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Current Gene Therapy

Maike Stahlhut, Teng-Cheong Ha, Michael Morgan, Axel Schambach, Olga S Kustikova
Tetracycline-regulated systems with efficient temporal and dose regulation of transgene expression are useful for development of new physiologic/pathophysiologic experimental models and gene therapy approaches. Lentiviral vectors with improved tetracycline-regulated promoters help to overcome the existing limitations such as basal activity in the drug absence, poor inducibility or unstable transgene expression. To compare conventional and improved tetracycline-regulated promoters in lentiviral based vectors in vivo, we investigated doxycycline-regulated gene transfer/expression levels in a long-term murine transplantation model and demonstrated that the lentiviral vector with the improved T11 promoter exhibited more efficient inducibility and higher gene transfer level...
October 13, 2016: Current Gene Therapy
Aastha Singh, Dwaipayan Sen
Although adeno-associated viral vectors have been studied for a long time, its importance as a viable gene therapy strategy has been thrust into the limelight only in the recent years. Due to the admirable characteristics of these vectors, their potential has been thoughtfully utilized in the treatment of several neurodegenerative diseases. This mini-review focuses at recapitulating the therapeutic advances of adeno-associated viral vectors in the treatment of Parkinson's disease by studying the various animal model experiments and clinical trials conducted since the advent of adeno-associated viral vector - based gene therapy...
July 29, 2016: Current Gene Therapy
Otto-Wilhelm Merten, Mauro Mezzina, Sylvain Fisson
No abstract text is available yet for this article.
2016: Current Gene Therapy
Rachel J Moser, Matthew L Hirsch
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...
2016: Current Gene Therapy
Yujia Cai, Jacob Giehm Mikkelsen
Viruses have evolved to traverse cellular barriers and travel to the nucleus by mechanisms that involve active transport through the cytoplasm and viral quirks to resist cellular restriction factors and innate immune responses. Virus-derived vector systems exploit the capacity of viruses to ferry genetic information into cells, and now - more than three decades after the discovery of HIV - lentiviral vectors based on HIV-1 have become instrumental in biomedical research and gene therapies that require genomic insertion of transgenes...
2016: Current Gene Therapy
Atze T Das, Liliane Tenenbaum, Ben Berkhout
The tetracycline-controlled Tet-Off and Tet-On gene expression systems are used to regulate the activity of genes in eukaryotic cells in diverse settings, varying from basic biological research to biotechnology and gene therapy applications. These systems are based on regulatory elements that control the activity of the tetracycline-resistance operon in bacteria. The Tet-Off system allows silencing of gene expression by administration of tetracycline (Tc) or tetracycline-derivatives like doxycycline (dox), whereas the Tet-On system allows activation of gene expression by dox...
2016: Current Gene Therapy
Nicole Schonrock, Nicky Jonkhout, John S Mattick
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...
2016: Current Gene Therapy
Jean-Baptiste Dupont
Despite the unprecedented beneficial effects of rAAV gene therapy in animal models of Duchenne muscular dystrophy (DMD), the need to inject large amounts of vector in vivo to improve phenotype raises obvious biosafety concerns. While rAAV vectors generally exhibit a good safety profile, specific pathological phenotypes such as those observed in dystrophin-deficient muscles may promote immunotoxic/genotoxic effects. Increasing the therapeutic index of rAAV in DMD muscles by reducing the effective dose could be a pivotal means of ensuring efficient clinical translation...
2016: Current Gene Therapy
Serena Scala, Lorena Leonardelli, Luca Biasco
Over the past years, clonal tracking has gained the center stage as a unique technology capable to unveil population dynamics and hierarchical relationships in vivo. We here highlighted the main open questions related to the in vivo clonal behavior of hematopoietic cells with a particular focus on hematopoietic stem and progenitor cells and T cells as main targets of cell- and gene-therapies. We walked through the current methods applied for tracing in vivo dynamics and functions of hematopoietic cells in animal models and we described the results of early studies conducted on humans...
2016: Current Gene Therapy
Marie Pierre Rols, Damijan Miklavcic
No abstract text is available yet for this article.
2016: Current Gene Therapy
Moinecha Madi, Marie-Pierre Rols, Laure Gibot
Gene electrotransfer into the skin is of particular interest for the development of medical applications including DNA vaccination, cancer treatment, wound healing or treatment of local skin disorders. However, such clinical applications are currently limited due to poor understanding of the mechanisms governing DNA electrotransfer within human tissue. Nowadays, most studies are carried out in rodent models but rodent skin varies from human skin in terms of cell composition and architecture. We used a tissue-engineering approach to study gene electrotransfer mechanisms in a human tissue context...
2016: Current Gene Therapy
Dimitrios Agas, Fabio Concetti, Melania Capitani, Giovanna Lacava, Antonio Concetti, Luigi Marchetti, Fulvio Laus, Andrea Marchegiani, Vasco Azevedo, Maria Giovanna Sabbieti, Franco Maria Venanzi
BACKGROUND: Plasmids coding protein aggregation polypeptides from different sources have been proposed as genetic adjuvants for DNA vaccines. We reported that a plasmid (pATRex), encompassing the DNA sequence for the von Willebrand A (vWA/A) domain of the Anthrax Toxin Receptor-1 (ANTXR-1, alias TEM8, Tumor Endothelial Marker 8), acts as strong immune adjuvant by inducing formation of insoluble intracellular aggregates and subsequent cell death. OBJECTIVE: In the present study we addressed the question of whether there is any substantial immunotoxicity associated with the use of self-aggregating proteins as genetic adjuvants...
2016: Current Gene Therapy
Urska Kamensek, Marie-Pierre Rols, Maja Cemazar, Muriel Golzio
BACKGROUND: Gene Electro Transfer (GET) is a promising method for therapeutic purposes. Intratumoral GET has reached clinical evaluation for antitumor treatment. An increasing number of studies suggests that antitumor effectiveness not only depends on the transfection efficiency, but also on the induction of immune responses and vascular effects that result in the nonspecific induction of cell death. Real time noninvasive optical imaging methods allow longitudinal studies of these dynamic biological processes...
2016: Current Gene Therapy
Amy Donate, Anna Bulysheva, Chelsea Edelblute, Derrick Jung, Mohammad A Malik, Siqi Guo, Niculina Burcus, Karl Schoenbach, Richard Heller
Gene electrotransfer is an effective approach for delivering plasmid DNA to a variety of tissues. Delivery of molecules with electric pulses requires control of the electrical parameters to achieve effective delivery. Since discomfort or tissue damage may occur with high applied voltage, the reduction of the applied voltage while achieving the desired expression may be an important improvement. One possible approach is to combine electrotransfer with exogenously applied heat. Previous work performed in vitro demonstrated that increasing temperature before pulsing can enhance gene expression and made it possible to reduce electric fields while maintaining expression levels...
2016: Current Gene Therapy
Christelle Rosazza, Sasa Haberl Meglic, Andreas Zumbusch, Marie-Pierre Rols, Damijan Miklavcic
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...
2016: Current Gene Therapy
Lumin Zhang, Tsurumi Tatsuya, Yukihiro Nishiyama
The high level of manipulability of viral genome has set up HSV-1 to be an ideal viral vector for oncolytic virotherapy. In the past two decades, several oncolytic HSV-1 viruses have been successfully developed and assessed in animal studies. Accumulated evidences show that oncolytic HSV- 1 can efficiently infect many tumor cells and augment anti-tumor effect by induction of systemic innate and adaptive immune responses. Inspiring results have been accomplished in several phase I clinical trials for glioma, head and neck squeous cells carcinoma and Melanoma using oncolytic HSV- 1 viruses...
2016: Current Gene Therapy
Ignacio Anegon, Tuan H Nguyen
No abstract text is available yet for this article.
2016: Current Gene Therapy
Shu Uin Gan, Maria Notaridou, Zhen Ying Fu, Kok Onn Lee, Kian Chuan Sia, Amit Chunilal Nathwani, Marco Della Peruta, Roy Yorke Calne
We report the correction of hyperglycemia of STZ induced diabetic mice using one intravenous systemic administration of a single stranded serotype 8 pseudotyped adeno-associated virus (ssAAV2/8) vector encoding the human proinsulin gene under a constitutive liver specific promoter. In vivo dose titration experiments were carried out and we identified an optimal range that achieved maintenance of euglycaemia or a mild diabetic condition for at least 9 months and ongoing to beyond 1 year for some animals, accompanied by human C-peptide secretion and weight gain...
2016: Current Gene Therapy
Imke Steffen, Graham Simmons
Previously unidentified viruses, such as Middle East respiratory syndrome coronavirus, continue to emerge and threaten populations, while powerful new techniques have identified many new human and animal viruses. Similarly, existing viruses, from Ebola virus to chikungunya virus, are reemerging and spreading to new geographical regions. These viruses often pose a challenge for researchers to study due to their highly pathogenic nature. Lentiviral and rhabdoviral pseudotypes are excellent tools for studying enveloped viruses and have contributed to many recent advances in areas such as receptor usage, viral entry and serology...
2016: Current Gene Therapy
Shigeru Miyagawa, Satsuki Fukushima, Yukiko Imanishi, Takuji Kawamura, Noriko Mochizuki-Oda, Shigeo Masuda, Yoshiki Sawa
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...
2016: Current Gene Therapy
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