki, Atsushi Ota, Xin Guan, Seiji Takemoto, Yuki Takahashi and Yoshinobu Takakura
Mol Ther advance online publication, September 1, 2009; doi:10.1038/mt.2009.203
Multiple Functions of the 37/67-kd Laminin Receptor Make It a Suitable Target for Novel Cancer Gene Therapy
Jonathan Scheiman, Jen-Chieh Tseng, Yun Zheng and Daniel Meruelo
Mol Ther advance online publication, September 1, 2009; doi:10.1038/mt.2009.199
August 18 2009
Original Articles
Preintegration HIV-1 Inhibition by a Combination Lentiviral Vector Containing a Chimeric TRIM5 Protein, a CCR5 shRNA, and a TAR Decoy
Joseph S Anderson, John Javien, Jan A Nolta and Gerhard Bauer
Mol Ther advance online publication, August 18, 2009; doi:10.1038/mt.2009.187
Genetic-induced Variations in the GAD65 T-cell Repertoire Governs Efficacy of Anti-CD3/GAD65 Combination Therapy in New-onset Type 1 Diabetes
Damien Bresson, Matthew Fradkin, Yulia Manenkova, Diane Rottembourg and Matthias von Herrath
Mol Ther advance online publication, August 18, 2009; doi:10.1038/mt.2009.197
Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease
Baodong Sun, Michael D Kulis, Sarah P Young, Amy C Hobeika, Songtao Li, Andrew Bird, Haoyue Zhang, Yifan Li, Timothy M Clay, Wesley Burks, Priya S Kishnani and Dwight D Koeberl
Mol Ther advance online publication, August 18, 2009; doi:10.1038/mt.2009.195
August 11 2009
Original Articles
Inner Limiting Membrane Barriers to AAV-mediated Retinal Transduction From the Vitreous
Deniz Dalkara, Kathleen D Kolstad, Natalia Caporale, Meike Visel, Ryan R Klimczak, David V Schaffer and John G Flannery
Mol Ther advance online publication, August 11, 2009; doi:10.1038/mt.2009.181
Deficiency of the Housekeeping Gene Hypoxanthine–Guanine Phosphoribosyltransferase (HPRT) Dysregulates Neurogenesis
Ghiabe-Henri Guibinga, Stephen Hsu and Theodore Friedmann
Mol Ther advance online publication, August 11, 2009; doi:10.1038/mt.2009.178
Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery
James T Koerber, Ryan Klimczak, Jae-Hyung Jang, Deniz Dalkara, John G Flannery and David V Schaffer
Mol Ther advance online publication, August 11, 2009; doi:10.1038/mt.2009.184
August 4 2009
Original Article
Adeno-associated Virus Gene Therapy With Cholesterol 24-Hydroxylase Reduces the Amyloid Pathology Before or After the Onset of Amyloid Plaques in Mouse Models of Alzheimer's Disease
Eloise Hudry, Debby Van Dam, Wim Kulik, Peter P De Deyn, Femke S Stet, Ornella Ahouansou, Abdellatif Benraiss, André Delacourte, Pierre Bougnères, Patrick Aubourg and Nathalie Cartier
Mol Ther advance online publication, August 4, 2009; doi:10.1038/mt.2009.175
July 28 2009
Original Articles
Cationic Lipid Formulations Alter the In Vivo Tropism of AAV2/9 Vector in Lung
David E Fein, Maria P Limberis, Sean F Maloney, Jack M Heath, James M Wilson and Scott L Diamond
Mol Ther advance online publication, July 28, 2009; doi:10.1038/mt.2009.173
Generating Differentially Targeted Amyloid- Specific Intrabodies as a Passive Vaccination Strategy for Alzheimer's Disease
Kelly L Sudol, Michael A Mastrangelo, Wade C Narrow, Maria E Frazer, Yona R Levites, Todd E Golde, Howard J Federoff and William J Bowers
Mol Ther advance online publication, July 28, 2009; doi:10.1038/mt.2009.174
July 21 2009
Original Articles
Design and Evaluation of Doxorubicin-containing Microbubbles for Ultrasound-triggered Doxorubicin Delivery: Cytotoxicity and Mechanisms Involved
Ine Lentacker, Bart Geers, Joseph Demeester, Stefaan C De Smedt and Niek N Sanders
Mol Ther advance online publication, July 21, 2009; doi:10.1038/mt.2009.160
Targeting a Dominant Negative Rho Kinase to Neurons Promotes Axonal Outgrowth and Partial Functional Recovery After Rat Rubrospinal Tract Lesion
Dongsheng Wu, Ping Yang, Xinyu Zhang, Juan Luo, Mohammed E Haque, John Yeh, Peter M Richardson, Yi Zhang and Xuenong Bo
Mol Ther advance online publication, July 21, 2009; doi:10.1038/mt.2009.168
July 14 2009
Original Article
Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium
Wuping Li, Liqun Zhang, Jarrod S Johnson, Wu Zhijian, Joshua C Grieger, Xiao Ping-Jie, Lauren M Drouin, Mavis Agbandje-McKenna, Raymond J Pickles and R Jude Samulski
Mol Ther advance online publication, July 14, 2009; doi:10.1038/mt.2009.155
