Current Gene Therapy

Editor-in-Chief:
Lung-Ji Chang
University of Florida
Gainesville, FL
USA

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Open Access Articles

Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy open Access plus

Volume: 19   Issue: 3
Pp: 197-207
Angela L. McCall*, Sylvia G. Stankov, Gabrielle Cowen, Denise Cloutier, Zizhao Zhang, Lin Yang, Nathalie Clement, Darin J. Falk and Barry J. Byrne*
DOI:10.2174/1566523219666190621113807

Three Cases of Leber’s Hereditary Optic Neuropathy with Rapid Increase in Visual Acuity After Gene Therapy open Access plus

Volume: 19   Issue: 2
Pp: 134-138
Yong Zhang, Jia-jia Yuan, Hong-li Liu, Zhen Tian, Si-wei Liu and Bin Li*
DOI:10.2174/1566523219666190618094505

Functional Improvement of Chimeric Antigen Receptor Through Intrinsic Interleukin-15Rα Signaling open Access plus

Volume: 19   Issue: 1
Pp: 40-53
Sushmita Nair, Jing-Bo Wang, Shih-Ting Tsao, Yuchen Liu, Wei Zhu, William B. Slayton, Jan S. Moreb, Lujia Dong and Lung-Ji Chang*
DOI:10.2174/1566523218666181116093857

Nanomedicine for Gene Delivery for the Treatment of Cardiovascular Diseases open Access plus

Volume: 19   Issue: 1
Pp: 20-30
Cen Yan, Xiao-Jiang Quan and Ying-Mei Feng*
DOI:10.2174/1566523218666181003125308

Clinical Observation of Patients with Leber’s Hereditary Optic Neuropathy Before Gene Therapy open Access plus

Volume: 18   Issue: 6
Pp: 386-392
Jiajia Yuan, Yong Zhang, Hongli Liu, Zhen Tian, Xin Li, Yichen Zheng, Qin Gao, Lin Song, Xiao Xiao, Jian Sun, Zhitao Wang and Bin Li*
DOI:10.2174/1566523218666181105125245

Clinical-Grade Oncolytic Adenovirus Purification Using Polysorbate 20 as an Alternative for Cell Lysis open Access plus

Volume: 18   Issue: 6
Pp: 366-374
Mafalda G. Moleirinho, Sara Rosa, Manuel J.T. Carrondo*, Ricardo J.S. Silva, Åsa Hagner-McWhirter, Gustaf Ahlén, Mats Lundgren, Paula M. Alves and Cristina Peixoto
DOI:10.2174/1566523218666181109141257

Emerging Therapeutic Approaches for Diamond Blackfan Anemia open Access plus

Volume: 18   Issue: 6
Pp: 327-335
Anna Aspesi*, Chiara Borsotti and Antonia Follenzi*
DOI:10.2174/1566523218666181109124538

EZH2 RIP-seq Identifies Tissue-specific Long Non-coding RNAs open Access plus

Volume: 18   Issue: 5
Pp: 275-285
Yan Wang, Yinping Xie, Lili Li, Yuan He, Di Zheng, Pengcheng Yu, Ling Yu, Lixu Tang, Yibin Wang* and Zhihua Wang*
DOI:10.2174/1566523218666181008125010

Human Disease System Biology open Access plus

Volume: 18   Issue: 5
Pp: 255-256
Liang Cheng and Yang Hu
DOI:10.2174/1566523218666181010101114

Oncolytic Virotherapy for Breast Cancer Treatment open Access plus

Volume: 18   Issue: 4
Pp: 192-205
Samia M. O`Bryan and J. Michael Mathis*
DOI:10.2174/1566523218666180910163805

Genetics and Therapies for GM2 Gangliosidosis open Access plus

Volume: 18   Issue: 2
Pp: 68-89
Maria Begona Cachon-Gonzalez*, Eva Zaccariotto and Timothy Martin Cox
DOI:10.2174/1566523218666180404162622

CX3CL1/CX3CR1 Axis, as the Therapeutic Potential in Renal Diseases: Friend or Foe? open Access plus

Volume: 17   Issue: 6
Pp: 442-452
Quan Zhuang, Ke Cheng* and Yingzi Ming*
DOI:10.2174/1566523218666180214092536

Molecular Adjuvants Based on Plasmids Encoding Protein Aggregation Domains Affect Bone Marrow Niche Homeostasis open Access plus

Volume: 17   Issue: 5
Pp: 391-397
Maria Giovanna Sabbieti, Giovanna Lacava, Andrea Amaroli, Luigi Marchetti, Roberta Censi, Piera Di Martino and Dimitrios Agas*
DOI:10.2174/1566523218666180105122626

The Progress of Gene Therapy for Leber's Optic Hereditary Neuropathy open Access plus

Volume: 17   Issue: 4
Pp: 320-326
Yong Zhang, Zhen Tian, Jiajia Yuan, Chang Liu, Hong li Liu, Si Qi Ma and Bin Li*
DOI:10.2174/1566523218666171129204926

First-in-human Phase 1 CRISPR Gene Editing Cancer Trials: Are We Ready? open Access plus

Volume: 17   Issue: 4
Pp: 309-319
Francoise Baylis* and Marcus McLeod
DOI:10.2174/1566523217666171121165935

Tet-On Systems For Doxycycline-inducible Gene Expression open Access plus

Volume: 16   Issue: 3
Pp: 156-167
Atze T. Das, Liliane Tenenbaum and Ben Berkhout
DOI:10.2174/1566523216666160524144041

Gene Electrotransfer: A Mechanistic Perspective open Access plus

Volume: 16   Issue: 2
Pp: 98-129
Christelle Rosazza, Sasa Haberl Meglic, Andreas Zumbusch, Marie-Pierre Rols and Damijan Miklavcic
DOI:10.2174/1566523216666160331130040

Building A New Treatment For Heart Failure-Transplantation of Induced Pluripotent Stem Cell-derived Cells into the Heart open Access plus

Volume: 16   Issue: 1
Pp: 5-13
Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura, Noriko Mochizuki- Oda, Shigeo Masuda and Yoshiki Sawa
DOI:10.2174/1566523216666160119094143

A Simple High Efficiency Intra-Islet Transduction Protocol Using Lentiviral Vectors open Access plus

Volume: 15   Issue: 4
Pp: 436-446
Carmen Maria Jimenez-Moreno, Irene de Gracia Herrera-Gomez, Livia Lopez-Noriega, Petra Isabel Lorenzo, Nadia Cobo-Vuilleumier, Esther Fuente-Martin, Jose Manuel Mellado-Gil, Geraldine Parnaud, Domenico Bosco, Benoit Raymond Gauthier and Alejandro Martin-Montalvo
DOI:10.2174/1566523215666150630121557

Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease open Access plus

Volume: 15   Issue: 1
Pp: 64-81
Olivier Negre, Cynthia Bartholomae, Yves Beuzard, Marina Cavazzana, Lauryn Christiansen, Celine Courne, Annette Deichmann, Maria Denaro, Edouard de Dreuzy, Mitchell Finer, Raffaele Fronza, Beatrix Gillet-Legrand, Christophe Joubert, Robert Kutner, Philippe Leboulch, Leila Maouche, Anais Paulard, Francis J. Pierciey, Michael Rothe, Byoung Ryu, Manfred Schmidt, Christof von Kalle, Emmanuel Payen and Gabor Veres
DOI:10.2174/1566523214666141127095336

Effects of APC De-Targeting and GAr Modification on the Duration of Luciferase Expression from Plasmid DNA Delivered to Skeletal Muscle open Access plus

Volume: 15   Issue: 1
Pp: 3-14
Maria C. Subang, Rewas Fatah, Ying Wu, Drew Hannaman, Jason Rice, Claire F. Evans, Yuti Chernajovsky and David Gould
DOI:10.2174/1566523214666141114204943

Gene/Cell Therapy Approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome open Access plus

Volume: 14   Issue: 6
Pp: 422-428
Laura Passerini, Francesca R. Santoni de Sio, Matthew H. Porteus and Rosa Bacchetta
DOI:10.2174/1566523214666141001123828

Impact of DNA Vector Topology on Non-Viral Gene Therapeutic Safety and Efficacy open Access plus

Volume: 14   Issue: 4
Pp: 309-329
Chi H. Sum, Shawn Wettig and Roderick A. Slavcev
DOI:10.2174/1566523214666140612154929

A Hantavirus Pulmonary Syndrome (HPS) DNA Vaccine Delivered Using a Spring-powered Jet Injector Elicits a Potent Neutralizing Antibody Response in Rabbits and Nonhuman Primates open Access plus

Volume: 14   Issue: 3
Pp: 200-210
Steve Kwilas, Jennifer M. Kishimori, Matthew Josleyn, Kurt Jerke, John Ballantyne, Michael Royals and Jay W. Hooper
DOI:10.2174/1566523214666140522122633

Environmental Risk Assessment of Clinical Trials Involving Modified Vaccinia Virus Ankara (MVA)-Based Vectors open Access plus

Volume: 13   Issue: 6
Pp: 413-420
Martine Goossens, Katia Pauwels, Nicolas Willemarck and Didier Breyer
DOI:10.2174/156652321306140103221941

Environmental Risk Assessment of Replication Competent Viral Vectors Applied in Clinical Trials: Potential Effects of Inserted Sequences open Access plus

Volume: 13   Issue: 6
Pp: 395-412
Eric van den Akker, Cecile J.B. van der Vlugt, Diederik A. Bleijs and Hans E. Bergmans
DOI:10.2174/156652321306140103221621

General Considerations on the Biosafety of Virus-derived Vectors Used in Gene Therapy and Vaccination open Access plus

Volume: 13   Issue: 6
Pp: 385-394
Aline Baldo, Eric van den Akker, Hans E. Bergmans, Filip Lim and Katia Pauwels
DOI:10.2174/15665232113136660005

Helper Dependent Adenovirus Vectors: Progress and Future Prospects open Access plus

Volume: 13   Issue: 5
Pp: 370-381
Dan Cots, Assumpció Bosch and Miguel Chillón
DOI:10.2174/156652321305131212125338

Targeted Delivery of Growth Factors by HSV-Mediated Gene Transfer for Peripheral Neuropathy open Access plus

Volume: 13   Issue: 5
Pp: 315-321
Munmun Chattopadhyay
DOI:10.2174/156652321305131212123237

Genome Engineering with TAL-Effector Nucleases and Alternative Modular Nuclease Technologies open Access plus

Volume: 13   Issue: 4
Pp: 291-303
Andrew M. Scharenberg, Philippe Duchateau and Julianne Smith
DOI:10.2174/15665232113139990026

Antibody Gene Therapy: Getting Closer to Clinical Application? open Access plus

Volume: 13   Issue: 4
Pp: 282-290
Irene Guijarro-Munoz, Marta Compte, Luis Alvarez-Vallina and Laura Sanz
DOI:10.2174/15665232113139990025

Insertional Mutagenesis by Retroviral Vectors: Current Concepts and Methods of Analysis open Access plus

Volume: 13   Issue: 3
Pp: 211-227
Sean Knight, Mary Collins and Yasuhiro Takeuchi
DOI:10.2174/1566523211313030006

Prevalence of Neutralizing Factors Against Adeno-Associated Virus Types 2 in Age-Related Macular Degeneration and Polypoidal Choroidal Vasculopathy open Access plus

Volume: 13   Issue: 3
Pp: 182-188
Yong Cheng, Lvzhen Huang, Xiaoxin Li, Huijun Qi, Peng Zhou, Wenzhen Yu, Yide A. Jiang, Samuel Wadsworth and Abraham Scaria
DOI:10.2174/1566523211313030003

Modelling Human Disease with Pluripotent Stem Cells open Access plus

Volume: 13   Issue: 2
Pp: 99-110
Richard Siller, Sebastian Greenhough, In-Hyun Park and Gareth J. Sullivan
DOI:10.2174/1566523211313020004

Technological Overview of iPS Induction from Human Adult Somatic Cells open Access plus

Volume: 13   Issue: 2
Pp: 73-92
Emilie Bayart and Odile Cohen-Haguenauer
DOI:10.2174/1566523211313020002

Human Pluripotent Stem Cells for Modelling Human Liver Diseases and Cell Therapy open Access plus

Volume: 13   Issue: 2
Pp: 120-132
Noushin Dianat, Clara Steichen, Ludovic Vallier, Anne Weber and Anne Dubart-Kupperschmitt
DOI:10.2174/1566523211313020006

Gene Therapy in Liver Diseases: State-of-the-Art and Future Perspectives open Access plus

Volume: 12   Issue: 6
Pp: 463-483
Kalliopi Domvri, Paul Zarogoulidis, Konstantinos Porpodis, Maria Koffa, Maria Lambropoulou, Stylianos Kakolyris, George Kolios, Konstantinos Zarogoulidis and Ekaterini Chatzaki
DOI:10.2174/156652312803519788

Development of Sendai Virus Vectors and their Potential Applications in Gene Therapy and Regenerative Medicine open Access plus

Volume: 12   Issue: 5
Pp: 410-416
Mahito Nakanishi and Makoto Otsu
DOI:10.2174/156652312802762518

Retroviral Protein Transfer: Falling Apart to Make an Impact open Access plus

Volume: 12   Issue: 5
Pp: 389-409
Tobias Maetzig, Christopher Baum and Axel Schambach
DOI:10.2174/156652312802762581

MicroRNAs and the Warburg Effect: New Players in an Old Arena open Access plus

Volume: 12   Issue: 4
Pp: 285-291
Ping Gao, Linchong Sun, Xiaoping He, Yang Cao and Huafeng Zhang
DOI:10.2174/156652312802083620

Exosomes and the Emerging Field of Exosome-Based Gene Therapy open Access plus

Volume: 12   Issue: 4
Pp: 262-274
Aisling J. O’Loughlin, Caroline A. Woffindale and Matthew J.A. Wood
DOI:10.2174/156652312802083594

Splicing Modulation Mediated by Small Nuclear RNAs as Therapeutic Approaches for Muscular Dystrophies open Access plus

Volume: 12   Issue: 3
Pp: 179-191
Rachid Benchaouir and Aurelie Goyenvalle
DOI:10.2174/156652312800840586

Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges open Access plus

Volume: 12   Issue: 3
Pp: 152-160
Virginia Arechavala-Gomeza, Karen Anthony, Jennifer Morgan and Francesco Muntoni
DOI:10.2174/156652312800840621

Enzymes To Die For: Exploiting Nucleotide Metabolizing Enzymes for Cancer Gene Therapy open Access plus

Volume: 12   Issue: 2
Pp: 77-91
Andressa Ardiani, Adam J. Johnson, Hongmei Ruan, Marilyn Sanchez-Bonilla, Kinta Serve and Margaret E. Black
DOI:10.2174/156652312800099571

Viral and Non-Viral Methods to Genetically Modify Dendritic Cells open Access plus

Volume: 12   Issue: 2
Pp: 127-136
Jean-Marc Humbert and Franck Halary
DOI:10.2174/156652312800099580

Novel Strategies to Improve DNA Vaccine Immunogenicity open Access plus

Volume: 11   Issue: 6
Pp: 479-484
Cevayir Coban, Kouji Kobiyama, Taiki Aoshi, Fumihiko Takeshita, Toshihiro Horii, Shizuo Akira and Ken J. Ishii
DOI:10.2174/156652311798192815

Immune Responses to AAV in Clinical Trials open Access plus

Volume: 11   Issue: 4
Pp: 321-330
Federico Mingozzi and Katherine A. High
DOI:10.2174/156652311796150354

Advances and Future Challenges in Adenoviral Vector Pharmacology and Targeting open Access plus

Volume: 11   Issue: 4
Pp: 241-258
Reeti Khare, Christopher Y. Chen, Eric A. Weaver and Michael A. Barry
DOI:10.2174/156652311796150363

Current Advances in Retroviral Gene Therapy open Access plus

Volume: 11   Issue: 3
Pp: 218-228
Youngsuk Yi, Moon Jong Noh and Kwan Hee Lee
DOI:10.2174/156652311795684740

Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65 open Access plus

Volume: 10   Issue: 5
Pp: 341-349
J. Fraser Wright, Jennifer Wellman and Katherine A. High
DOI:10.2174/156652310793180715

Gene Electrotransfer to Skin; Review of Existing Literature and Clinical Perspectives open Access plus

Volume: 10   Issue: 4
Pp: 287-299
Anita Gothelf and Julie Gehl
DOI:10.2174/156652310791823443

State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations open Access plus

Volume: 9   Issue: 6
Pp: 459-474
Katia Pauwels, Rik Gijsbers, Jaan Toelen, Axel Schambach, Karen Willard-Gallo, Celine Verheust, Zeger Debyser and Philippe Herman
DOI:10.2174/156652309790031120

Gene Therapy Targeting Nuclear Factor-κB: Towards Clinical Application in Inflammatory Diseases and Cancer open Access plus

Volume: 9   Issue: 3
Pp: 160-170
Sander W. Tas, Margriet J.B.M. Vervoordeldonk and Paul P. Tak
DOI:10.2174/156652309788488569

Immune Responses to AAV in Clinical Trials open Access plus

Volume: 7   Issue: 5
Pp: 316-324
Federico Mingozzi and Katherine A. High
DOI:10.2174/156652307782151425

Current Advances and Future Challenges in Adenoviral Vector Biology and Targeting open Access plus

Volume: 7   Issue: 3
Pp: 189-204
Samuel K. Campos and Michael A. Barry
DOI:10.2174/156652307780859062

Amplicons as Vaccine Vectors open Access plus

Volume: 6   Issue: 3
Pp: 383-392
Kathlyn Santos, Cindy M.P. Duke and Stephen Dewhurst
DOI:10.2174/156652306777592009

Electroporation-Enhanced Nonviral Gene Transfer for the Prevention or Treatment of Immunological, Endocrine and Neoplastic Diseases open Access plus

Volume: 6   Issue: 2
Pp: 243-273
Gerald J. Prud'homme, Yelena Glinka, Amir S. Khan and Ruxandra Draghia-Akli
DOI:10.2174/156652306776359504