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Advances in Gene and Cell Therapy 2.0
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Advances in Gene and Cell Therapy 2.0

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".

Deadline for manuscript submissions: 20 September 2024 | Viewed by 4464

Special Issue Editor

Dr. Anne-Catherine Prats

E-Mail Website
Guest Editor
UMR 1297-I2MC, Université de Toulouse, Inserm, UT3, FHU IMPACT, 31432 Toulouse, France
Interests: translation control; RNA; IRES; hypoxia; angiogenesis; cardiovascular disease; cancer; gene therapy
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The concept of gene therapy was introduced when M. Bishop and H. Varmus, laureates of the Nobel Prize in 1989, discovered that viral oncogenes are cellular genes picked up by retroviruses. Since this discovery, many efforts have been made to develop viral and non-viral vectors that bear therapeutic genes. After many years and disappointments, gene therapy is now successfully used to treat several rare monogenic pathologies, including immunological, hematological, neurodegenerative and neuromuscular diseases. Gene therapy is also an important perspective to consider for frequently occurring diseases, such as cancer and cardiovascular diseases.

Cell therapy consists of implanting cells to repair or regenerate an organ. These cells may be adult, embryonic or induced pluripotent stem cells, and are the subject of many research programs and clinical assays, in particular, in the field of cardiopathies and skin diseases. Immunotherapy treatment of cancer, using CAR-T, is also a cell therapy that is being increasingly employed.

In recent years, a technological health revolution has appeared, with the possibility of editing the genome, mainly using the CRISPR-Cas9 system. Two women, J. Doudna and E. Charpentier, received the 2020 Nobel Prize for developing this technology. Another medical revolution is the use of mRNA for transient gene transfer, as used in COVID-19 vaccines.

Following the Special Issue ”Advances in Gene and Cell Therapy 1.0”, the present Special Issue aims to update these fields, including preclinical research aspects of molecular and cell biology that design the medicine of the future.

Potential topics include, but are not limited to, the following:

  • Gene therapy (preclinical research and clinical assays);
  • Cell therapy (preclinical research and clinical assays);
  • RNA-based therapies or vaccines;
  • Immunotherapy;
  • Genome editing;
  • Development of vectors;
  • Oncolytic vectors.

Dr. Anne-Catherine Prats
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

  • gene therapy
  • cell therapy
  • immunotherapy
  • RNA
  • genome editing
  • vectorology

Related Special Issue

Published Papers (3 papers)

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Research

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14 pages, 2701 KiB  
Article
CRISPR-Cas9-Based Gene Knockout of Immune Checkpoints in Expanded NK Cells
by Tahereh Mohammadian Gol, Miso Kim, Ralph Sinn, Guillermo Ureña-Bailén, Sarah Stegmeyer, Paul Gerhard Gratz, Fatemeh Zahedipour, Alicia Roig-Merino, Justin S. Antony and Markus Mezger
Int. J. Mol. Sci. 2023, 24(22), 16065; https://doi.org/10.3390/ijms242216065 - 08 Nov 2023
Viewed by 1472
Abstract
Natural killer (NK) cell immunotherapy has emerged as a novel treatment modality for various cancer types, including leukemia. The modulation of inhibitory signaling pathways in T cells and NK cells has been the subject of extensive investigation in both preclinical and clinical settings [...] Read more.
Natural killer (NK) cell immunotherapy has emerged as a novel treatment modality for various cancer types, including leukemia. The modulation of inhibitory signaling pathways in T cells and NK cells has been the subject of extensive investigation in both preclinical and clinical settings in recent years. Nonetheless, further research is imperative to optimize antileukemic activities, especially regarding NK-cell-based immunotherapies. The central scientific question of this study pertains to the potential for boosting cytotoxicity in expanded and activated NK cells through the inhibition of inhibitory receptors. To address this question, we employed the CRISPR-Cas9 system to target three distinct inhibitory signaling pathways in NK cells. Specifically, we examined the roles of A2AR within the metabolic purinergic signaling pathway, CBLB as an intracellular regulator in NK cells, and the surface receptors NKG2A and CD96 in enhancing the antileukemic efficacy of NK cells. Following the successful expansion of NK cells, they were transfected with Cas9+sgRNA RNP to knockout A2AR, CBLB, NKG2A, and CD96. The analysis of indel frequencies for all four targets revealed good knockout efficiencies in expanded NK cells, resulting in diminished protein expression as confirmed by flow cytometry and Western blot analysis. Our in vitro killing assays demonstrated that NKG2A and CBLB knockout led to only a marginal improvement in the cytotoxicity of NK cells against AML and B-ALL cells. Furthermore, the antileukemic activity of CD96 knockout NK cells did not yield significant enhancements, and the blockade of A2AR did not result in significant improvement in killing efficiency. In conclusion, our findings suggest that CRISPR-Cas9-based knockout strategies for immune checkpoints might not be sufficient to efficiently boost the antileukemic functions of expanded (and activated) NK cells and, at the same time, point to the need for strong cellular activating signals, as this can be achieved, for example, via transgenic chimeric antigen receptor expression. Full article
(This article belongs to the Special Issue Advances in Gene and Cell Therapy 2.0)
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Review

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28 pages, 1857 KiB  
Review
Advances in HIV Gene Therapy
by Rose Kitawi, Scott Ledger, Anthony D. Kelleher and Chantelle L. Ahlenstiel
Int. J. Mol. Sci. 2024, 25(5), 2771; https://doi.org/10.3390/ijms25052771 - 28 Feb 2024
Viewed by 1511
Abstract
Early gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, [...] Read more.
Early gene therapy studies held great promise for the cure of heritable diseases, but the occurrence of various genotoxic events led to a pause in clinical trials and a more guarded approach to progress. Recent advances in genetic engineering technologies have reignited interest, leading to the approval of the first gene therapy product targeting genetic mutations in 2017. Gene therapy (GT) can be delivered either in vivo or ex vivo. An ex vivo approach to gene therapy is advantageous, as it allows for the characterization of the gene-modified cells and the selection of desired properties before patient administration. Autologous cells can also be used during this process which eliminates the possibility of immune rejection. This review highlights the various stages of ex vivo gene therapy, current research developments that have increased the efficiency and safety of this process, and a comprehensive summary of Human Immunodeficiency Virus (HIV) gene therapy studies, the majority of which have employed the ex vivo approach. Full article
(This article belongs to the Special Issue Advances in Gene and Cell Therapy 2.0)
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22 pages, 3380 KiB  
Review
VEGF and Other Gene Therapies Improve Flap Survival—A Systematic Review and Meta-Analysis of Preclinical Studies
by Wiktor Paskal, Mateusz Gotowiec, Albert Stachura, Michał Kopka and Paweł Włodarski
Int. J. Mol. Sci. 2024, 25(5), 2622; https://doi.org/10.3390/ijms25052622 - 23 Feb 2024
Viewed by 968
Abstract
Surgical flaps are basic tools in reconstructive surgery. Their use may be limited by ischemia and necrosis. Few therapies address or prevent them. Genetic therapy could improve flap outcomes, but primary studies in this field present conflicting results. This systematic review and meta-analysis [...] Read more.
Surgical flaps are basic tools in reconstructive surgery. Their use may be limited by ischemia and necrosis. Few therapies address or prevent them. Genetic therapy could improve flap outcomes, but primary studies in this field present conflicting results. This systematic review and meta-analysis aimed to appraise the efficacy of external gene delivery to the flap for its survival in preclinical models. This review was registered with PROSPERO (CRD42022359982). PubMed, Embase, Web of Science, and Scopus were searched to identify studies using animal models reporting flap survival outcomes following any genetic modifications. Random-effects meta-analysis was used to calculate mean differences in flap survival with accompanying 95% CI. The risk of bias was assessed using the SYRCLE tool. Subgroup and sensitivity analyses were performed to ascertain the robustness of primary analyses, and the evidence was assessed using the GRADE approach. The initial search yielded 690 articles; 51 were eventually included, 36 of which with 1576 rats were meta-analyzed. VEGF gene delivery to different flap types significantly improved flap survival area by 15.66% (95% CI 11.80–19.52). Other interventions had smaller or less precise effects: PDGF—13.44% (95% CI 3.53–23.35); VEGF + FGF—8.64% (95% CI 6.94–10.34); HGF—5.61% (95% CI 0.43–10.78); FGF 3.84% (95% CI 1.13–6.55). Despite considerable heterogeneity, moderate risk of bias, and low quality of evidence, the efficacy of VEGF gene therapy remained significant in all sensitivity analyses. Preclinical data indicate that gene therapy is effective for increasing flap survival, but further animal studies are required for successful clinical translation. Full article
(This article belongs to the Special Issue Advances in Gene and Cell Therapy 2.0)
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