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Genome Editing Therapy Using Viral Vectors of Adenovirus and Lentivirus
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Genome Editing Therapy Using Viral Vectors of Adenovirus and Lentivirus

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: closed (31 December 2022) | Viewed by 7925

Special Issue Editor

Prof. Dr. Izumu Saito

E-Mail Website
Guest Editor
Laboratory of Molecular Genetics, Institute of Medical Science, University of Tokyo, Minato-ku, Tokyo 108-8639, Japan
Interests: development of replication-incompetent adenovirus vectors; genome editing using viral vectors; viral vectors expressing multiplex guide RNAs
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Genome editing is attractive because it allows the impossible to become possible. This technique could offer novel therapies for infectious diseases, cancers, and genetic diseases. Furthermore, this technique is used in various biological studies in both basic and applied areas. Viral vectors are effective in these fields and have wide potential for novel applications and developments. Although AAV vectors are often used in these fields, their small capacity is an inevitable problem and limits further application and developments.

The presented Special Issue focuses on the development and application of adenovirus and lentivirus vectors. Their large genomes and capacity have a wide potential in the field of genome editing. However, they are yet to experience popular usage. More studies, reports, and applications are needed to show their abilities and advantages.

The topics in this Special Issue include both basic and applied studies. Novel methods are also welcome. Studies using AAV vectors may be included if the advantages and disadvantages related to adenovirus and/or lentivirus vectors are sufficiently discussed. I hope that your contribution will provide valuable information to scientists aiming to cure diseases that currently lack effective treatments.

Prof. Dr. Izumu Saito
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 submissions that pass pre-check are 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.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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

  • adenovirus vectors
  • viral vectors
  • genome editing
  • guide RNA
  • short-hairpin RNA (shRNA)
  • gene therapy

Published Papers (2 papers)

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Review

13 pages, 501 KiB  
Review
Therapeutic Applications for Oncolytic Self-Replicating RNA Viruses
by Kenneth Lundstrom
Int. J. Mol. Sci. 2022, 23(24), 15622; https://doi.org/10.3390/ijms232415622 - 9 Dec 2022
Cited by 7 | Viewed by 2281
Abstract
Self-replicating RNA viruses have become attractive delivery vehicles for therapeutic applications. They are easy to handle, can be rapidly produced in large quantities, and can be delivered as recombinant viral particles, naked or nanoparticle-encapsulated RNA, or plasmid DNA-based vectors. The self-replication of RNA [...] Read more.
Self-replicating RNA viruses have become attractive delivery vehicles for therapeutic applications. They are easy to handle, can be rapidly produced in large quantities, and can be delivered as recombinant viral particles, naked or nanoparticle-encapsulated RNA, or plasmid DNA-based vectors. The self-replication of RNA in infected host cells provides the means for generating much higher transgene expression levels and the possibility to apply substantially reduced amounts of RNA to achieve similar expression levels or immune responses compared to conventional synthetic mRNA. Alphaviruses and flaviviruses, possessing a single-stranded RNA genome of positive polarity, as well as measles viruses and rhabdoviruses with a negative-stranded RNA genome, have frequently been utilized for therapeutic applications. Both naturally and engineered oncolytic self-replicating RNA viruses providing specific replication in tumor cells have been evaluated for cancer therapy. Therapeutic efficacy has been demonstrated in animal models. Furthermore, the safe application of oncolytic viruses has been confirmed in clinical trials. Multiple myeloma patients treated with an oncolytic measles virus (MV-NIS) resulted in increased T-cell responses against the measles virus and several tumor-associated antigen responses and complete remission in one patient. Furthermore, MV-CEA administration to patients with ovarian cancer resulted in a stable disease and more than doubled the median overall survival. Full article
(This article belongs to the Special Issue Genome Editing Therapy Using Viral Vectors of Adenovirus and Lentivirus)
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18 pages, 741 KiB  
Review
The Arrival of Gene Therapy for Patients with Hemophilia A
by Giancarlo Castaman, Giovanni Di Minno, Raimondo De Cristofaro and Flora Peyvandi
Int. J. Mol. Sci. 2022, 23(18), 10228; https://doi.org/10.3390/ijms231810228 - 6 Sep 2022
Cited by 10 | Viewed by 5281
Abstract
Historically, the standard of care for hemophilia A has been intravenous administration of exogenous factor VIII (FVIII), either as prophylaxis or episodically. The development of emicizumab, a humanized bispecific monoclonal antibody mimicking activated FVIII, was a subsequent advance in treatment. However, both exogenous [...] Read more.
Historically, the standard of care for hemophilia A has been intravenous administration of exogenous factor VIII (FVIII), either as prophylaxis or episodically. The development of emicizumab, a humanized bispecific monoclonal antibody mimicking activated FVIII, was a subsequent advance in treatment. However, both exogenous FVIII and emicizumab require repeated and lifelong administration, negatively impacting patient quality of life. A recent breakthrough has been the development of gene therapy. This allows a single intravenous treatment that could result in long-term expression of FVIII, maintenance of steady-state plasma concentrations, and minimization (or possibly elimination) of bleeding episodes for the recipient’s lifetime. Several gene therapies have been assessed in clinical trials, with positive outcomes. Valoctocogene roxaparvovec (an adeno-associated viral 5-based therapy encoding human B domain-deleted FVIII) is expected to be the first approved gene therapy in European countries, including Italy, in 2022. Some novel challenges exist including refining patient selection criteria, managing patient expectations, further elucidation of the durability and variability of transgene expression and long-term safety, and the development of standardized ‘hub and spoke’ centers to optimize and monitor this innovative treatment. Gene therapy represents a paradigm shift, and may become a new reference standard for treating patients with hemophilia A. Full article
(This article belongs to the Special Issue Genome Editing Therapy Using Viral Vectors of Adenovirus and Lentivirus)
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