Special Issue "Gene Therapy Coming of Age"

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Gene and Cell Therapy".

Deadline for manuscript submissions: closed (31 March 2021).

Special Issue Editor

Dr. Kenneth Lundstrom
E-Mail Website
Guest Editor
PanTherapeutics, Rue des Remparts 4, CH1095 Lutry, Switzerland
Interests: viral gene therapy; viral vaccines; gene expression using viral vectors; structural biology; epigenetics; nutrigenomics
Special Issues and Collections in MDPI journals

Special Issue Information

Dear Colleagues,

Gene therapy was once considered as the medicine of the future, back in the late 1990s, but it then suffered some unexpected setbacks in terms of virus-based delivery approaches. Nevertheless, gene therapy is now experiencing a second coming due to its improved efficacy and safety of vector delivery. This has led to the initiation of a large number of clinical trials and even approval of the first gene therapy drugs. Furthermore, recent progress in RNA interference, RNA delivery, and gene-editing technologies has broadened the application range of gene therapy. Today, both viral and nonviral vectors have found various applications for treatment of several diseases, such as cancer, metabolic, neurodegenerative, cardiovascular, and muscular diseases. The Special Issue on Gene Therapy in Biomedicines is therefore a particularly topical addition to the field, providing up-to-date insight into delivery vector types, safety-related issues, proof-of-principle in preclinical disease models, clinical trials in healthy volunteers and patients, and approval of gene therapy-based drugs.   

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.

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 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
  • nonviral vectors
  • RNA interference
  • gene editing
  • therapy
  • prevention
  • clinical trials

Published Papers (3 papers)

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Review

Review
Coronavirus Pandemic—Therapy and Vaccines
Biomedicines 2020, 8(5), 109; https://doi.org/10.3390/biomedicines8050109 - 03 May 2020
Cited by 28 | Viewed by 5257
Abstract
The current coronavirus COVID-19 pandemic, which originated in Wuhan, China, has raised significant social, psychological and economic concerns in addition to direct medical issues. The rapid spread of severe acute respiratory syndrome-coronavirus (SARS-CoV)-2 to almost every country on the globe and the failure [...] Read more.
The current coronavirus COVID-19 pandemic, which originated in Wuhan, China, has raised significant social, psychological and economic concerns in addition to direct medical issues. The rapid spread of severe acute respiratory syndrome-coronavirus (SARS-CoV)-2 to almost every country on the globe and the failure to contain the infections have contributed to fear and panic worldwide. The lack of available and efficient antiviral drugs or vaccines has further worsened the situation. For these reasons, it cannot be overstated that an accelerated effort for the development of novel drugs and vaccines is needed. In this context, novel approaches in both gene therapy and vaccine development are essential. Previous experience from SARS- and MERS-coronavirus vaccine and drug development projects have targeted glycoprotein epitopes, monoclonal antibodies, angiotensin receptor blockers and gene silencing technologies, which may be useful for COVID-19 too. Moreover, existing antivirals used for other types of viral infections have been considered as urgent action is necessary. This review aims at providing a background of coronavirus genetics and biology, examples of therapeutic and vaccine strategies taken and potential innovative novel approaches in progress. Full article
(This article belongs to the Special Issue Gene Therapy Coming of Age)
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Review
Viral Vector-Based Melanoma Gene Therapy
Biomedicines 2020, 8(3), 60; https://doi.org/10.3390/biomedicines8030060 - 16 Mar 2020
Cited by 3 | Viewed by 1709
Abstract
Gene therapy applications of oncolytic viruses represent an attractive alternative for cancer treatment. A broad range of oncolytic viruses, including adenoviruses, adeno-associated viruses, alphaviruses, herpes simplex viruses, retroviruses, lentiviruses, rhabdoviruses, reoviruses, measles virus, Newcastle disease virus, picornaviruses and poxviruses, have been used in [...] Read more.
Gene therapy applications of oncolytic viruses represent an attractive alternative for cancer treatment. A broad range of oncolytic viruses, including adenoviruses, adeno-associated viruses, alphaviruses, herpes simplex viruses, retroviruses, lentiviruses, rhabdoviruses, reoviruses, measles virus, Newcastle disease virus, picornaviruses and poxviruses, have been used in diverse preclinical and clinical studies for the treatment of various diseases, including colon, head-and-neck, prostate and breast cancer as well as squamous cell carcinoma and glioma. The majority of studies have focused on immunotherapy and several drugs based on viral vectors have been approved. However, gene therapy for malignant melanoma based on viral vectors has not been utilized to its full potential yet. This review represents a summary of the achievements of preclinical and clinical studies using viral vectors, with the focus on malignant melanoma. Full article
(This article belongs to the Special Issue Gene Therapy Coming of Age)
Review
Emerging Concepts and Challenges in Rheumatoid Arthritis Gene Therapy
Biomedicines 2020, 8(1), 9; https://doi.org/10.3390/biomedicines8010009 - 09 Jan 2020
Cited by 10 | Viewed by 2202
Abstract
Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient. Moreover, classical regimens are based on nontargeted systemic immune suppression and have significant side effects. Biological [...] Read more.
Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient. Moreover, classical regimens are based on nontargeted systemic immune suppression and have significant side effects. Biological treatment has advanced considerably but efficacy and specificity issues remain. Gene therapy is one of the potential future directions for RA therapy, which is rapidly developing. Several gene therapy trials done so far have been of moderate success, but experimental and genetics studies have yielded novel targets. As a result, the arsenal of gene therapy tools keeps growing. Currently, both viral and nonviral delivery systems are used for RA therapy. Herein, we review recent approaches for RA gene therapy. Full article
(This article belongs to the Special Issue Gene Therapy Coming of Age)
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