Molecular Therapy - Table of Contents alert Volume 23 Issue 3

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TABLE OF CONTENTS Volume 23, Issue 3 (March 2015) In this issue Editorial In This Issue Research Highlights Original Articles Also new AOP Sign up for e-alerts Recommend to your library Web feed Subscribe Advertisement 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/   Editorial Top 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 Full Text In This Issue Top In This Issue Mol Ther 2015 23: 412; 10.1038/mt.2015.24 Full Text Research Highlights Top Research Highlights Mol Ther 2015 23: 413; 10.1038/mt.2015.25 Full Text Original Articles Top 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text 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 Abstract | Full Text Please note that you need to be a subscriber or site-license holder to enjoy full-text access to Molecular Therapy. 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