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Feature Papers in Gene and Cell Therapy
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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

Dr. Bernard Lebleu

E-Mail Website
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
Special Issues, Collections and Topics in MDPI journals

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

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 collection 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.

Published Papers (6 papers)

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2024

Jump to: 2022

6 pages, 208 KiB  
Opinion
Structural Changes Likely Cause Chemical Differences between Empty and Full AAV Capsids
by Caryn L. Heldt, Molly A. Skinner and Ganesh S. Anand
Biomedicines 2024, 12(9), 2128; https://doi.org/10.3390/biomedicines12092128 - 19 Sep 2024
Viewed by 1749
Abstract
Due to the success of adeno associated viruses (AAVs) in treating single-gene diseases, improved manufacturing technology is now needed to meet their demand. The largest challenge is creating a process to separate empty and full capsids. Patients received larger capsid doses than necessary [...] Read more.
Due to the success of adeno associated viruses (AAVs) in treating single-gene diseases, improved manufacturing technology is now needed to meet their demand. The largest challenge is creating a process to separate empty and full capsids. Patients received larger capsid doses than necessary due to the presence of empty capsids. By enabling the better separation of empty and full capsids, patients would receive the greatest therapeutic benefit with the least amount of virus capsids, thus limiting potential side effects from empty capsids. The two most common empty/full separation methods used in downstream processing are ultracentrifugation and anion exchange chromatography. Both processes have limitations, leading to a need for the identification of other structural differences that can be exploited to separate empty and full capsids. Here, we describe four possible theories of the structural changes that occur when AAV capsids envelop a genome. These theories include conformational changes occurring due to either the expansion or contraction of the capsid in the presence of nucleic acids, the constraining of the N-terminus into the five-fold pore when the genome is present, and the increased number of VP3 proteins in full capsids. These theories may reveal structural differences that can be exploited to separate full and empty capsids during manufacturing. Full article
13 pages, 3461 KiB  
Article
DCTPP1 Expression as a Predictor of Chemotherapy Response in Luminal A Breast Cancer Patients
by Juan P. Muñoz, Diego Soto-Jiménez and Gloria M. Calaf
Biomedicines 2024, 12(8), 1732; https://doi.org/10.3390/biomedicines12081732 - 2 Aug 2024
Viewed by 893
Abstract
Breast cancer (BRCA) remains a significant global health challenge due to its prevalence and lethality, exacerbated by the development of resistance to conventional therapies. Therefore, understanding the molecular mechanisms underpinning chemoresistance is crucial for improving therapeutic outcomes. Human deoxycytidine triphosphate pyrophosphatase 1 (DCTPP1) [...] Read more.
Breast cancer (BRCA) remains a significant global health challenge due to its prevalence and lethality, exacerbated by the development of resistance to conventional therapies. Therefore, understanding the molecular mechanisms underpinning chemoresistance is crucial for improving therapeutic outcomes. Human deoxycytidine triphosphate pyrophosphatase 1 (DCTPP1) has emerged as a key player in various cancers, including BRCA. DCTPP1, involved in nucleotide metabolism and maintenance of genomic stability, has been linked to cancer cell proliferation, survival, and drug resistance. This study evaluates the role of DCTPP1 in BRCA prognosis and chemotherapy response. Data from the Cancer Genome Atlas Program (TCGA), Genotype-Tissue Expression (GTEx), and Gene Expression Omnibus (GEO) repositories, analyzed using GEPIA and Kaplan–Meier Plotter, indicate that high DCTPP1 expression correlates with poorer overall survival and increased resistance to chemotherapy in BRCA patients. Further analysis reveals that DCTPP1 gene expression is up-regulated in non-responders to chemotherapy, particularly in estrogen receptor (ER)-positive, luminal A subtype patients, with significant predictive power. Additionally, in vitro studies show that DCTPP1 gene expression increases in response to 5-fluorouracil and doxorubicin treatments in luminal A BRCA cell lines, suggesting a hypothetical role in chemoresistance. These findings highlight DCTPP1 as a potential biomarker for predicting chemotherapy response and as a therapeutic target to enhance chemotherapy efficacy in BRCA patients. Full article
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28 pages, 13803 KiB  
Article
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells
by Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond, Dagmar Berghuis, Arjan C. Lankester, Isabelle André, Richard Gabriel, Marina Cavazzana, Kasrin Pike-Overzet, Frank J. T. Staal and Chantal Lagresle-Peyrou
Biomedicines 2024, 12(7), 1495; https://doi.org/10.3390/biomedicines12071495 - 5 Jul 2024
Cited by 1 | Viewed by 1736
Abstract
Recombinase-activating gene (RAG)-deficient SCID patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. The two RAG genes act as a required dimer to initiate gene recombination. Gene therapy is a valid treatment alternative for [...] Read more.
Recombinase-activating gene (RAG)-deficient SCID patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. The two RAG genes act as a required dimer to initiate gene recombination. Gene therapy is a valid treatment alternative for RAG-SCID patients who lack a suitable bone marrow donor, but developing such therapy for RAG1/2 has proven challenging. Using a clinically approved lentiviral vector with a codon-optimized RAG1 gene, we report here preclinical studies using CD34+ cells from four RAG1-SCID patients. We used in vitro T cell developmental assays and in vivo assays in xenografted NSG mice. The RAG1-SCID patient CD34+ cells transduced with the RAG1 vector and transplanted into NSG mice led to restored human B and T cell development. Together with favorable safety data on integration sites, these results substantiate an ongoing phase I/II clinical trial for RAG1-SCID. Full article
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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
Cited by 1 | Viewed by 3538
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 1357
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 11 | Viewed by 2975
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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