Special Issue "Gene Therapy with Emphasis on RNA Interference"

A special issue of Viruses (ISSN 1999-4915).

Deadline for manuscript submissions: closed (28 February 2015).

Special Issue Editor

Dr. Kenneth Lundstrom

Guest Editor
PanTherapeutics, Rue des Remparts 4, CH-1095 Lutry, Switzerland
Interests: viral gene therapy; viral vaccines; gene expression using viral vectors; structural biology

Special Issue Information

Dear Colleagues,

The progress in gene therapy has been hampered by delivery inefficacy and safety concerns. Recent progress has casted some promising light on future development. A number of viral vectors have been re-engineered for improved delivery and enhanced safety. In this context, adenovirus, adeno-associated virus, lentivirus, retroviruses and other virus vectors have provided therapeutic efficacy for various diseases such as severe combined immunodeficiency, hemophilia, cancer and infectious diseases. The discovery of RNA interference, particularly the use of short interfering RNA and micro-RNA for reversible gene silencing, has substantially widened the application range of gene therapy. In this Special Issue, applications of various viral vectors and their use in gene therapy are reviewed with a special emphasis on RNA interference.

Dr. Kenneth Lundstrom
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2000 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • viral vectors
  • severe combined immunodeficiency
  • hemophilia
  • cancer
  • infectious diseases
  • RNA interference
  • gene silencing

Published Papers (5 papers)

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Editorial

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Open AccessEditorial
Special Issue: Gene Therapy with Emphasis on RNA Interference
Viruses 2015, 7(8), 4482-4487; https://doi.org/10.3390/v7082830 - 06 Aug 2015
Cited by 4
Abstract
Gene therapy was originally thought to cover replacement of malfunctioning genes in treatment of various diseases. Today, the field has been expanded to application of viral and non-viral vectors for delivery of recombinant proteins for the compensation of missing or insufficient proteins, anti-cancer [...] Read more.
Gene therapy was originally thought to cover replacement of malfunctioning genes in treatment of various diseases. Today, the field has been expanded to application of viral and non-viral vectors for delivery of recombinant proteins for the compensation of missing or insufficient proteins, anti-cancer genes and proteins for destruction of tumor cells, immunostimulatory genes and proteins for stimulation of the host defense system against viral agents and tumors. Recently, the importance of RNA interference and its application in gene therapy has become an attractive alternative for drug development. Full article
(This article belongs to the Special Issue Gene Therapy with Emphasis on RNA Interference)

Research

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Open AccessArticle
RNase P Ribozymes Inhibit the Replication of Human Cytomegalovirus by Targeting Essential Viral Capsid Proteins
Viruses 2015, 7(7), 3345-3360; https://doi.org/10.3390/v7072775 - 24 Jun 2015
Cited by 4
Abstract
An engineered RNase P-based ribozyme variant, which was generated using the in vitro selection procedure, was used to target the overlapping mRNA region of two proteins essential for human cytomegalovirus (HCMV) replication: capsid assembly protein (AP) and protease (PR). In vitro studies showed [...] Read more.
An engineered RNase P-based ribozyme variant, which was generated using the in vitro selection procedure, was used to target the overlapping mRNA region of two proteins essential for human cytomegalovirus (HCMV) replication: capsid assembly protein (AP) and protease (PR). In vitro studies showed that the generated variant, V718-A, cleaved the target AP mRNA sequence efficiently and its activity was about 60-fold higher than that of wild type ribozyme M1-A. Furthermore, we observed a reduction of 98%–99% in AP/PR expression and an inhibition of 50,000 fold in viral growth in cells with V718-A, while a 75% reduction in AP/PR expression and a 500-fold inhibition in viral growth was found in cells with M1-A. Examination of the antiviral effects of the generated ribozyme on the HCMV replication cycle suggested that viral DNA encapsidation was inhibited and as a consequence, viral capsid assembly was blocked when the expression of AP and PR was inhibited by the ribozyme. Thus, our study indicates that the generated ribozyme variant is highly effective in inhibiting HCMV gene expression and blocking viral replication, and suggests that engineered RNase P ribozyme can be potentially developed as a promising gene-targeting agent for anti-HCMV therapy. Full article
(This article belongs to the Special Issue Gene Therapy with Emphasis on RNA Interference)
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Open AccessArticle
Lentiviral Vector Mediated Claudin1 Silencing Inhibits Epithelial to Mesenchymal Transition in Breast Cancer Cells
Viruses 2015, 7(6), 2965-2979; https://doi.org/10.3390/v7062755 - 10 Jun 2015
Cited by 16
Abstract
Breast cancer has a high incidence and mortality rate worldwide. Several viral vectors including lentiviral, adenoviral and adeno-associated viral vectors have been used in gene therapy for various forms of human cancer, and have shown promising effects in controlling tumor development. Claudin1 (CLDN1) [...] Read more.
Breast cancer has a high incidence and mortality rate worldwide. Several viral vectors including lentiviral, adenoviral and adeno-associated viral vectors have been used in gene therapy for various forms of human cancer, and have shown promising effects in controlling tumor development. Claudin1 (CLDN1) is a member of the tetraspan transmembrane protein family that plays a major role in tight junctions and is associated with tumor metastasis. However, the role of CLDN1 in breast cancer is largely unexplored. In this study, we tested the therapeutic potential of silencing CLDN1 expression in two breast cancer (MDA-MB-231 and MCF7) cell lines using lentiviral vector mediated RNA interference. We found that a CLDN1 short hairpin (shRNA) construct efficiently silenced CLDN1 expression in both breast cancer cell lines, and CLDN1 knockdown resulted in reduced cell proliferation, survival, migration and invasion. Furthermore, silencing CLDN1 inhibited epithelial to mesenchymal transition (EMT) by upregulating the epithelial cell marker, E-cadherin, and downregulating mesenchymal markers, smooth muscle cell alpha-actin (SMA) and Snai2. Our data demonstrated that lentiviral vector mediated CLDN1 RNA interference has great potential in breast cancer gene therapy by inhibiting EMT and controlling tumor cell growth. Full article
(This article belongs to the Special Issue Gene Therapy with Emphasis on RNA Interference)
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Review

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Open AccessReview
Alphaviruses in Gene Therapy
Viruses 2015, 7(5), 2321-2333; https://doi.org/10.3390/v7052321 - 07 May 2015
Cited by 17
Abstract
Alphavirus vectors present an attractive approach for gene therapy applications due to the rapid and simple recombinant virus particle production and their broad range of mammalian host cell transduction. Mainly three types of alphavirus vectors, namely naked RNA, recombinant particles and DNA/RNA layered [...] Read more.
Alphavirus vectors present an attractive approach for gene therapy applications due to the rapid and simple recombinant virus particle production and their broad range of mammalian host cell transduction. Mainly three types of alphavirus vectors, namely naked RNA, recombinant particles and DNA/RNA layered vectors, have been subjected to preclinical studies with the goal of achieving prophylactic or therapeutic efficacy, particularly in oncology. In this context, immunization with alphavirus vectors has provided protection against challenges with tumor cells. Moreover, alphavirus intratumoral and systemic delivery has demonstrated substantial tumor regression and significant prolonged survival rates in various animal tumor models. Recent discoveries of the strong association of RNA interference and disease have accelerated gene therapy based approaches, where alphavirus-based gene delivery can play an important role. Full article
(This article belongs to the Special Issue Gene Therapy with Emphasis on RNA Interference)
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Open AccessReview
Baculovirus-mediated Gene Delivery and RNAi Applications
Viruses 2015, 7(4), 2099-2125; https://doi.org/10.3390/v7042099 - 22 Apr 2015
Cited by 20
Abstract
Baculoviruses are widely encountered in nature and a great deal of data is available about their safety and biology. Recently, these versatile, insect-specific viruses have demonstrated their usefulness in various biotechnological applications including protein production and gene transfer. Multiple in vitro and in [...] Read more.
Baculoviruses are widely encountered in nature and a great deal of data is available about their safety and biology. Recently, these versatile, insect-specific viruses have demonstrated their usefulness in various biotechnological applications including protein production and gene transfer. Multiple in vitro and in vivo studies exist and support their use as gene delivery vehicles in vertebrate cells. Recently, baculoviruses have also demonstrated high potential in RNAi applications in which several advantages of the virus make it a promising tool for RNA gene transfer with high safety and wide tropism. Full article
(This article belongs to the Special Issue Gene Therapy with Emphasis on RNA Interference)
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