Celebrating the 25th Anniversary of SELEX: An Aptamer Symposium
Supported by the American Society of Gene & Cell Therapy, the Aptamers Workshop will provide a unique opportunity to celebrate the 25th anniversary of aptamers and to meet researchers around the world developing nucleic acid aptamers as high affinity targeting reagents for both diagnosis and therapy.
Find out more: www.asgct.org/am15/ |  |
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EDITORIAL
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Molecular Therapy: At the Cutting Edge of Methodology and Clinical Development
Roland W Herzog
Mol Ther 2015 23: 409-410; 10.1038/mt.2015.23
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IN THIS ISSUE
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In This Issue
Mol Ther 2015 23: 412; 10.1038/mt.2015.24
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RESEARCH HIGHLIGHTS
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Research Highlights
Mol Ther 2015 23: 413; 10.1038/mt.2015.25
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ORIGINAL ARTICLES
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Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates
Jagdeep S Walia, Naderah Altaleb, Alexander Bello, Christa Kruck, Matthew C LaFave, Gaurav K Varshney, Shawn M Burgess, Biswajit Chowdhury, David Hurlbut, Richard Hemming, Gary P Kobinger and Barbara Triggs-Raine
Mol Ther 2015 23: 414-422; advance online publication, December 17, 2014; 10.1038/mt.2014.240
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Let-7 microRNAs Regenerate Peripheral Nerve Regeneration by Targeting Nerve Growth FactorMTOPEN
Shiying Li, Xinghui Wang, Yun Gu, Chu Chen, Yaxian Wang, Jie Liu, Wen Hu, Bin Yu, Yongjun Wang, Fei Ding, Yan Liu and Xiaosong Gu
Mol Ther 2015 23: 423-433; advance online publication, November 14, 2014; 10.1038/mt.2014.220
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Development of Novel Activin-Targeted Therapeutics
Justin L Chen, Kelly L Walton, Sara L Al-Musawi, Emily K Kelly, Hongwei Qian, Mylinh La, Louis Lu, George Lovrecz, Mark Ziemann, Ross Lazarus, Assam El-Osta, Paul Gregorevic and Craig A Harrison
Mol Ther 2015 23: 434-444; advance online publication, November 17, 2014; 10.1038/mt.2014.221
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In Vivo AAV1 Transduction With hRheb(S16H) Protects Hippocampal Neurons by BDNF Production
Min-Tae Jeon, Jin Han Nam, Won-Ho Shin, Eunju Leem, Kyoung Hoon Jeong, Un Ju Jung, Young-Seuk Bae, Young-Ho Jin, Nikolai Kholodilov, Robert E Burke, Seok-Geun Lee, Byung Kwan Jin and Sang Ryong Kim
Mol Ther 2015 23: 445-455; advance online publication, December 15, 2014; 10.1038/mt.2014.241
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Enzyme Enhancers for the Treatment of Fabry and Pompe Disease
Jan Lukas, Anne-Marie Pockrandt, Susanne Seemann, Muhammad Sharif, Franziska Runge, Susann Pohlers, Chaonan Zheng, Anne Gläser, Matthias Beller, Arndt Rolfs and Anne-Katrin Giese
Mol Ther 2015 23: 456-464; advance online publication, November 20, 2014; 10.1038/mt.2014.224
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A New Cell-penetrating Peptide That Blocks the Autoinhibitory XIP Domain of NCX1 and Enhances Antiporter Activity
Pasquale Molinaro, Anna Pannaccione, Maria José Sisalli, Agnese Secondo, Ornella Cuomo, Rossana Sirabella, Maria Cantile, Roselia Ciccone, Antonella Scorziello, Gianfranco di Renzo and Lucio Annunziato
Mol Ther 2015 23: 465-476; advance online publication, December 11, 2014; 10.1038/mt.2014.231
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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer, Lyndsey Braun, Vicki McGovern, Shibi Likhite, Olivia Michels, Alessandra Govoni, Julie Fitzgerald, Pablo Morales, Kevin D Foust, Jerry R Mendell, Arthur H M Burghes and Brian K Kaspar
Mol Ther 2015 23: 477-487; advance online publication, October 31, 2014; 10.1038/mt.2014.210
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Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid MutantsMTOPEN
Matthew J Benskey, Nathan C Kuhn, James J Galligan, Joanna Garcia, Shannon E Boye, William W Hauswirth, Christian Mueller, Sanford L Boye and Fredric P Manfredsson
Mol Ther 2015 23: 488-500; advance online publication, January 16, 2015; 10.1038/mt.2015.7
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Biological Ablation of Sentinel Lymph Node Metastasis in Submucosally Invaded Early Gastrointestinal Cancer
Satoru Kikuchi, Hiroyuki Kishimoto, Hiroshi Tazawa, Yuuri Hashimoto, Shinji Kuroda, Masahiko Nishizaki, Takeshi Nagasaka, Yasuhiro Shirakawa, Shunsuke Kagawa, Yasuo Urata, Robert M Hoffman and Toshiyoshi Fujiwara
Mol Ther 2015 23: 501-509; advance online publication, December 19, 2014; 10.1038/mt.2014.244
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Sourcing of an Alternative Pericyte-Like Cell Type from Peripheral Blood in Clinically Relevant Numbers for Therapeutic Angiogenic Applications
Anna Blocki, Yingting Wang, Maria Koch, Anna Goralczyk, Sebastian Beyer, Nikita Agarwal, Michelle Lee, Shehzahdi Moonshi, Jean-Yves Dewavrin, Priscilla Peh, Herbert Schwarz, Kishore Bhakoo and Michael Raghunath
Mol Ther 2015 23: 510-522; advance online publication, December 12, 2014; 10.1038/mt.2014.232
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Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger NucleasesMTOPEN
David G Ousterout, Ami M Kabadi, Pratiksha I Thakore, Pablo Perez-Pinera, Matthew T Brown, William H Majoros, Timothy E Reddy and Charles A Gersbach
Mol Ther 2015 23: 523-532; advance online publication, December 10, 2014; 10.1038/mt.2014.234
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Transplantation of Glial Progenitors That Overexpress Glutamate Transporter GLT1 Preserves Diaphragm Function Following Cervical SCI
Ke Li, Elham Javed, Tamara J Hala, Daniel Sannie, Kathleen A Regan, Nicholas J Maragakis, Megan C Wright, David J Poulsen and Angelo C Lepore
Mol Ther 2015 23: 533-548; advance online publication, December 10, 2014; 10.1038/mt.2014.236
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Mesenchymal Stem Cells Correct Inappropriate Epithelial–mesenchyme Relation in Pulmonary Fibrosis Using Stanniocalcin-1
Manabu Ono, Shinya Ohkouchi, Masahiko Kanehira, Naoki Tode, Makoto Kobayashi, Masahito Ebina, Toshihiro Nukiwa, Toshiya Irokawa, Hiromasa Ogawa, Takaaki Akaike, Yoshinori Okada, Hajime Kurosawa, Toshiaki Kikuchi and Masakazu Ichinose
Mol Ther 2015 23: 549-560; advance online publication, November 6, 2014; 10.1038/mt.2014.217
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Depletion of Bone Marrow CCSP-Expressing Cells Delays Airway Regeneration
Martha L Bustos, Marco Mura, David Hwang, Olga Ludkovski, Amy P Wong, Armand Keating and Thomas K Waddell
Mol Ther 2015 23: 561-569; advance online publication, November 20, 2014; 10.1038/mt.2014.223
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Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs
Cory Smith, Leire Abalde-Atristain, Chaoxia He, Brett R Brodsky, Evan M Braunstein, Pooja Chaudhari, Yoon-Young Jang, Linzhao Cheng and Zhaohui Ye
Mol Ther 2015 23: 570-577; advance online publication, November 24, 2014; 10.1038/mt.2014.226
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Functional Aspects of Intrahepatic Hepatitis B Virus-specific T Cells Induced by Therapeutic DNA VaccinationMTOPEN
Anette Brass, Lars Frelin, David R Milich, Matti Sällberg and Gustaf Ahlén
Mol Ther 2015 23: 578-590; advance online publication, December 10, 2014; 10.1038/mt.2014.233
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Synthetic Consensus HIV-1 DNA Induces Potent Cellular Immune Responses and Synthesis of Granzyme B, Perforin in HIV Infected Individuals
Matthew P Morrow, Pablo Tebas, Jian Yan, Lorenzo Ramirez, Anna Slager, Kim Kraynyak, Malissa Diehl, Divya Shah, Amir Khan, Jessica Lee, Jean Boyer, J Joseph Kim, Niranjan Y Sardesai, David B Weiner and Mark L Bagarazzi
Mol Ther 2015 23: 591-601; advance online publication, December 22, 2014; 10.1038/mt.2014.245
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Phase 1 Study of Intratumoral Pexa-Vec (JX-594), an Oncolytic and Immunotherapeutic Vaccinia Virus, in Pediatric Cancer Patients
Timothy P Cripe, Minhtran C Ngo, James I Geller, Chrystal U Louis, Mark A Currier, John M Racadio, Alexander J Towbin, Cliona M Rooney, Adina Pelusio, Anne Moon, Tae-Ho Hwang, James M Burke, John C Bell, David H Kirn and Caroline J Breitbach
Mol Ther 2015 23: 602-608; advance online publication, December 22, 2014; 10.1038/mt.2014.243
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Editorial
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