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Molecular Therapy - Table of Contents alert Volume 24 Issue 5

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Molecular Therapy

TABLE OF CONTENTS

Volume 24, Issue 5 (May 2016)

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Editorial
In This Issue
Research Highlights
Commentaries
Original Articles

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Editorial

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Overcoming Challenges in CAR T-cell Product CGMP Release

Concetta Quintarelli, Franco Locatelli, Ignazio Caruana and Biagio De Angelis

Mol Ther 2016 24: 845-846; 10.1038/mt.2016.72

Full Text

In This Issue

Top

In This Issue

Mol Ther 2016 24: 847; 10.1038/mt.2016.73

Full Text

Research Highlights

Top

Research Highlights

Mol Ther 2016 24: 848; 10.1038/mt.2016.74

Full Text

Commentaries

Top

Multistage Delivery Technologies: Multifunctional, Interdisciplinary Approaches to Nanomedicine

Matthew T Haynes and Leaf Huang

Mol Ther 2016 24: 849-851; 10.1038/mt.2016.75

Full Text

Endogenous Transposase Source in Human Cells Mobilizes piggyBac Transposons

Zoltán Ivics

Mol Ther 2016 24: 851-854; 10.1038/mt.2016.76

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Original Articles

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Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Monir Shababi, Zhihua Feng, Eric Villalon, Christine M Sibigtroth, Erkan Y Osman, Madeline R Miller, Patricka A Williams-Simon, Abby Lombardi, Thalia H Sass, Arleigh K Atkinson, Michael L Garcia, Chien-Ping Ko and Christian L Lorson

Mol Ther 2016 24: 855-866; advance online publication, February 10, 2016; 10.1038/mt.2016.33

Abstract | Full Text

AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs

Virginie Pichard, Nathalie Provost, Alexandra Mendes-Madeira, Lyse Libeau, Philippe Hulin, Kizito-Tshitoko Tshilenge, Marine Biget, Baptiste Ameline, Jack-Yves Deschamps, Michel Weber, Guylène Le Meur, Marie-Anne Colle, Philippe Moullier and Fabienne Rolling

Mol Ther 2016 24: 867-876; advance online publication, February 9, 2016; 10.1038/mt.2016.37

Abstract | Full Text

Gene Transfer of Brain-derived Neurotrophic Factor (BDNF) Prevents Neurodegeneration Triggered by FXN Deficiency

Yurika Katsu-Jiménez, Frida Loría, Juan Carlos Corona and Javier Díaz-Nido

Mol Ther 2016 24: 877-889; advance online publication, February 5, 2016; 10.1038/mt.2016.32

Abstract | Full Text

Development and Validation of a Small Single-domain Antibody That Effectively Inhibits Matrix Metalloproteinase 8

Delphine Demeestere, Eline Dejonckheere, Sophie Steeland, Paco Hulpiau, Jurgen Haustraete, Nick Devoogdt, Rielana Wichert, Christoph Becker-Pauly, Elien Van Wonterghem, Sylviane Dewaele, Griet Van Imschoot, Jeroen Aerts, Lutgarde Arckens, Yvan Saeys, Claude Libert and Roosmarijn E Vandenbroucke

Mol Ther 2016 24: 890-902; advance online publication, January 18, 2016; 10.1038/mt.2016.2

Abstract | Full Text

SSRP1 Contributes to the Malignancy of Hepatocellular Carcinoma and Is Negatively Regulated by miR-497MTOPEN

Qianshan Ding, Ke He, Tao Luo, Yunchao Deng, Hanning Wang, Hao Liu, Jinqian Zhang, Kaiyun Chen, Jinfeng Xiao, Xiaopeng Duan, Rui Huang, Zhenglin Xia, Wenjie Zhou, Jinliang He, Honggang Yu, Xingyuan Jiao and Guoan Xiang

Mol Ther 2016 24: 903-914; advance online publication, January 12, 2016; 10.1038/mt.2016.9

Abstract | Full Text

Chop Deficiency Protects Mice Against Bleomycin-induced Pulmonary Fibrosis by Attenuating M2 Macrophage Production

Yingying Yao, Yi Wang, Zhijun Zhang, Long He, Jianghui Zhu, Meng Zhang, Xiaoyu He, Zhenshun Cheng, Qilin Ao, Yong Cao, Ping Yang, Yunchao Su, Jianping Zhao, Shu Zhang, Qilin Yu, Qin Ning, Xudong Xiang, Weining Xiong, Cong-Yi Wang and Yongjian Xu

Mol Ther 2016 24: 915-925; advance online publication, February 17, 2016; 10.1038/mt.2016.36

Abstract | Full Text

Inhibiting Notch Activity in Breast Cancer Stem Cells by Glucose Functionalized Nanoparticles Carrying γ-secretase InhibitorsMTOPEN

Veronika Mamaeva, Rasmus Niemi, Michaela Beck, Ezgi Özliseli, Diti Desai, Sebastian Landor, Tove Gronroos, Pauliina Kronqvist, Ina K N Pettersen, Emmet McCormack, Jessica M Rosenholm, Mika Linden and Cecilia Sahlgren

Mol Ther 2016 24: 926-936; advance online publication, February 26, 2016; 10.1038/mt.2016.42

Abstract | Full Text

MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular AtrophyMTOPEN

Naemeh Pourshafie, Philip R Lee, Ke-lian Chen, George G Harmison, Laura C Bott, Masahisa Katsuno, Gen Sobue, Barrington G Burnett, Kenneth H Fischbeck and Carlo Rinaldi

Mol Ther 2016 24: 937-945; advance online publication, January 12, 2016; 10.1038/mt.2016.13

Abstract | Full Text

Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA MimicsMTOPEN

Masayuki Matsui, Thazha P Prakash and David R Corey

Mol Ther 2016 24: 946-955; advance online publication, February 23, 2016; 10.1038/mt.2016.39

Abstract | Full Text

Induced Apoptosis Investigation in Wild-type and FLT3-ITD Acute Myeloid Leukemia Cells by Nanochannel Electroporation and Single-cell qRT-PCR

Keliang Gao, Xiaomeng Huang, Chi-Ling Chiang, Xinmei Wang, Lingqian Chang, Pouyan Boukany, Guido Marcucci, Robert Lee and Ly James Lee

Mol Ther 2016 24: 956-964; advance online publication, January 19, 2016; 10.1038/mt.2016.6

Abstract | Full Text

Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington’s Disease Mouse ModelsMTOPEN

Kari Pollock, Heather Dahlenburg, Haley Nelson, Kyle D Fink, Whitney Cary, Kyle Hendrix, Geralyn Annett, Audrey Torrest, Peter Deng, Joshua Gutierrez, Catherine Nacey, Karen Pepper, Stefanos Kalomoiris, Johnathon D Anderson, Jeannine McGee, William Gruenloh, Brian Fury, Gerhard Bauer, Alexandria Duffy, Theresa Tempkin, Vicki Wheelock and Jan A Nolta

Mol Ther 2016 24: 965-977; advance online publication, January 14, 2016; 10.1038/mt.2016.12

Abstract | Full Text

A Role for the Long Noncoding RNA SENCR in Commitment and Function of Endothelial CellsMTOPEN

Mounia Boulberdaa, Elizabeth Scott, Margaret Ballantyne, Raquel Garcia, Betty Descamps, Gianni D Angelini, Mairi Brittan, Amanda Hunter, Martin McBride, John McClure, Joseph M Miano, Costanza Emanueli, Nicholas L Mills, Joanne C Mountford and Andrew H Baker

Mol Ther 2016 24: 978-990; advance online publication, February 22, 2016; 10.1038/mt.2016.41

Abstract | Full Text

H7N9 Live Attenuated Influenza Vaccine Is Highly Immunogenic, Prevents Virus Replication, and Protects Against Severe Bronchopneumonia in Ferrets

Jørgen de Jonge, Irina Isakova-Sivak, Harry van Dijken, Sanne Spijkers, Justin Mouthaan, Rineke de Jong, Tatiana Smolonogina, Paul Roholl and Larisa Rudenko

Mol Ther 2016 24: 991-1002; advance online publication, January 22, 2016; 10.1038/mt.2016.23

Abstract | Full Text

Development of an Interleukin-1β Vaccine in Patients with Type 2 Diabetes

Claudia Cavelti-Weder, Katharina Timper, Eleonora Seelig, Cornelia Keller, Martin Osranek, Ute Lässing, Gunther Spohn, Patrik Maurer, Philipp Müller, Gary T Jennings, Joerg Willers, Philippe Saudan, Marc Y Donath and Martin F Bachmann

Mol Ther 2016 24: 1003-1012; advance online publication, December 21, 2015; 10.1038/mt.2015.227

Abstract | Full Text

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