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Animal Models for Human Diseases
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Animal Models for Human Diseases

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: 31 December 2025 | Viewed by 3391

Special Issue Editor

Dr. Yuning Song

E-Mail Website
Guest Editor
Jilin Provincial Key Laboratory of Animal Embryo Engineering, College of Veterinary Medicine, Jilin University, Changchun 130062, China
Interests: gene editing technology; development; animal models; mechanism

Special Issue Information

Dear Colleagues,

Animal models remain central to unraveling human disease mechanisms, from gene-editing tools (e.g., CRISPR-Cas9) to humanized rodent models, and promoting advancements in disease biology and therapeutic development. Recent breakthroughs in organoid cultures and AI-driven drug screening have further enhanced our understanding of disease dynamics. Global resource-sharing initiatives (e.g., phenotypic databases, standardized protocols) have accelerated the translation of basic research into clinical applications. Key challenges include physiological disparities between species, the oversimplified modeling of complex diseases, and insufficient reproducibility. Future efforts should prioritize model refinement, standardized experimental workflows, and ethical alternatives. Establishing unified global reporting guidelines and quality evaluation frameworks will be critical to enhancing reliability.

This Special Issue welcomes the submission of original research, reviews, and perspectives that address the following topics: the innovative application of animal model technologies (e.g., gene editing, humanized models); the development of strategies able to bridge preclinical–clinical translation gaps; and the standardization of experimental design and reproducibility practices.

We aim to advance research concerning animal models, enhancing integration between basic science and clinical practice.

Dr. Yuning Song
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

  • animal models
  • translational research
  • preclinical studies
  • biomedical innovation
  • reproducibility

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Published Papers (3 papers)

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Research

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17 pages, 1986 KB  
Article
OxyVita®C Hemoglobin-Based Oxygen Carrier Improves Viability and Reduces Tubular Necrosis in Ex Vivo Preserved Rabbit Kidneys
by Waldemar Grzegorzewski, Łukasz Smyk, Łukasz Puchała, Leszek Adadynski, Marta Szadurska-Noga, Joanna Wojtkiewicz, Maria Derkaczew, Jacek Wollocko, Brian Wollocko and Hanna Wollocko
Int. J. Mol. Sci. 2025, 26(19), 9266; https://doi.org/10.3390/ijms26199266 - 23 Sep 2025
Cited by 1 | Viewed by 565
Abstract
Organ transplantation has significantly progressed since the 1950s, with notable advancements in surgical procedures and immunosuppression. However, current organ preservation techniques, mainly static cold storage, have not evolved at the same pace and remain insufficient to prevent ischemic and oxidative damage. This damage, [...] Read more.
Organ transplantation has significantly progressed since the 1950s, with notable advancements in surgical procedures and immunosuppression. However, current organ preservation techniques, mainly static cold storage, have not evolved at the same pace and remain insufficient to prevent ischemic and oxidative damage. This damage, primarily caused by the cessation of aerobic metabolism, limits organ viability and transplant outcomes. In this study, we investigated whether supplementing a storage solution with a hemoglobin-based oxygen carrier (HBOC) could improve the condition of ex vivo rabbit kidneys by maintaining oxygenation and supporting aerobic metabolism. In a paired, randomized design, contralateral rabbit kidneys were preserved either in a Krebs-Ringer-based solution enriched with the polymerized hemoglobin OxyVita®C (15 g/L, p50 4–6 mmHg, MW ≈ 17 MDa, pH adjusted to 7.4) or in an HBOC-free control solution. Physicochemical characterization of OxyVita®C included oxygen equilibrium curves, zeta potential, polydispersity index, and dynamic light scattering. Biochemical markers (AST, ALT, LDH, K+, pH) and histopathological assessments were used to evaluate tissue integrity over 24 h. Histology was additionally stratified according to rinsing protocols (unwashed, NaCl single flush, triple flush), and tubular necrosis was scored by blinded pathologists. Group comparisons were analyzed using ANOVA with Tukey’s HSD test. The HBOC-enriched solution showed improved tissue preservation, higher cell survivability, and better histomorphological profiles, with significantly reduced tubular necrosis scores compared to controls. These findings suggest that active oxygen delivery via HBOCs offers a promising strategy to mitigate ischemic damage during ex vivo kidney storage. Limitations include the lack of transplantation outcomes and direct ROS quantification, which will be addressed in future work integrating hypothermic and normothermic machine perfusion. Full article
(This article belongs to the Special Issue Animal Models for Human Diseases)
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16 pages, 1985 KB  
Article
Purified Native Collagen Extracellular Matrix Plus Polyhexamethylene Biguanide Functions as a Barrier to Protect Complex Wounds in an In Vivo Model
by Rami A. Nasrallah, Kelly A. Kimmerling, James L. Cook, Chantelle C. Bozynski, Aaron M. Stoker, James P. Stannard and Katie C. Mowry
Int. J. Mol. Sci. 2025, 26(18), 9195; https://doi.org/10.3390/ijms26189195 - 20 Sep 2025
Viewed by 456
Abstract
Complex surgical wounds often necessitate repeated irrigation and debridement (I&D), resulting in substantial burdens. Purified native collagen extracellular matrix plus polyhexamethylene biguanide (PCMP) is a protective antimicrobial barrier that supports innate wound healing and was hypothesized to protect an in vivo complex wound. [...] Read more.
Complex surgical wounds often necessitate repeated irrigation and debridement (I&D), resulting in substantial burdens. Purified native collagen extracellular matrix plus polyhexamethylene biguanide (PCMP) is a protective antimicrobial barrier that supports innate wound healing and was hypothesized to protect an in vivo complex wound. Canines underwent bilateral fibular defects, which were stabilized using screws and plates pre-incubated with methicillin-resistant Staphylococcus aureus. Wounds were clinically infected seven days post-op and underwent I&D prior to application with either PCMP or non-adherent pads. Outcome assessments included radiography, wound scoring, microbial culture, histology, and gene expression. PCMP application resulted in significantly improved clinical wound healing scores at days 3 and 7, while histological analysis trended towards improved wound repair. Radiological assessments showed no loosening, confirming implant stability. Quantitative microbial assessments showed a minor reduction in bacterial load (0.5 log fold-change) on day 10. Gene expression analysis showed significant upregulation of matrix metalloproteinases and immune-modulating cytokines. Protection of the wound with PCMP resulted in improved wound scores, reduced bacterial load, and significant upregulation in key gene expression pathways compared to controls. Overall, this study suggests that PCMP effectively limited bioburden and biofilm reformation and supported wound progression in a challenging environment. These findings suggest PCMP could enhance complex wound patient outcomes. Full article
(This article belongs to the Special Issue Animal Models for Human Diseases)
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Review

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19 pages, 1029 KB  
Review
Advancing Gene Therapy for Phenylketonuria: From Precision Editing to Clinical Translation
by Inseon Yu and Jaemin Jeong
Int. J. Mol. Sci. 2025, 26(17), 8722; https://doi.org/10.3390/ijms26178722 - 7 Sep 2025
Viewed by 1925
Abstract
Phenylketonuria (PKU) is an inherited disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene that result in the amino acid phenylalanine (Phe) building up in the blood. Current therapies suggest low-Phe dietary management and (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin (BH4) therapy, which are [...] Read more.
Phenylketonuria (PKU) is an inherited disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene that result in the amino acid phenylalanine (Phe) building up in the blood. Current therapies suggest low-Phe dietary management and (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin (BH4) therapy, which are limited in efficacy and require lifelong treatment. Recent advances in gene therapy, including gene editing and viral-mediated gene delivery, produce therapeutic effects. Advancements in gene editing technologies, notably adenine base editors (ABEs) and CRISPR-based systems, in conjunction with enhanced delivery methods such as lipid nanoparticles (LNPs) and recombinant viruses, have demonstrated substantial promise in preclinical studies. This review details the pathophysiology of PKU treatment, and progress in preclinical and clinical gene therapy strategies. Emphasis is on adenine base editing using LNPs, recombinant adeno-associated virus (rAAV)-mediated gene transfer, and the translational challenges associated with these technologies. We also discuss future directions for therapeutic reach and ensuring long-term safety and efficacy. Full article
(This article belongs to the Special Issue Animal Models for Human Diseases)
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