Open Access Articles

Genetics and Therapies for GM2 Gangliosidosis

, 2018; 18(2): 68 - 89

Maria Begona Cachon-Gonzalez*, Eva Zaccariotto and Timothy Martin Cox




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

, 2017; 17(6): 442 - 452

Quan Zhuang, Ke Cheng* and Yingzi Ming*





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

, 2017; 17(4): 309 - 319

Francoise Baylis* and Marcus McLeod





Tet-On Systems For Doxycycline-inducible Gene Expression

, 2016; 16(3): 156 - 167

Atze T. Das, Liliane Tenenbaum and Ben Berkhout




Gene Electrotransfer: A Mechanistic Perspective

, 2016; 16(2): 98 - 129

Christelle Rosazza, Sasa Haberl Meglic, Andreas Zumbusch, Marie-Pierre Rols and Damijan Miklavcic




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

, 2016; 16(1): 5 - 13

Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura, Noriko Mochizuki- Oda, Shigeo Masuda and Yoshiki Sawa





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

, 2015; 15(4): 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




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

, 2015; 15(1): 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




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

, 2015; 15(1): 3 - 14

Maria C. Subang, Rewas Fatah, Ying Wu, Drew Hannaman, Jason Rice, Claire F. Evans, Yuti Chernajovsky and David Gould





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

, 2014; 14(4): 309 - 329

Chi H. Sum, Shawn Wettig and Roderick A. Slavcev





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

, 2013; 13(6): 413 - 420

Martine Goossens, Katia Pauwels, Nicolas Willemarck and Didier Breyer





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

, 2013; 13(6): 385 - 394

Aline Baldo, Eric van den Akker, Hans E. Bergmans, Filip Lim and Katia Pauwels





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

, 2013; 13(5): 315 - 321

Munmun Chattopadhyay





Antibody Gene Therapy: Getting Closer to Clinical Application?

, 2013; 13(4): 282 - 290

Irene Guijarro-Munoz, Marta Compte, Luis Alvarez-Vallina and Laura Sanz





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

, 2013; 13(3): 182 - 188

Yong Cheng, Lvzhen Huang, Xiaoxin Li, Huijun Qi, Peng Zhou, Wenzhen Yu, Yide A. Jiang, Samuel Wadsworth and Abraham Scaria




Modelling Human Disease with Pluripotent Stem Cells

, 2013; 13(2): 99 - 110

Richard Siller, Sebastian Greenhough, In-Hyun Park and Gareth J. Sullivan




Technological Overview of iPS Induction from Human Adult Somatic Cells

, 2013; 13(2): 73 - 92

Emilie Bayart and Odile Cohen-Haguenauer




Human Pluripotent Stem Cells for Modelling Human Liver Diseases and Cell Therapy

, 2013; 13(2): 120 - 132

Noushin Dianat, Clara Steichen, Ludovic Vallier, Anne Weber and Anne Dubart-Kupperschmitt




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

, 2012; 12(6): 463 - 483

Kalliopi Domvri, Paul Zarogoulidis, Konstantinos Porpodis, Maria Koffa, Maria Lambropoulou, Stylianos Kakolyris, George Kolios, Konstantinos Zarogoulidis and Ekaterini Chatzaki





Retroviral Protein Transfer: Falling Apart to Make an Impact

, 2012; 12(5): 389 - 409

Tobias Maetzig, Christopher Baum and Axel Schambach





Exosomes and the Emerging Field of Exosome-Based Gene Therapy

, 2012; 12(4): 262 - 274

Aisling J. O’Loughlin, Caroline A. Woffindale and Matthew J.A. Wood





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

, 2012; 12(3): 152 - 160

Virginia Arechavala-Gomeza, Karen Anthony, Jennifer Morgan and Francesco Muntoni




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

, 2012; 12(2): 77 - 91

Andressa Ardiani, Adam J. Johnson, Hongmei Ruan, Marilyn Sanchez-Bonilla, Kinta Serve and Margaret E. Black




Viral and Non-Viral Methods to Genetically Modify Dendritic Cells

, 2012; 12(2): 127 - 136

Jean-Marc Humbert and Franck Halary




Novel Strategies to Improve DNA Vaccine Immunogenicity

, 2011; 11(6): 479 - 484

Cevayir Coban, Kouji Kobiyama, Taiki Aoshi, Fumihiko Takeshita, Toshihiro Horii, Shizuo Akira and Ken J. Ishii




Immune Responses to AAV in Clinical Trials

, 2011; 11(4): 321 - 330

Federico Mingozzi and Katherine A. High





Current Advances in Retroviral Gene Therapy

, 2011; 11(3): 218 - 228

Youngsuk Yi, Moon Jong Noh and Kwan Hee Lee





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

, 2010; 10(4): 287 - 299

Anita Gothelf and Julie Gehl




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

, 2009; 9(6): 459 - 474

Katia Pauwels, Rik Gijsbers, Jaan Toelen, Axel Schambach, Karen Willard-Gallo, Celine Verheust, Zeger Debyser and Philippe Herman




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

, 2009; 9(3): 160 - 170

Sander W. Tas, Margriet J.B.M. Vervoordeldonk and Paul P. Tak





Current Advances and Future Challenges in Adenoviral Vector Biology and Targeting

, 2007; 7(3): 189 - 204

Samuel K. Campos and Michael A. Barry




Amplicons as Vaccine Vectors

, 2006; 6(3): 383 - 392

Kathlyn Santos, Cindy M.P. Duke and Stephen Dewhurst




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

, 2006; 6(2): 243 - 273

Gerald J. Prud'homme, Yelena Glinka, Amir S. Khan and Ruxandra Draghia-Akli





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