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Wednesday, November 9, 2016
Call For Paper
Bentham Science Publishers would like to invite you to submit your research paper for publishing in the Journal of
Current Gene Therapy
Current Gene Therapy
Wednesday, November 2, 2016
Highlighted Article: Seq and You Will Find
Seq and You Will Find
[ Vol. 16 , Issue. 3 ]
Author(s):
Nicole Schonrock, Nicky Jonkhout and John S. Mattick Pages 220 - 229 ( 10 )
Abstract:
The human genome sequence is freely available, nearly complete and is providing a foundation of research opportunities that are overturning our current understanding of human biology. The advent of next generation sequencing has revolutionized the way we can interrogate the genome and its transcriptional products and how we analyze, diagnose, monitor and even treat human disease. Personal genetic profiles are increasing dramatically in medical value as researchers accumulate more and more knowledge about the interaction between genetic and environmental factors that contribute to the onset of common disorders. As the cost of sequencing plummets, whole genome sequencing of individuals is becoming a reality and the field of personalized genomic medicine is rapidly developing. Now there is great need for accurate annotation of all functionally important sequences in the human genome and the variations within them that contribute to health and disease. The vast majority of our genome gives rise to RNA transcripts. This extraordinarily versatile molecule not only encodes protein information but also has great structural dynamics and plasticity, capacity for DNA/RNA/protein interactions and catalytic activity. It is a key regulator of biological networks with clear links to human disease and a more comprehensive understanding of its function is needed to maximise its use in medical practice. This review focuses on the complexity of our genome and the impact of sequencing technologies in understanding its many products and functions in health and disease.
Keywords:
Next generation sequencing, Non-coding RNA, GWAS, Personalized medicine, Capture-sequencing.
Affiliation:
Garvan Institute of Medical Research, Darlinghurst, 2010, NSW, Australia.
For More Information Please Visit Our Website Current Gene Therapy
Wednesday, October 26, 2016
Most Accessed Article: AAV Vectorization of DSB-mediated Gene Editing Technologies
AAV Vectorization of DSB-mediated Gene Editing Technologies
Author(s):
Rachel J. Moser and Matthew L. HirschPages 207-219 (13)
Abstract:
Recent work both at the bench and the bedside demonstrate zinc-finger nucleases (ZFNs), CRISPR/Cas9, and other programmable site-specific endonuclease technologies are being successfully utilized within and alongside AAV vectors to induce therapeutically relevant levels of directed gene editing within the human chromosome. Studies from past decades acknowledge that AAV vector genomes are enhanced substrates for homology-directed repair in the presence or absence of targeted DNA damage within the host genome. Additionally, AAV vectors are currently the most efficient format for in vivo gene delivery with no vector related complications in >100 clinical trials for diverse diseases. At the same time, advancements in the design of custom-engineered site-specific endonucleases and the utilization of elucidated endonuclease formats have resulted in efficient and facile genetic engineering for basic science and for clinical therapies. AAV vectors and gene editing technologies are an obvious marriage, using AAV for the delivery of repair substrate and/or a gene encoding a designer endonuclease; however, while efficient delivery and enhanced gene targeting by vector genomes are advantageous, other attributes of AAV vectors are less desirable for gene editing technologies. This review summarizes the various roles that AAV vectors play in gene editing technologies and provides insight into its trending applications for the treatment of genetic diseases.
Keywords:
AAV Vectorization, DSB, CRISPR/Cas9, DNA, ZFNs.
Affiliation:
Department of Ophthalmology, Gene Therapy Center, University of North Carolina at Chapel Hill, Campus Box 7352, Chapel Hill, NC 27599-7352, USA.
For More Information Please Visit Our Website Current Gene Therapy
Thursday, October 20, 2016
Thursday, October 6, 2016
A Short Video on Bentham Science Flagship Journal Current Gene Therapy
A Short Video on Bentham Science Flagship Journal Current Gene Therapy
YouTube: http://bit.ly/1zci8cR
Access the complete journal here: http://bit.ly/1qtBwL6
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Wednesday, October 5, 2016
Bentham Open Access Plus Article From the Journal Current Gene Therapy
The article entitled, ‘Gene/Cell Therapy Approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome,’ in the journal Current Gene Therapy, 2014, 14, 422-428 is now open for all to view and access.
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New Issue ::: Current Gene Therapy Vol. 15 issue 5
Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of genetic diseases. Cell therapy manuscripts can also include application in non-genetic diseases when cells have been genetically modified. Current Gene Therapy publishes reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of genetic diseases.
Articles from the journal in Current Gene Therapy, 15 issue 5
- Article: CFTR Inactivation by Lentiviral Vector-mediated RNA Interference and CRISPR-Cas9 Genome Editing in Human Airway Epithelial Cells
- Article: Molecular Imaging to Monitor Repair of Myocardial Infarction Using Genetically Engineered Bone Marrow-Derived Mesenchymal Stem Cells
- Article: Folate-conjugated Chitosan-poly(ethylenimine) Copolymer As An Efficient and Safe Vector For Gene Delivery in Cancer Cells
- Article: Biodistribution of LV-TSTA Transduced Rat Bone Marrow Cells Used for “Ex-vivo” Regional Gene Therapy for Bone Repair
- Article: Bifunctional siRNA Containing Immunostimulatory Motif Enhances Protection Against Pandemic H1N1 Virus Infection
- Article: Therapy for Dominant Inherited Diseases by Allele-Specific RNA Interference: Successes and Pitfalls
- Article: Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer
For details, please visit: http://bit.ly/1FODIUz
courtesy by : https://benthamsciencepublishers.wordpress.com/2015/11/09/new-issue-current-gene-therapy-vol-15-issue-5/
Recently Published Issue of the Journal in Current Gene Therapy
Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of genetic diseases. Cell therapy manuscripts can also include application in non-genetic diseases when cells have been genetically modified. Current Gene Therapy publishes reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of genetic diseases.
Following are the articles from the journal in Current Gene Therapy, 15issue 5:
Article: CFTR Inactivation by Lentiviral Vector-mediated RNA Interference and CRISPR-Cas9 Genome Editing in Human Airway Epithelial Cells
Author(s): Jessica Bellec, Marc Bacchetta, Davide Losa, Ignacio Anegon, Marc Chanson and Tuan Huy Nguyen
Article: Molecular Imaging to Monitor Repair of Myocardial Infarction Using Genetically Engineered Bone Marrow-Derived Mesenchymal Stem Cells
Author(s): Shuo Shi, Min Zhang, Rui Guo, Ying Miao, Xiangming Zhang and Biao Li
Article: Folate-conjugated Chitosan-poly(ethylenimine) Copolymer As An Efficient and Safe Vector For Gene Delivery in Cancer Cells
Author(s): Wing-Fu Lai and Marie C. Lin
Article: Biodistribution of LV-TSTA Transduced Rat Bone Marrow Cells Used for “Ex-vivo” Regional Gene Therapy for Bone Repair
Author(s): Farhang Alaee, Cynthia Bartholomae, Osamu Sugiyama, Mandeep S. Virk, Hicham Drissi, Qian Wu, Manfred Schmidt and Jay R. Lieberman
Article: Bifunctional siRNA Containing Immunostimulatory Motif Enhances Protection Against Pandemic H1N1 Virus Infection
Author(s): Gaurav Joshi, Paban Kumar Dash, Ankita Agarwal, Shashi Sharma and Manmohan Parida
Article: Therapy for Dominant Inherited Diseases by Allele-Specific RNA Interference: Successes and Pitfalls
Author(s): Delphine Trochet, Bernard Prudhon, Stéphane Vassilopoulos and Marc Bitoun
Article: Current Genome Editing Tools in Gene Therapy: New Approaches to Treat Cancer
Author(s): Oleg Shuvalov, Alexey Petukhov, Alexandra Daks, Olga Fedorova, Alexander Ermakov, Gerry Melino and Nickolai A Barlev
For details, please visit: http://bit.ly/1FODIUz
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Contributions by Japanese Authors in Bentham Science Journal ‘Current Gene Therapy’
Manganese Superoxide Dismutase Gene Therapy Protects Against Irradiation- Induced Intestinal Injury
Author(s): Chao Yang, Hai-Xu Chen, Yong Zhou, Min-Xia Liu, Yan Wang, Jie-Xi Wang, Su-Ping Ren, Ying Han and Ben-Yan Wu
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Affiliation: Beijing Institute of Transfusion Medicine, Beijing 100850, China.
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Affiliation: Beijing Institute of Transfusion Medicine, Beijing 100850, China.
Abstract: Radiation-induced intestinal injury is a common complication in radiotherapy for solid organ malignancies in abdomen or pelvis. However, currently there are no approved medical countermeasures for radiation-induced intestinal injury. Therefore, it is urgent to develop new treatments for radiation-induced intestinal injury. In the present study, we demonstrated that bone marrow derived mesenchymal stem cells (MSCs) and overexpression of human manganese superoxide dismutase (MnSOD) could ameliorate radiation-induced intestinal syndrome.
Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of genetic diseases. Cell therapy manuscripts can also include application in non-genetic diseases when cells have been genetically modified. Current Gene Therapy publishes reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of genetic diseases.
courtesy by : https://benthamsciencepublishers.wordpress.com/2015/07/17/contributions-by-japanese-authors-in-bentham-science-journal-current-gene-therapy-2/
Highlighted Article Flyer for the journal “Current Gene Therapy” Volume 16, Number 1
courtesy by : https://benthamsciencepublishers.wordpress.com/2016/06/30/highlighted-article-flyer-for-the-journal-current-gene-therapy/
Wednesday, July 27, 2016
Effects of APC De-Targeting and GAr Modification on the Duration of Luciferase Expression from Plasmid DNA Delivered to Skeletal Muscle
Author(s):
Maria C. Subang, Rewas Fatah, Ying Wu, Drew Hannaman, Jason Rice, Claire F. Evans, Yuti Chernajovsky and David GouldPages 3-14 (12)
Abstract:
Immune responses to expressed foreign transgenes continue to hamper progress of gene therapy development. Translated foreign proteins with intracellular location are generally less accessible to the immune system, nevertheless they can be presented to the immune system through both MHC Class I and Class II pathways. When the foreign protein luciferase was expressed following intramuscular delivery of plasmid DNA in outbred mice, expression rapidly declined over 4 weeks. Through modifications to the expression plasmid and the luciferase transgene we examined the effect of detargeting expression away from antigen-presenting cells (APCs), targeting expression to skeletal muscle and fusion with glycine-alanine repeats (GAr) that block MHC-Class I presentation on the duration of luciferase expression. De-targeting expression from APCs with miR142-3p target sequences incorporated into the luciferase 3’UTR reduced the humoral immune response to both native and luciferase modified with a short GAr sequence but did not prolong the duration of expression. When a skeletal muscle specific promoter was combined with the miR target sequences the humoral immune response was dampened and luciferase expression persisted at higher levels for longer. Interestingly, fusion of luciferase with a longer GAr sequence promoted the decline in luciferase expression and increased the humoral immune response to luciferase. These studies demonstrate that expression elements and transgene modifications can alter the duration of transgene expression but other factors will need to overcome before foreign transgenes expressed in skeletal muscle are immunologically silent.
Keywords:
Gene therapy, luciferase, microRNA, plasmid DNA, skeletal muscle, tissue-specific promoter, transgene immunogenicity.
Affiliation:
Bone & Joint Research Unit, Queen Mary University of London, William Harvey Research Institute, Charterhouse Square, London EC1M 6BQ, UK.
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Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
Author(s):
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 VeresPages 64-81 (18)
Abstract:
A previously published clinical trial demonstrated the benefit of autologous CD34+ cells transduced with a selfinactivating lentiviral vector (HPV569) containing an engineered β-globin gene (βA-T87Q-globin) in a subject with β thalassemia major. This vector has been modified to increase transduction efficacy without compromising safety. In vitro analyses indicated that the changes resulted in both increased vector titers (3 to 4 fold) and increased transduction efficacy (2 to 3 fold). An in vivo study in which 58 β-thalassemic mice were transplanted with vector- or mock-transduced syngenic bone marrow cells indicated sustained therapeutic efficacy. Secondary transplantations involving 108 recipients were performed to evaluate long-term safety. The six month study showed no hematological or biochemical toxicity. Integration site (IS) profile revealed an oligo/polyclonal hematopoietic reconstitution in the primary transplants and reduced clonality in secondary transplants. Tumor cells were detected in the secondary transplant mice in all treatment groups (including the control group), without statistical differences in the tumor incidence. Immunohistochemistry and quantitative PCR demonstrated that tumor cells were not derived from transduced donor cells. This comprehensive efficacy and safety data provided the basis for initiating two clinical trials with this second generation vector (BB305) in Europe and in the USA in patients with β-thalassemia major and sickle cell disease.
Keywords:
β-hemoglobinopathy, β-thalassemia, gene therapy, lentiviral vector, mouse model.
Affiliation:
bluebird bio, 150 Second Street, Cambridge, MA 02141, USA.
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Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
Author(s):
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 VeresPages 64-81 (18)
Abstract:
A previously published clinical trial demonstrated the benefit of autologous CD34+ cells transduced with a selfinactivating lentiviral vector (HPV569) containing an engineered β-globin gene (βA-T87Q-globin) in a subject with β thalassemia major. This vector has been modified to increase transduction efficacy without compromising safety. In vitro analyses indicated that the changes resulted in both increased vector titers (3 to 4 fold) and increased transduction efficacy (2 to 3 fold). An in vivo study in which 58 β-thalassemic mice were transplanted with vector- or mock-transduced syngenic bone marrow cells indicated sustained therapeutic efficacy. Secondary transplantations involving 108 recipients were performed to evaluate long-term safety. The six month study showed no hematological or biochemical toxicity. Integration site (IS) profile revealed an oligo/polyclonal hematopoietic reconstitution in the primary transplants and reduced clonality in secondary transplants. Tumor cells were detected in the secondary transplant mice in all treatment groups (including the control group), without statistical differences in the tumor incidence. Immunohistochemistry and quantitative PCR demonstrated that tumor cells were not derived from transduced donor cells. This comprehensive efficacy and safety data provided the basis for initiating two clinical trials with this second generation vector (BB305) in Europe and in the USA in patients with β-thalassemia major and sickle cell disease.
Keywords:
β-hemoglobinopathy, β-thalassemia, gene therapy, lentiviral vector, mouse model.
Affiliation:
bluebird bio, 150 Second Street, Cambridge, MA 02141, USA.
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A Simple High Efficiency Intra-Islet Transduction Protocol Using Lentiviral Vectors
Author(s):
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-MontalvoPages 436-446 (11)
Abstract:
Successful normalization of blood glucose in patients transplanted with pancreatic islets isolated from cadaveric donors established the proof-of-concept that Type 1 Diabetes Mellitus is a curable disease. Nonetheless, major caveats to the widespread use of this cell therapy approach have been the shortage of islets combined with the low viability and functional rates subsequent to transplantation. Gene therapy targeted to enhance survival and performance prior to transplantation could offer a feasible approach to circumvent these issues and sustain a durable functional β-cell mass in vivo. However, efficient and safe delivery of nucleic acids to intact islet remains a challenging task. Here we describe a simple and easy-to-use lentiviral transduction protocol that allows the transduction of approximately 80 % of mouse and human islet cells while preserving islet architecture, metabolic function and glucose-dependent stimulation of insulin secretion. Our protocol will facilitate to fully determine the potential of gene expression modulation of therapeutically promising targets in entire pancreatic islets for xenotransplantation purposes.
Keywords:
Diabetes Mellitus, Gene transfer, Infection, Lentivirus, Pancreatic islet, Transduction.
Affiliation:
Pancreatic Islet Development and Regeneration Unit, Department of Stem Cells, CABIMER-Andalusian Center for Molecular Biology and Regenerative Medicine, Avenida Americo Vespucio, Parque Científico y Tecnologico Cartuja 93, 41092 Sevilla, Spain.
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Building A New Treatment For Heart Failure-Transplantation of Induced Pluripotent Stem Cell-derived Cells into the Heart
Author(s):
Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura, Noriko Mochizuki- Oda, Shigeo Masuda and Yoshiki SawaPages 5-13 (9)
Abstract:
Advanced cardiac failure is a progressive intractable disease and is the main cause of mortality and morbidity worldwide. Since this pathology is represented by a definite decrease in cardiomyocyte number, supplementation of functional cardiomyocytes into the heart would hypothetically be an ideal therapeutic option. Recently, unlimited in vitro production of human functional cardiomyocytes was established by using induced pluripotent stem cell (iPSC) technology, which avoids the use of human embryos. A number of basic studies including ours have shown that transplantation of iPSCderived cardiomyocytes (iPSC-CMs) into the damaged heart leads to recovery of cardiac function, thereby establishing “proof-of-concept” of this iPSC-transplantation therapy. However, considering clinical application of this therapy, its feasibility, safety, and therapeutic efficacy need to be further investigated in the pre-clinical stage. This review summarizes up-to-date important topics related to safety and efficacy of iPSC-CMs transplantation therapy for cardiac disease and discusses the prospects for this treatment in clinical studies.
Keywords:
iPSC, Cardiomyocytes, Transplantation, Heart failure, Cardiomyoplasty, Immunogenicity.
Affiliation:
Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, 2-2 Yamadaoka, Suita, Osaka, 565-0871, Japan.
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Gene Electrotransfer: A Mechanistic Perspective
Author(s):
Christelle Rosazza, Sasa Haberl Meglic, Andreas Zumbusch, Marie-Pierre Rols and Damijan MiklavcicPages 98-129 (32)
Abstract:
Gene electrotransfer is a powerful method of DNA delivery offering several medical applications, among the most promising of which are DNA vaccination and gene therapy for cancer treatment. Electroporation entails the application of electric fields to cells which then experience a local and transient change of membrane permeability. Although gene electrotransfer has been extensively studied in in vitro and in vivo environments, the mechanisms by which DNA enters and navigates through cells are not fully understood. Here we present a comprehensive review of the body of knowledge concerning gene electrotransfer that has been accumulated over the last three decades. For that purpose, after briefly reviewing the medical applications that gene electrotransfer can provide, we outline membrane electropermeabilization, a key process for the delivery of DNA and smaller molecules. Since gene electrotransfer is a multipart process, we proceed our review in describing step by step our current understanding, with particular emphasis on DNA internalization and intracellular trafficking. Finally, we turn our attention to in vivo testing and methodology for gene electrotransfer.
Keywords:
Electric field, Electroporation, Gene electrotransfer, Plasmid DNA, Gene therapy, DNA vaccination.
Affiliation:
Institute of Pharmacology and Structural Biology (IPBS), CNRS UMR5089, 205 route de Narbonne, 31077 Toulouse, France.
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