Feature Papers in Gene and Cell Therapy

A topical collection in Biomedicines (ISSN 2227-9059). This collection belongs to the section "Gene and Cell Therapy".

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Editor


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Collection Editor
Laboratory of Pathogen Host Interactions, Biology-Health Department, Montpellier University, Montpellier, France
Interests: oligonucleotides-based therapeutics; peptide (cell penetrating peptides)-based delivery vectors; antisense oligonucleotides cellular trafficking
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Topical Collection Information

Dear Colleagues,

Recent progresses in gene and cell therapies have been the incentive for this Biomedicine «Feature Papers in Gene and Cell Therapy» Topical Collection. Contributions from the editorial board members, as well as from distinguished scholars in this rapidly growing field will be acknowledged.

Among the recent achievements in the field of gene therapy, CRISPR-directed gene edition has now reached clinical translation, with promising data in the treatment of sickle-cell anemia and thransthyrethin amyloidosis. The great potential of the technology has lead to partnerships between start-up companies and large pharmaceutical companies. Likewise, the successes of mRNA-based vaccines in the SARS-CoV-2 pandemic is giving rise to many developments of mRNA-based platforms to fight human diseases through vaccination or protein-replacement therapies.

Contributions addressing fundamental or clinical researches dealing with nucleic acids-based strategies from oligonucleotides to mRNAs and DNAs, as well as key issues on viral or non-viral delivery vectors will be welcomed.

Cell therapy-based strategies also offer a great potential for the treatment of human diseases. As an example, pluripotent stem cells can be generated from somatic cells with the capacity to proliferate indefinitely and to be reprogrammed for differentiation at will. Challenges dealing with tumorigenicity and immunogenicity have had to be considered, which has delayed the onset of clinical trials until recently.

Contributions dealing with various aspects and applications of cell-based strategies will be welcomed, from animal models to human diseases clinical studies.

Dr. Bernard Lebleu
Guest Editor

Manuscript Submission Information

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Published Papers (3 papers)

2024

Jump to: 2022

33 pages, 2733 KiB  
Review
Regenerative Therapy for Corneal Scarring Disorders
by Christine Chandran, Mithun Santra, Elizabeth Rubin, Moira L. Geary and Gary Hin-Fai Yam
Biomedicines 2024, 12(3), 649; https://doi.org/10.3390/biomedicines12030649 - 14 Mar 2024
Viewed by 919
Abstract
The cornea is a transparent and vitally multifaceted component of the eye, playing a pivotal role in vision and ocular health. It has primary refractive and protective functions. Typical corneal dysfunctions include opacities and deformities that result from injuries, infections, or other medical [...] Read more.
The cornea is a transparent and vitally multifaceted component of the eye, playing a pivotal role in vision and ocular health. It has primary refractive and protective functions. Typical corneal dysfunctions include opacities and deformities that result from injuries, infections, or other medical conditions. These can significantly impair vision. The conventional challenges in managing corneal ailments include the limited regenerative capacity (except corneal epithelium), immune response after donor tissue transplantation, a risk of long-term graft rejection, and the global shortage of transplantable donor materials. This review delves into the intricate composition of the cornea, the landscape of corneal regeneration, and the multifaceted repercussions of scar-related pathologies. It will elucidate the etiology and types of dysfunctions, assess current treatments and their limitations, and explore the potential of regenerative therapy that has emerged in both in vivo and clinical trials. This review will shed light on existing gaps in corneal disorder management and discuss the feasibility and challenges of advancing regenerative therapies for corneal stromal scarring. Full article
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14 pages, 461 KiB  
Article
The Impacts of Intervertebral Disc Degeneration of the Spine, Alcohol Consumption, Smoking Tobacco Products, and Glycemic Disorders on the Expression Profiles of Neurotrophins-3 and -4
by Rafał Staszkiewicz, Dorian Gładysz, Dawid Sobański, Filip Bolechała, Edward Golec, Werner Dammermann and Beniamin Oskar Grabarek
Biomedicines 2024, 12(2), 427; https://doi.org/10.3390/biomedicines12020427 - 13 Feb 2024
Cited by 1 | Viewed by 635
Abstract
In the etiology of discogenic pain, attention is paid to the role of neurotrophic factors, which include classic neurotrophins (NTs). This study aimed to assess changes in the concentrations of NT-3 and NT-4 in the intervertebral discs (IVDs) of the lumbosacral (L/S) spine [...] Read more.
In the etiology of discogenic pain, attention is paid to the role of neurotrophic factors, which include classic neurotrophins (NTs). This study aimed to assess changes in the concentrations of NT-3 and NT-4 in the intervertebral discs (IVDs) of the lumbosacral (L/S) spine depending on the advancement of degenerative changes, pain severity, habits, and comorbidities. The study group included 113 patients who underwent microdiscectomy due to degenerative IVD disease of the L/S spine. The severity of degenerative IVD changes was assessed using the five-point Pfirrmann scale, and the pain intensity was assessed according to the visual analog scale (VAS). In turn, the control group included 81 participants from whom IVDs of the L/S section of the spine were collected post-mortem during forensic autopsy or organ donation. At the mRNA level, we noted NT-3 overexpression in the test samples compared with the controls (fold change (FC) = 9.12 ± 0.56; p < 0.05), while NT-4 transcriptional activity was decreased in the test samples compared with the controls (FC = 0.33 ± 0.07; p < 0.05). However, at the protein level, the concentrations of NT-3 (134 ± 5.78 pg/mL vs. 6.78 ± 1.17 pg/mL; p < 0.05) and NT-4 (316.77 ± 8.19 pg/mL vs. 76.92 ± 4.82 pg/mL; p < 0.05) were significantly higher in the test samples compared with the control samples. Nevertheless, the concentration of both proteins did not statistically significantly change depending on the advancement of degenerative changes and the pain intensity (p > 0.05). In addition, higher levels of NT-3 and NT-4 were noted in IVD samples from patients who consumed alcohol, smoked tobacco, were overweight/obese, or had comorbid diabetes compared with patients without these risk factors (p < 0.05). Our analysis confirmed that differences in the degenerative process of IVD, energy metabolism, and lifestyle are related to changes in the concentration profiles of NT-3 and NT-4. Full article
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2022

Jump to: 2024

16 pages, 619 KiB  
Review
Recent Advances in Extracellular Vesicle-Based Therapies Using Induced Pluripotent Stem Cell-Derived Mesenchymal Stromal Cells
by Giuliana Minani Bertolino, Marie Maumus, Christian Jorgensen and Danièle Noël
Biomedicines 2022, 10(9), 2281; https://doi.org/10.3390/biomedicines10092281 - 14 Sep 2022
Cited by 7 | Viewed by 2362
Abstract
Extracellular vesicles (EVs) are being widely investigated as acellular therapeutics in regenerative medicine applications. EVs isolated from mesenchymal stromal cells (MSCs) are by far the most frequently used in preclinical models for diverse therapeutic applications, including inflammatory, degenerative, or acute diseases. Although they [...] Read more.
Extracellular vesicles (EVs) are being widely investigated as acellular therapeutics in regenerative medicine applications. EVs isolated from mesenchymal stromal cells (MSCs) are by far the most frequently used in preclinical models for diverse therapeutic applications, including inflammatory, degenerative, or acute diseases. Although they represent promising tools as cell-free therapeutic agents, one limitation to their use is related to the batch-to-batch unreliability that may arise from the heterogeneity between MSC donors. Isolating EVs from MSCs derived from induced pluripotent stem cells (iMSCs) might allow unlimited access to cells with a more stable phenotype and function. In the present review, we first present the latest findings regarding the functional aspects of EVs isolated from iMSCs and their interest in regenerative medicine for the treatment of various diseases. We will then discuss future directions for their translation to clinics with good manufacturing practice implementation. Full article
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