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Viruses
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Novel Viral Vector Systems for Gene Therapy
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Novel Viral Vector Systems for Gene Therapy

A special issue of Viruses (ISSN 1999-4915). This special issue belongs to the section "Animal Viruses".

Deadline for manuscript submissions: closed (30 December 2009) | Viewed by 151771

Special Issue Editor

Dr. Daniel Stone

E-Mail Website
Guest Editor
Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center in Seattle, Mailstop E5-110, 1100 Fairview Ave. N., Seattle, WA 98109, USA
Interests: development of novel viral vector systems for gene therapy; events that initiate innate immunity and toxicity following virus exposure; mechanisms that facilitate virus attachment, entry, replication and pathogenesis

Keywords

  • foamy virus
  • adenovirus
  • adeno-associated virus
  • alpha virus
  • lentivirus
  • oncolytic viral vectors
  • cardiovascular gene therapy
  • musculoskeletal gene therapy

Published Papers (10 papers)

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Editorial

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36 KiB  
Editorial
Novel Viral Vector Systems for Gene Therapy
by Daniel Stone
Viruses 2010, 2(4), 1002-1007; https://doi.org/10.3390/v2041002 - 14 Apr 2010
Cited by 33 | Viewed by 9648
Abstract
Over the last three decades, interest in the field of gene therapy seems to have fluctuated between hot and cold. Encouraging pre-clinical and clinical data has demonstrated the potential of genetic therapies and yet setbacks in clinical trials have cast doubts in some [...] Read more.
Over the last three decades, interest in the field of gene therapy seems to have fluctuated between hot and cold. Encouraging pre-clinical and clinical data has demonstrated the potential of genetic therapies and yet setbacks in clinical trials have cast doubts in some minds over the clinical future of gene therapy [1-3]. In the last two years, a number of studies have demonstrated therapeutic benefits in clinical trials aimed towards specific monogenetic disorders [4-6], and this has brought renewed optimism to the field. [...] Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)

Review

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658 KiB  
Review
PEGylated Adenoviruses: From Mice to Monkeys
by Piyanuch Wonganan and Maria A. Croyle
Viruses 2010, 2(2), 468-502; https://doi.org/10.3390/v2020468 - 01 Feb 2010
Cited by 46 | Viewed by 14488
Abstract
Covalent modification with polyethylene glycol (PEG), a non-toxic polymer used in food, cosmetic and pharmaceutical preparations for over 60 years, can profoundly influence the pharmacokinetic, pharmacologic and toxciologic profile of protein and peptide-based therapeutics. This review summarizes the history of PEGylation and PEG [...] Read more.
Covalent modification with polyethylene glycol (PEG), a non-toxic polymer used in food, cosmetic and pharmaceutical preparations for over 60 years, can profoundly influence the pharmacokinetic, pharmacologic and toxciologic profile of protein and peptide-based therapeutics. This review summarizes the history of PEGylation and PEG chemistry and highlights the value of this technology in the context of the design and development of recombinant viruses for gene transfer, vaccination and diagnostic purposes. Specific emphasis is placed on the application of this technology to the adenovirus, the most potent viral vector with the most highly characterized toxicity profile to date, in several animal models. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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234 KiB  
Review
Lentiviral Vectors and Cystic Fibrosis Gene Therapy
by Stefano Castellani and Massimo Conese
Viruses 2010, 2(2), 395-412; https://doi.org/10.3390/v2020395 - 29 Jan 2010
Cited by 22 | Viewed by 14504
Abstract
Cystic fibrosis (CF) is a chronic autosomic recessive syndrome, caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene, a chloride channel expressed on the apical side of the airway epithelial cells. The lack of CFTR activity brings a dysregulated exchange of [...] Read more.
Cystic fibrosis (CF) is a chronic autosomic recessive syndrome, caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene, a chloride channel expressed on the apical side of the airway epithelial cells. The lack of CFTR activity brings a dysregulated exchange of ions and water through the airway epithelium, one of the main aspects of CF lung disease pathophysiology. Lentiviral (LV) vectors, of the Retroviridae family, show interesting properties for CF gene therapy, since they integrate into the host genome and allow long-lasting gene expression. Proof-of-principle that LV vectors can transduce the airway epithelium and correct the basic electrophysiological defect in CF mice has been given. Initial data also demonstrate that LV vectors can be repeatedly administered to the lung and do not give rise to a gross inflammatory process, although they can elicit a T cell-mediated response to the transgene. Future studies will clarify the efficacy and safety profile of LV vectors in new complex animal models with CF, such as ferrets and pigs. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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348 KiB  
Review
Development of Viral Vectors for Use in Cardiovascular Gene Therapy
by Paul D. Williams, Parisa Ranjzad, Salik J. Kakar and Paul A. Kingston
Viruses 2010, 2(2), 334-371; https://doi.org/10.3390/v2020334 - 27 Jan 2010
Cited by 32 | Viewed by 13811
Abstract
Cardiovascular disease represents the most common cause of mortality in the developed world but, despite two decades of promising pre-clinical research and numerous clinical trials, cardiovascular gene transfer has so far failed to demonstrate convincing benefits in the clinical setting. In this review [...] Read more.
Cardiovascular disease represents the most common cause of mortality in the developed world but, despite two decades of promising pre-clinical research and numerous clinical trials, cardiovascular gene transfer has so far failed to demonstrate convincing benefits in the clinical setting. In this review we discuss the various targets which may be suitable for cardiovascular gene therapy and the viral vectors which have to date shown the most potential for clinical use. We conclude with a summary of the current state of clinical cardiovascular gene therapy and the key trials which are ongoing. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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130 KiB  
Review
Virus Infection Recognition and Early Innate Responses to Non-Enveloped Viral Vectors
by Dmitry M. Shayakhmetov
Viruses 2010, 2(1), 244-261; https://doi.org/10.3390/v2010244 - 19 Jan 2010
Cited by 8 | Viewed by 11378
Abstract
Numerous human genetic and acquired diseases could be corrected or ameliorated if viruses are harnessed to safely and effectively deliver therapeutic genes to diseased cells and tissues in vivo. Innate immune and inflammatory response represents one of the key stumbling blocks during [...] Read more.
Numerous human genetic and acquired diseases could be corrected or ameliorated if viruses are harnessed to safely and effectively deliver therapeutic genes to diseased cells and tissues in vivo. Innate immune and inflammatory response represents one of the key stumbling blocks during the development of viral-based therapies. In this review, current data on the early innate immune responses to viruses and to the most commonly used gene therapy vectors (using adenovirus and adeno-associated virus) will be discussed. Recent findings in the field may help develop new approaches to moderate these innate immune anti-viral responses and thus improve the safety of viral vectors for human gene therapy applications. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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351 KiB  
Review
Oncolytic Viruses for Cancer Therapy: Overcoming the Obstacles
by Han Hsi Wong, Nicholas R. Lemoine and Yaohe Wang
Viruses 2010, 2(1), 78-106; https://doi.org/10.3390/v2010078 - 11 Jan 2010
Cited by 134 | Viewed by 27658
Abstract
Targeted therapy of cancer using oncolytic viruses has generated much interest over the past few years in the light of the limited efficacy and side effects of standard cancer therapeutics for advanced disease. In 2006, the world witnessed the first government-approved oncolytic virus [...] Read more.
Targeted therapy of cancer using oncolytic viruses has generated much interest over the past few years in the light of the limited efficacy and side effects of standard cancer therapeutics for advanced disease. In 2006, the world witnessed the first government-approved oncolytic virus for the treatment of head and neck cancer. It has been known for many years that viruses have the ability to replicate in and lyse cancer cells. Although encouraging results have been demonstrated in vitro and in animal models, most oncolytic viruses have failed to impress in the clinical setting. The explanation is multifactorial, determined by the complex interactions between the tumor and its microenvironment, the virus, and the host immune response. This review focuses on discussion of the obstacles that oncolytic virotherapy faces and recent advances made to overcome them, with particular reference to adenoviruses. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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485 KiB  
Review
Applying Genomic and Bioinformatic Resources to Human Adenovirus Genomes for Use in Vaccine Development and for Applications in Vector Development for Gene Delivery
by Jason Seto, Michael P. Walsh, Padmanabhan Mahadevan, Qiwei Zhang and Donald Seto
Viruses 2010, 2(1), 1-26; https://doi.org/10.3390/v2010001 - 06 Jan 2010
Cited by 15 | Viewed by 13261
Abstract
Technological advances and increasingly cost-effect methodologies in DNA sequencing and computational analysis are providing genome and proteome data for human adenovirus research. Applying these tools, data and derived knowledge to the development of vaccines against these pathogens will provide effective prophylactics. The same [...] Read more.
Technological advances and increasingly cost-effect methodologies in DNA sequencing and computational analysis are providing genome and proteome data for human adenovirus research. Applying these tools, data and derived knowledge to the development of vaccines against these pathogens will provide effective prophylactics. The same data and approaches can be applied to vector development for gene delivery in gene therapy and vaccine delivery protocols. Examination of several field strain genomes and their analyses provide examples of data that are available using these approaches. An example of the development of HAdV-B3 both as a vaccine and also as a vector is presented. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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660 KiB  
Review
Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution?
by Nadine Müther, Nadja Noske and Anja Ehrhardt
Viruses 2009, 1(3), 1295-1324; https://doi.org/10.3390/v1031295 - 15 Dec 2009
Cited by 14 | Viewed by 17269
Abstract
The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based on these findings major [...] Read more.
The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based on these findings major improvements in vector design including the development of viral hybrid vectors for somatic integration have been achieved. This review provides a state-of-the-art overview of available hybrid vectors utilizing viruses for high transduction efficiencies in concert with various integration machineries for random and targeted integration patterns. It discusses advantages but also limitations of each vector system. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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369 KiB  
Review
Herpes Virus Amplicon Vectors
by Suresh De Silva and William J. Bowers
Viruses 2009, 1(3), 594-629; https://doi.org/10.3390/v1030594 - 29 Oct 2009
Cited by 28 | Viewed by 16744
Abstract
Since its emergence onto the gene therapy scene nearly 25 years ago, the replication-defective Herpes Simplex Virus Type-1 (HSV-1) amplicon has gained significance as a versatile gene transfer platform due to its extensive transgene capacity, widespread cellular tropism, minimal immunogenicity, and its amenability [...] Read more.
Since its emergence onto the gene therapy scene nearly 25 years ago, the replication-defective Herpes Simplex Virus Type-1 (HSV-1) amplicon has gained significance as a versatile gene transfer platform due to its extensive transgene capacity, widespread cellular tropism, minimal immunogenicity, and its amenability to genetic manipulation. Herein, we detail the recent advances made with respect to the design of the HSV amplicon, its numerous in vitro and in vivo applications, and the current impediments this virus-based gene transfer platform faces as it navigates a challenging path towards future clinical testing. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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653 KiB  
Review
Alphaviruses in Gene Therapy
by Kenneth Lundstrom
Viruses 2009, 1(1), 13-25; https://doi.org/10.3390/v1010013 - 21 Apr 2009
Cited by 28 | Viewed by 11516
Abstract
Alphaviruses are enveloped single stranded RNA viruses, which as gene therapy vectors provide high-level transient gene expression. Semliki Forest virus (SFV), Sindbis virus (SIN) and Venezuelan Equine Encephalitis (VEE) virus have been engineered as efficient replication-deficient and -competent expression vectors. Alphavirus vectors have [...] Read more.
Alphaviruses are enveloped single stranded RNA viruses, which as gene therapy vectors provide high-level transient gene expression. Semliki Forest virus (SFV), Sindbis virus (SIN) and Venezuelan Equine Encephalitis (VEE) virus have been engineered as efficient replication-deficient and -competent expression vectors. Alphavirus vectors have frequently been used as vehicles for tumor vaccine generation. Moreover, SFV and SIN vectors have been applied for intratumoral injections in animals implanted with tumor xenografts. SIN vectors have demonstrated natural tumor targeting, which might permit systemic vector administration. Another approach for systemic delivery of SFV has been to encapsulate replication-deficient viral particles in liposomes, which can provide passive targeting to tumors and allow repeated administration without host immune responses. This approach has demonstrated safe delivery of encapsulated SFV particles to melanoma and kidney carcinoma patients in a phase I trial. Finally, the prominent neurotropism of alphaviruses make them attractive for the treatment of CNS-related diseases. Full article
(This article belongs to the Special Issue Novel Viral Vector Systems for Gene Therapy)
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