Tuesday, March 8, 2016

Molecular Therapy - Table of Contents alert Volume 24 Issue 3

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Molecular Therapy

TABLE OF CONTENTS

Volume 24, Issue 3 (March 2016)

In this issue
Editorial
In This Issue
Research Highlights
Commentaries
Review
Original Articles

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Editorial

Top

A New Induction to the Gene and Cell Therapy Hall Of Fame: Genome Editing

Michel Sadelain

Mol Ther 2016 24: 407-408; 10.1038/mt.2016.25

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In This Issue

Top

In This Issue

Mol Ther 2016 24: 410; 10.1038/mt.2016.26

Full Text

Research Highlights

Top

Research Highlights

Mol Ther 2016 24: 411; 10.1038/mt.2016.27

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Commentaries

Top

A Perspective on the State of Genome Editing

Dana Carroll

Mol Ther 2016 24: 412-413; 10.1038/mt.2016.28

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CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy

Thierry VandenDriessche and Marinee K Chuah

Mol Ther 2016 24: 414-416; 10.1038/mt.2016.29

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A CRISPR Approach for Reactivating Latent HIV-1

Angel Lin and Zachary Klase

Mol Ther 2016 24: 416-418; 10.1038/mt.2016.30

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Patent Law and Genome Engineering: A Short Guide to a Rapidly Changing Landscape

Ross Cloney

Mol Ther 2016 24: 419-421; 10.1038/mt.2016.31

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Review

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Salient Features of Endonuclease Platforms for Therapeutic Genome Editing

Michael T Certo and Richard A Morgan

Mol Ther 2016 24: 422-429; advance online publication, January 22, 2016; 10.1038/mt.2016.21

Abstract | Full Text

Genome-editing Technologies for Gene and Cell TherapyMTOPEN

Morgan L Maeder and Charles A Gersbach

Mol Ther 2016 24: 430-446; advance online publication, January 12, 2016; 10.1038/mt.2016.10

Abstract | Full Text

Engineered Viruses as Genome Editing DevicesMTOPEN

Xiaoyu Chen and Manuel A F V Gonçalves

Mol Ther 2016 24: 447-457; advance online publication, September 4, 2015; 10.1038/mt.2015.164

Abstract | Full Text

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

Thomas Gaj, Benjamin E Epstein and David V Schaffer

Mol Ther 2016 24: 458-464; advance online publication, September 16, 2015; 10.1038/mt.2015.151

Abstract | Full Text

Current Progress in Therapeutic Gene Editing for Monogenic Diseases

Versha Prakash, Marc Moore and Rafael J Yáñez-Muñoz

Mol Ther 2016 24: 465-474; advance online publication, January 14, 2016; 10.1038/mt.2016.5

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Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome EditingMTOPEN

Ciaran M Lee, Thomas J Cradick, Eli J Fine and Gang Bao

Mol Ther 2016 24: 475-487; advance online publication, January 11, 2016; 10.1038/mt.2016.1

Abstract | Full Text

Original Articles

Top

Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator ComplexMTOPEN

Sheena M Saayman, Daniel C Lazar, Tristan A Scott, Jonathan R Hart, Mayumi Takahashi, John C Burnett, Vicente Planelles, Kevin V Morris and Marc S Weinberg

Mol Ther 2016 24: 488-498; advance online publication, November 19, 2015; 10.1038/mt.2015.202

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CRISPR-mediated Activation of Latent HIV-1 Expression

Prajit Limsirichai, Thomas Gaj and David V Schaffer

Mol Ther 2016 24: 499-507; advance online publication, November 26, 2015; 10.1038/mt.2015.213

Abstract | Full Text

Specific Reactivation of Latent HIV-1 by dCas9-SunTag-VP64-mediated Guide RNA Targeting the HIV-1 Promoter

Haiyan Ji, Zhengtao Jiang, Panpan Lu, Li Ma, Chuan Li, Hanyu Pan, Zheng Fu, Xiying Qu, Pengfei Wang, Junxiao Deng, Xinyi Yang, Jianhua Wang and Huanzhang Zhu

Mol Ther 2016 24: 508-521; advance online publication, January 18, 2016; 10.1038/mt.2016.7

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CRISPR-Cas9 Can Inhibit HIV-1 Replication but NHEJ Repair Facilitates Virus EscapeMTOPEN

Gang Wang, Na Zhao, Ben Berkhout and Atze T Das

Mol Ther 2016 24: 522-526; advance online publication, January 22, 2016; 10.1038/mt.2016.24

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CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy

Charis L Himeda, Takako I Jones and Peter L Jones

Mol Ther 2016 24: 527-535; advance online publication, November 3, 2015; 10.1038/mt.2015.200

Abstract | Full Text

Re-expression of Selected Epigenetically Silenced Candidate Tumor Suppressor Genes in Cervical Cancer by TET2-directed Demethylation

Christian Huisman, Monique G P van der Wijst, Matthijs Schokker, Pilar Blancafort, Martijn M Terpstra, Klaas Kok, Ate G J van der Zee, Ed Schuuring, G Bea A Wisman and Marianne G Rots

Mol Ther 2016 24: 536-547; advance online publication, December 21, 2015; 10.1038/mt.2015.226

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Protein Delivery of an Artificial Transcription Factor Restores Widespread Ube3a Expression in an Angelman Syndrome Mouse BrainMTOPEN

Barbara J Bailus, Benjamin Pyles, Michelle M McAlister, Henriette O'Geen, Sarah H Lockwood, Alexa N Adams, Jennifer Trang T Nguyen, Abigail Yu, Robert F Berman and David J Segal

Mol Ther 2016 24: 548-555; advance online publication, January 4, 2016; 10.1038/mt.2015.236

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In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis PigmentosaMTOPEN

Benjamin Bakondi, Wenjian Lv, Bin Lu, Melissa K Jones, Yuchun Tsai, Kevin J Kim, Rachelle Levy, Aslam Abbasi Akhtar, Joshua J Breunig, Clive N Svendsen and Shaomei Wang

Mol Ther 2016 24: 556-563; advance online publication, December 15, 2015; 10.1038/mt.2015.220

Abstract | Full Text

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

Li Xu, Ki Ho Park, Lixia Zhao, Jing Xu, Mona El Refaey, Yandi Gao, Hua Zhu, Jianjie Ma and Renzhi Han

Mol Ther 2016 24: 564-569; advance online publication, October 9, 2015; 10.1038/mt.2015.192

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Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

Mark J Osborn, Beau R Webber, Friederike Knipping, Cara-lin Lonetree, Nicole Tennis, Anthony P DeFeo, Amber N McElroy, Colby G Starker, Catherine Lee, Sarah Merkel, Troy C Lund, Karen S Kelly-Spratt, Michael C Jensen, Daniel F Voytas, Christof von Kalle, Manfred Schmidt, Richard Gabriel, Keli L Hippen, Jeffrey S Miller, Andrew M Scharenberg, Jakub Tolar and Bruce R Blazar

Mol Ther 2016 24: 570-581; advance online publication, October 27, 2015; 10.1038/mt.2015.197

Abstract | Full Text

Silent IL2RG Gene Editing in Human Pluripotent Stem Cells

Li B Li, Chao Ma, Geneve Awong, Marion Kennedy, German Gornalusse, Gordon Keller, Dan S Kaufman and David W Russell

Mol Ther 2016 24: 582-591; advance online publication, October 7, 2015; 10.1038/mt.2015.190

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Genome-wide Profiling Reveals Remarkable Parallels Between Insertion Site Selection Properties of the MLV Retrovirus and the piggyBac Transposon in Primary Human CD4+ T CellsMTOPEN

Andreas Gogol-Döring, Ismahen Ammar, Saumyashree Gupta, Mario Bunse, Csaba Miskey, Wei Chen, Wolfgang Uckert, Thomas F Schulz, Zsuzsanna Izsvák and Zoltán Ivics

Mol Ther 2016 24: 592-606; advance online publication, January 12, 2016; 10.1038/mt.2016.11

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Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human CellsMTOPEN

Jaichandran Sivalingam, Dimitar Kenanov, Hao Han, Ajit Johnson Nirmal, Wai Har Ng, Sze Sing Lee, Jeyakumar Masilamani, Toan Thang Phan, Sebastian Maurer-Stroh and Oi Lian Kon

Mol Ther 2016 24: 607-619; advance online publication, December 22, 2015; 10.1038/mt.2015.223

Abstract | Full Text

Sleeping Beauty Transposon Vectors in Liver-directed Gene Delivery of LDLR and VLDLR for Gene Therapy of Familial Hypercholesterolemia

Tytteli A K Turunen, Jere Kurkipuro, Tommi Heikura, Taina Vuorio, Elisa Hytönen, Zsuzsanna Izsvák and Seppo Ylä-Herttuala

Mol Ther 2016 24: 620-635; advance online publication, December 16, 2015; 10.1038/mt.2015.221

Abstract | Full Text

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

Maximilian Müller, Ciaran M Lee, Giedrius Gasiunas, Timothy H Davis, Thomas J Cradick, Virginijus Siksnys, Gang Bao, Toni Cathomen and Claudio Mussolino

Mol Ther 2016 24: 636-644; advance online publication, December 14, 2015; 10.1038/mt.2015.218

Abstract | Full Text

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian CellsMTOPEN

Ciaran M Lee, Thomas J Cradick and Gang Bao

Mol Ther 2016 24: 645-654; advance online publication, January 19, 2016; 10.1038/mt.2016.8

Abstract | Full Text

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