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Biomedicines
Special Issues
Human Stem Cells in Disease Modelling and Treatment
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Human Stem Cells in Disease Modelling and Treatment

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Biomedical Engineering and Materials".

Deadline for manuscript submissions: 31 May 2025 | Viewed by 9839

Special Issue Editors

Dr. Sofia M. Calado

E-Mail Website
Guest Editor
Faculty of Sciences and Technology, Universidade dos Açores, 9500-321 Ponta Delgada, São Miguel, Azores, Portugal
Interests: stem cell; iPSC; disease modelling; retinal diseases; advanced therapies; cell therapy; regenerative medicine
Special Issues, Collections and Topics in MDPI journals
Dr. Álvaro Plaza Reyes

E-Mail Website
Guest Editor
Department of Regeneration and Cell Therapy, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Avda. Américo Vespucio 24, 41092 Seville, Spain
Interests: stem cells; genome editing; cell therapy; ATMPs; retinal degeneration; AMD; stargardt; retinitis pigmentosa
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Over the last few decades, human stem cells have gained significant attention in biomedical research due to their unprecedented applications, both in terms of disease modelling and therapy. In addition, although rodents have been widely used as a model, they often fail to mirror the pathophysiology of human diseases due to species-specific differences.

This Special Issue of Biomedicines aims to highlight the most recent advances in the field of human stem cell modelling, translation, and clinical application. Topics of interest include, but are not limited to, the following:

  • Novel applications of human stem cells to study disease mechanisms;
  • The use of human stem cells in regenerative medicine;
  • Obstacles related to the cGMP compliance of human stem cells in regenerative medicine applications;
  • Other limitations in the clinical translation of human stem cells for regenerative medicine.

Dr. Sofia M. Calado
Dr. Álvaro Plaza Reyes
Guest Editors

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

  • induced pluripotent stem cells (iPSCs)
  • embryonic stem cells (ESCs)
  • disease models
  • cell therapies
  • cell therapy medicinal products (CTMPs)
  • regenerative medicine

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Published Papers (5 papers)

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Research

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17 pages, 2933 KiB  
Article
Efficacy of Engraftment and Safety of Human Umbilical Di-Chimeric Cell (HUDC) Therapy after Systemic Intraosseous Administration in an Experimental Model
by Maria Siemionow, Lucile Chambily and Sonia Brodowska
Biomedicines 2024, 12(5), 1064; https://doi.org/10.3390/biomedicines12051064 - 11 May 2024
Cited by 1 | Viewed by 1107
Abstract
Cell-based therapies hold promise for novel therapeutic strategies in regenerative medicine. We previously characterized in vitro human umbilical di-chimeric cells (HUDCs) created via the ex vivo fusion of human umbilical cord blood (UCB) cells derived from two unrelated donors. In this in vivo [...] Read more.
Cell-based therapies hold promise for novel therapeutic strategies in regenerative medicine. We previously characterized in vitro human umbilical di-chimeric cells (HUDCs) created via the ex vivo fusion of human umbilical cord blood (UCB) cells derived from two unrelated donors. In this in vivo study, we assessed HUDC safety and biodistribution in the NOD SCID mouse model at 90 days following the systemic intraosseous administration of HUDCs. Twelve NOD SCID mice (n = 6/group) received intraosseous injection of donor UCB cells (3.0 × 106) in Group 1, or HUDCs (3.0 × 106) in Group 2, without immunosuppression. Flow cytometry assessed hematopoietic cell surface markers in peripheral blood and the presence of HLA-ABC class I antigens in lymphoid and non-lymphoid organs. HUDC safety was assessed by weekly evaluations, magnetic resonance imaging (MRI), and at autopsy for tumorigenicity. At 90 days after intraosseous cell administration, the comparable expression of HLA-ABC class I antigens in selected organs was found in UCB control and HUDC therapy groups. MRI and autopsy confirmed safety by no signs of tumor growth. This study confirmed HUDC biodistribution to selected lymphoid organs following intraosseous administration, without immunosuppression. These data introduce HUDCs as a novel promising approach for immunomodulation in transplantation. Full article
(This article belongs to the Special Issue Human Stem Cells in Disease Modelling and Treatment)
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14 pages, 2126 KiB  
Article
Influence of the Tissue Collection Procedure on the Adipogenic Differentiation of Human Stem Cells: Ischemic versus Well-Vascularized Adipose Tissue
by Pallabi Pal, Abelardo Medina, Sheetal Chowdhury, Courtney A. Cates, Ratna Bollavarapu, Jon M. Person, Benjamin McIntyre, Joshua S. Speed and Amol V. Janorkar
Biomedicines 2024, 12(5), 997; https://doi.org/10.3390/biomedicines12050997 - 1 May 2024
Cited by 1 | Viewed by 1748
Abstract
Clinical and basic science applications using adipose-derived stem cells (ADSCs) are gaining popularity. The current adipose tissue harvesting procedures introduce nonphysiological conditions, which may affect the overall performance of the isolated ADSCs. In this study, we elucidate the differences between ADSCs isolated from [...] Read more.
Clinical and basic science applications using adipose-derived stem cells (ADSCs) are gaining popularity. The current adipose tissue harvesting procedures introduce nonphysiological conditions, which may affect the overall performance of the isolated ADSCs. In this study, we elucidate the differences between ADSCs isolated from adipose tissues harvested within the first 5 min of the initial surgical incision (well-vascularized, nonpremedicated condition) versus those isolated from adipose tissues subjected to medications and deprived of blood supply during elective free flap procedures (ischemic condition). ADSCs isolated from well-vascularized and ischemic tissues positively immunostained for several standard stem cell markers. Interestingly, the percent change in the CD36 expression for ADSCs isolated from ischemic versus well-vascularized tissue was significantly lower in males than females (p < 0.05). Upon differentiation and maturation to adipocytes, spheroids formed using ADSCs isolated from ischemic adipose tissue had lower triglyceride content compared to those formed using ADSCs isolated from the well-vascularized tissue (p < 0.05). These results indicate that ADSCs isolated from ischemic tissue either fail to uptake fatty acids or fail to efficiently convert those fatty acids into triglycerides. Therefore, more robust ADSCs suitable to establish in vitro adipose tissue models can be obtained by harvesting well-vascularized and nonpremedicated adipose tissues. Full article
(This article belongs to the Special Issue Human Stem Cells in Disease Modelling and Treatment)
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Review

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40 pages, 3256 KiB  
Review
In Vitro Models of Cardiovascular Disease: Embryoid Bodies, Organoids and Everything in Between
by Theodora M. Stougiannou, Konstantinos C. Christodoulou and Dimos Karangelis
Biomedicines 2024, 12(12), 2714; https://doi.org/10.3390/biomedicines12122714 - 27 Nov 2024
Viewed by 1414
Abstract
Cardiovascular disease comprises a group of disorders affecting or originating within tissues and organs of the cardiovascular system; most, if not all, will eventually result in cardiomyocyte dysfunction or death, negatively impacting cardiac function. Effective models of cardiac disease are thus important for [...] Read more.
Cardiovascular disease comprises a group of disorders affecting or originating within tissues and organs of the cardiovascular system; most, if not all, will eventually result in cardiomyocyte dysfunction or death, negatively impacting cardiac function. Effective models of cardiac disease are thus important for understanding crucial aspects of disease progression, while recent advancements in stem cell biology have allowed for the use of stem cell populations to derive such models. These include three-dimensional (3D) models such as stem cell-based models of embryos (SCME) as well as organoids, many of which are frequently derived from embryoid bodies (EB). Not only can they recapitulate 3D form and function, but the developmental programs governing the self-organization of cell populations into more complex tissues as well. Many different organoids and SCME constructs have been generated in recent years to recreate cardiac tissue and the complex developmental programs that give rise to its cellular composition and unique tissue morphology. It is thus the purpose of this narrative literature review to describe and summarize many of the recently derived cardiac organoid models as well as their use for the recapitulation of genetic and acquired disease. Owing to the cellular composition of the models examined, this review will focus on disease and tissue injury associated with embryonic/fetal tissues. Full article
(This article belongs to the Special Issue Human Stem Cells in Disease Modelling and Treatment)
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19 pages, 995 KiB  
Review
The Potential of Mesenchymal Stem Cells in Treating Spinocerebellar Ataxia: Advances and Future Directions
by Gi Beom Lee, Se Min Park, Un Ju Jung and Sang Ryong Kim
Biomedicines 2024, 12(11), 2507; https://doi.org/10.3390/biomedicines12112507 - 1 Nov 2024
Viewed by 2295
Abstract
Spinocerebellar ataxia (SCA) is a heterogeneous disorder characterized by impaired balance and coordination caused by cerebellar dysfunction. The absence of treatments approved by the U.S. Food and Drug Administration for SCA has driven the investigation of alternative therapeutic strategies, including stem cell therapy. [...] Read more.
Spinocerebellar ataxia (SCA) is a heterogeneous disorder characterized by impaired balance and coordination caused by cerebellar dysfunction. The absence of treatments approved by the U.S. Food and Drug Administration for SCA has driven the investigation of alternative therapeutic strategies, including stem cell therapy. Mesenchymal stem cells (MSCs), known for their multipotent capabilities, have demonstrated significant potential in treating SCA. This review examines how MSCs may promote neuronal growth, enhance synaptic connectivity, and modulate brain inflammation. Recent findings from preclinical and clinical studies are also reviewed, emphasizing the promise of MSC therapy in addressing the unmet needs of SCA patients. Furthermore, ongoing clinical trials and future directions are proposed to address the limitations of the current approaches. Full article
(This article belongs to the Special Issue Human Stem Cells in Disease Modelling and Treatment)
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9 pages, 2370 KiB  
Review
Ovarian Stem Cells for Women’s Infertility: State of the Art
by Krzysztof Grettka, Katarzyna Idzik, Katarzyna Lewandowska, Ksena Świętek, Simone Palini and Franco Silvestris
Biomedicines 2024, 12(6), 1139; https://doi.org/10.3390/biomedicines12061139 - 21 May 2024
Cited by 1 | Viewed by 2030
Abstract
Today, women’s infertility is considered a social disease in females, occurring not only as an effect of POF (premature ovarian failure) but also as CTRI (cancer treatment-related infertility) in oncologic patients. Several procedures for FP (fertility preservation) are currently adopted to prevent this [...] Read more.
Today, women’s infertility is considered a social disease in females, occurring not only as an effect of POF (premature ovarian failure) but also as CTRI (cancer treatment-related infertility) in oncologic patients. Several procedures for FP (fertility preservation) are currently adopted to prevent this condition, mostly based on utilization of retrieved eggs from the patients with subsequent IVF (in vitro fertilization) or cryopreservation. However, great interest has recently been devoted to OSCs (ovarian stem cells), whose isolation from female ovaries, followed by their in vitro culture, led to their maturation to OLCs (oocyte-like cells), namely, neo-oocytes comparable to viable eggs suitable for IVF. Translation of these data to FP clinical application creates new hope in the treatment of infertility. Thus, in line with the significant progress in using stem cells in the regenerative medicine field, neo-oogenesis via OSCs, which is currently unapplicable in fertility preservation procedures, will provide novel possibilities for young and adult females in motherhood programs in the future. Full article
(This article belongs to the Special Issue Human Stem Cells in Disease Modelling and Treatment)
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