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Biomedicines
Special Issues
Gene Delivery and Gene Editing
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Gene Delivery and Gene Editing

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular Genetics and Genetic Diseases".

Deadline for manuscript submissions: 31 March 2025 | Viewed by 3995

Special Issue Editor

Dr. Kosuke Ishikawa

E-Mail Website
Guest Editor
Japan Biological Informatics Consortium (JBiC), 2-4-32 Aomi, Koto-ku, Tokyo 135-8073, Japan
Interests: transposon; random screening; gene editing

Special Issue Information

Dear Colleagues,

Advances in gene delivery and editing technologies are bringing us closer to realizing fundamental and curative treatments for diseases caused by genetic mutations. Genetic recombinants can now be easily produced on a laboratory scale, serving as model cells or animals for various diseases, such as cancer, immunological disorders, and neurological conditions. These sources and technologies are instrumental in establishing model systems for signaling pathways, investigating fundamental causes, and developing treatments.

While gene delivery and editing technologies have become commonplace due to the prevalence of highly advanced methods, it is essential to recognize that ongoing development is imperative. The ultimate role of genetic engineers is to acquire a flawless technology that can, in a specific and efficient manner, revert mutated or defective genes to their healthy wild-type form without any side effects.

In this Special Issue, we aim to compile reviews addressing the current status, challenges, and issues related to gene editing, gene delivery technologies, and peripheral technologies in drug development, diagnostics, and medicine. We also expect comprehensive articles that present new insights from diverse perspectives. We are particularly interested in exploring the potential of new functional modules for CRISPR-Cas, proteins beyond typical Cas proteins, advancements in TALEN, the discovery and characterization of unknown type I or II transposons and viruses, and the medical applications stemming from these discoveries.

Dr. Kosuke Ishikawa
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. Biomedicines 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 2600 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

  • gene delivery
  • gene editing
  • novel module
  • transposon
  • genetic engineering

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Published Papers (2 papers)

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Research

19 pages, 4770 KiB  
Article
Establishment and Characterization of a Stable Producer Cell Line Generation Platform for the Manufacturing of Clinical-Grade Lentiviral Vectors
by Ane Arrasate, Igone Bravo, Carlos Lopez-Robles, Ane Arbelaiz-Sarasola, Maddi Ugalde, Martha Lucia Meijueiro, Miren Zuazo, Ana Valero, Soledad Banos-Mateos, Juan Carlos Ramirez, Carmen Albo, Andrés Lamsfus-Calle and Marie J. Fertin
Biomedicines 2024, 12(10), 2265; https://doi.org/10.3390/biomedicines12102265 - 4 Oct 2024
Viewed by 1779
Abstract
Background/Objectives: To date, nearly 300 lentiviral-based gene therapy clinical trials have been conducted, with eight therapies receiving regulatory approval for commercialization. These advances, along with the increased number of advanced-phase clinical trials, have prompted contract development and manufacturing organizations (CDMOs) to develop innovative [...] Read more.
Background/Objectives: To date, nearly 300 lentiviral-based gene therapy clinical trials have been conducted, with eight therapies receiving regulatory approval for commercialization. These advances, along with the increased number of advanced-phase clinical trials, have prompted contract development and manufacturing organizations (CDMOs) to develop innovative strategies to address the growing demand for large-scale batches of lentiviral vectors (LVVs). Consequently, manufacturers have focused on optimizing processes under good manufacturing practices (GMPs) to improve cost-efficiency, increase process robustness, and ensure regulatory compliance. Nowadays, the LVV production process mainly relies on the transient transfection of four plasmids encoding for the lentiviral helper genes and the transgene. While this method is efficient at small scales and has also proven to be scalable, the industry is exploring alternative processes due to the high cost of GMP reagents, and the batch-to-batch variability predominantly attributed to the transfection step. Methods: Here, we report the development and implementation of a reliable and clinical-grade envisioned platform based on the generation of stable producer cell lines (SCLs) from an initial well-characterized lentiviral packaging cell line (PCL). Results: This platform enables the production of VSV-G-pseudotyped LVVs through a fully transfection-free manufacturing process. Our data demonstrate that the developed platform will facilitate successful technological transfer to large-scale LVV production for clinical application. Conclusions: With this simple and robust stable cell line generation strategy, we address key concerns associated with the costs and reproducibility of current manufacturing processes. Full article
(This article belongs to the Special Issue Gene Delivery and Gene Editing)
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11 pages, 867 KiB  
Article
Gene Therapy of Thromboangiitis Obliterans with Growth Factor Plasmid (VEGF165) and Autologous Bone Marrow Cells
by Piotr Barć, Paweł Lubieniecki, Maciej Antkiewicz, Diana Kupczyńska, Jan Barć, Katarzyna Frączkowska-Sioma, Tomasz Dawiskiba, Tadeusz Dorobisz, Wojciech Sekula, Błażej Czuwara, Małgorzata Małodobra-Mazur, Dagmara Baczyńska, Wojciech Witkiewicz, Jan Paweł Skóra and Dariusz Janczak
Biomedicines 2024, 12(7), 1506; https://doi.org/10.3390/biomedicines12071506 - 6 Jul 2024
Viewed by 1231
Abstract
Background: We performed gene therapy for critical limb ischemia in thromboangiitis obliterans (TAO) by the intramuscular administration of plasmids of the vascular endothelial growth factor gene (VEGF 165) with or without bone marrow-derived stem cells. Methods: The 21 patients were randomly assigned to [...] Read more.
Background: We performed gene therapy for critical limb ischemia in thromboangiitis obliterans (TAO) by the intramuscular administration of plasmids of the vascular endothelial growth factor gene (VEGF 165) with or without bone marrow-derived stem cells. Methods: The 21 patients were randomly assigned to three groups: A—with dual therapy, cells and plasmid; B—plasmid only; and C—control group, where patients received intramuscular injections of saline. Serum VEGF levels, the ankle–brachial index (ABI), transcutaneous oxygen pressure (TcPO2), and the rest pain measured by the visual analog scale (VAS) were determined sequentially before treatment, and then 1 and 3 months after treatment. Results: In the treatment groups, serum VEGF levels increased by 4 weeks and returned to baseline values after 3 months. ABI after 12 weeks increased by an average of 0.18 in group A, and 0.09 in group B and group C. TcPO2 increased by an average of 17.3 mmHg in group A, 14.1 mmHg in group B, and 10.7 mmHg in group C. The largest pain decrease was observed in group A and averaged 5.43 less pain intensity. Conclusions: Gene therapy using the VEGF plasmid along with or without bone marrow-derived mononuclear cells administered intramuscularly into an ischemic limb in TAO is a safe and effective therapy. Full article
(This article belongs to the Special Issue Gene Delivery and Gene Editing)
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