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Pharmaceuticals
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AdV and AAV Mediated Gene Delivery
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AdV and AAV Mediated Gene Delivery

A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Pharmaceutical Technology".

Deadline for manuscript submissions: closed (20 September 2023) | Viewed by 10765

Special Issue Editors

Dr. Risini Weeratna

E-Mail Website
Guest Editor
Human Health Therapeutics Research Centre, National Research Council Canada, Ottawa, ON K1A 0R6, Canada
Interests: immunology; immunotherapy; cell therapy; vaccinilogy
Dr. Alaka Mullick

E-Mail Website
Guest Editor
Human Health Therapeutics Research Centre, National Research Council Canada, Montreal, QC H4P 2R2, Canada
Interests: adeno-associated virus; gene expression; gene therapy; biomanufacturing

Special Issue Information

Dear Colleagues,

Gene delivery is a process by which foreign nucleic acid is introduced into a host cell. Although this is thought of synonymously with gene therapy, which entails the replacement of a defective gene in an effort to treat or stop disease, gene delivery also encompasses the delivery of a wide range of therapeutic agents, including vaccine antigens into cells for prevention and/or treatment of disease. Gene delivery can be accomplished using mechanical (electroporation, micro-injection, biolistic) or biological (viral or non-viral) means. Apart from the delivery of DNA vaccines using electroporation and biolistics, the primary means of gene delivery in vivo is via the use of biological methods. Viral vector-mediated gene delivery was the primary mode of gene delivery until the success of lipid nanoparticle-mediated delivery of mRNA coding for SARS-Cov2 spike protein for vaccines against COVID-19.

Viral vectors are a powerful tool for gene delivery due to their intrinsic ability to enter cells and replicate their genetic material. Adeno and adeno-associated viral vectors are two of the main types of viral vectors used in gene delivery to date. This Special Issue focuses the current status of and new developments in the use of adenoviral and adeno-associated viral vectors in gene delivery.

As such, we welcome authors to submit original research articles or review articles outlining pre-clinical and clinical development of these vectors for delivery of genetic material for the prevention or treatment of disease. 

Dr. Risini Weeratna
Dr. Alaka Mullick
Guest Editors

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. Pharmaceuticals is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 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

  • AAV
  • Adenovirus
  • biomanufacturing
  • gene therapy
  • vaccine
  • targeting

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Published Papers (3 papers)

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Research

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23 pages, 10884 KiB  
Article
Engineering Rapalog-Inducible Genetic Switches Based on Split-T7 Polymerase to Regulate Oncolytic Virus-Driven Production of Tumour-Localized IL-12 for Anti-Cancer Immunotherapy
by Nikolas T. Martin, Mathieu J. F. Crupi, Zaid Taha, Joanna Poutou, Jack T. Whelan, Sydney Vallati, Julia Petryk, Ricardo Marius, Bradley Austin, Taha Azad, Mason Boulanger, Tamara Burgess, Ilson Sanders, Camille Victoor, Bryan C. Dickinson, Jean-Simon Diallo, Carolina S. Ilkow and John C. Bell
Pharmaceuticals 2023, 16(5), 709; https://doi.org/10.3390/ph16050709 - 7 May 2023
Cited by 4 | Viewed by 3831
Abstract
The approval of different cytokines as anti-neoplastic agents has been challenged by dose-limiting toxicities. Although reducing dose levels affords improved tolerability, efficacy is precluded at these suboptimal doses. Strategies combining cytokines with oncolytic viruses have proven to elicit potent survival benefits in vivo, [...] Read more.
The approval of different cytokines as anti-neoplastic agents has been challenged by dose-limiting toxicities. Although reducing dose levels affords improved tolerability, efficacy is precluded at these suboptimal doses. Strategies combining cytokines with oncolytic viruses have proven to elicit potent survival benefits in vivo, despite promoting rapid clearance of the oncolytic virus itself. Herein, we developed an inducible expression system based on a Split-T7 RNA polymerase for oncolytic poxviruses to regulate the spatial and temporal expression of a beneficial transgene. This expression system utilizes approved anti-neoplastic rapamycin analogues for transgene induction. This treatment regimen thus offers a triple anti-tumour effect through the oncolytic virus, the induced transgene, and the pharmacologic inducer itself. More specifically, we designed our therapeutic transgene by fusing a tumour-targeting chlorotoxin (CLTX) peptide to interleukin-12 (IL-12), and demonstrated that the constructs were functional and cancer-selective. We next encoded this construct into the oncolytic vaccinia virus strain Copenhagen (VV-iIL-12mCLTX), and were able to demonstrate significantly improved survival in multiple syngeneic murine tumour models through both localized and systemic virus administration, in combination with rapalogs. In summary, our findings demonstrate that rapalog-inducible genetic switches based on Split-T7 polymerase allow for regulation of the oncolytic virus-driven production of tumour-localized IL-12 for improved anti-cancer immunotherapy. Full article
(This article belongs to the Special Issue AdV and AAV Mediated Gene Delivery)
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12 pages, 1613 KiB  
Article
rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs
by Guoshuai Tian, Chunyu Cao, Shuyue Li, Wei Wang, Ye Zhang and Yafeng Lv
Pharmaceuticals 2023, 16(4), 624; https://doi.org/10.3390/ph16040624 - 20 Apr 2023
Cited by 3 | Viewed by 2240
Abstract
Krabbe disease is a rare neurodegenerative fatal disease. It is caused by deficiency of the lysosomal enzyme galactocerebrosidase (GALC), which results in progressive accumulation of galactolipid substrates in myelin-forming cells. However, there is still a lack of appropriate neural models and effective approaches [...] Read more.
Krabbe disease is a rare neurodegenerative fatal disease. It is caused by deficiency of the lysosomal enzyme galactocerebrosidase (GALC), which results in progressive accumulation of galactolipid substrates in myelin-forming cells. However, there is still a lack of appropriate neural models and effective approaches for Krabbe disease. We generated induced pluripotent stem cells (iPSCs) from a Krabbe patient previously. Here, Krabbe patient-derived neural stem cells (K-NSCs) were induced from these iPSCs. By using nine kinds of recombinant adeno-associated virus (rAAV) vectors to infect K-NSCs, we found that the rAAV2 vector has high transduction efficiency for K-NSCs. Most importantly, rAAV2-GALC rescued GALC enzymatic activity in K-NSCs. Our findings not only establish a novel patient NSC model for Krabbe disease, but also firstly indicate the potential of rAAV2-mediated gene therapy for this devastating disease. Full article
(This article belongs to the Special Issue AdV and AAV Mediated Gene Delivery)
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Review

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33 pages, 1138 KiB  
Review
Recent Advances in the Development of Adenovirus-Vectored Vaccines for Parasitic Infections
by Cal Koger-Pease, Dilhan J. Perera and Momar Ndao
Pharmaceuticals 2023, 16(3), 334; https://doi.org/10.3390/ph16030334 - 22 Feb 2023
Cited by 5 | Viewed by 3874
Abstract
Vaccines against parasites have lagged centuries behind those against viral and bacterial infections, despite the devastating morbidity and widespread effects of parasitic diseases across the globe. One of the greatest hurdles to parasite vaccine development has been the lack of vaccine strategies able [...] Read more.
Vaccines against parasites have lagged centuries behind those against viral and bacterial infections, despite the devastating morbidity and widespread effects of parasitic diseases across the globe. One of the greatest hurdles to parasite vaccine development has been the lack of vaccine strategies able to elicit the complex and multifaceted immune responses needed to abrogate parasitic persistence. Viral vectors, especially adenovirus (AdV) vectors, have emerged as a potential solution for complex disease targets, including HIV, tuberculosis, and parasitic diseases, to name a few. AdVs are highly immunogenic and are uniquely able to drive CD8+ T cell responses, which are known to be correlates of immunity in infections with most protozoan and some helminthic parasites. This review presents recent developments in AdV-vectored vaccines targeting five major human parasitic diseases: malaria, Chagas disease, schistosomiasis, leishmaniasis, and toxoplasmosis. Many AdV-vectored vaccines have been developed for these diseases, utilizing a wide variety of vectors, antigens, and modes of delivery. AdV-vectored vaccines are a promising approach for the historically challenging target of human parasitic diseases. Full article
(This article belongs to the Special Issue AdV and AAV Mediated Gene Delivery)
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