In drug development, reproductive and developmental toxicity tests play a crucial role in safeguarding the health of future generations. However, effective methods for predicting toxicity have not yet been established. Differences among species make toxicity testing challenging because it is difficult to extrapolate animal testing results to humans. Recently, in vitro testing using human cells has gained attention as a promising alternative, offering the potential to complement or partially replace traditional animal reproductive and developmental toxicity tests. In this review, we introduce several ‘placental barrier models’ for assessing chemical transfer from the mother to the fetus, including both conventional approaches and our latest in vitro methods.

Single-dose vaccines represent a transformative advancement in immunization, offering durable and effective immunity through a single administration. The incorporation of artificial intelligence (AI) and computational modeling has significantly enhanced the development of these vaccines. This review provides an in-depth analysis of AI’s contributions to epitope identification, immunogenicity prediction, optimization of vaccine stability, and controlled-release mechanisms. Furthermore, real-world applications are examined through case studies that demonstrate the impact of AI-driven methodologies in accelerating and refining vaccine development.

Facioscapulohumeral muscular dystrophy (FSHD) is the third most common subtype of muscular dystrophy. Patients with FSHD show patchy and slowly progressive muscle weakness, and 20% of the patients over the age of 50 are deprived of their independent ambulation and require the use of a wheelchair. To date, no therapeutic drugs have been established for this disease. However, investigations on FSHD have recently progressed, including fundamental studies and clinical trials of potential therapies. Dysregulation of the expression of the double homeobox 4 (DUX4) gene, encoding a transcription factor that shows skeletal muscle toxicity, is regarded as a causative factor for skeletal muscle injury in FSHD. DUX4 is located in the D4Z4 macrosatellite repeat units of the subtelomere on chromosome 4q35. Contraction of the repeat number of the D4Z4 macrosatellite region to ≤10 (FSHD type 1) and/or pathological gene mutation of several chromatin regulators (FSHD type 2) induce DUX4 expression in skeletal muscle tissues via hypomethylation and chromatin relaxation of the D4Z4 macrosatellite. Recently, some model animals have been reported that imitate the pathophysiology of FSHD, including dysregulation of D4Z4 epigenetic control, DUX4 overexpression, and overexpression of DUX4-related factors. Therapeutic investigations using these model animals have contributed to the elucidation of the pathophysiological mechanisms of FSHD and the development of candidate therapeutic drugs. This review provides an overview of pathophysiological and therapeutic investigations using these model animals, as well as clinical trials.

My research group recently developed a novel method for isolating extracellular vesicles (EVs) using cellulose nanofiber (CNF) sheets, termed as EV sheets, which are designed with tunable pore sizes. EVs, including exosomes, play a crucial role in cellular communication and are key targets of disease mechanisms. EV sheets capture intact EVs from minimal volumes of biofluid, such as the surfaces of organs, and store them under dry conditions until analysis. These EV sheets addressed the current limitations of EV research. CNF Sheets provide a breakthrough by effectively isolating and preserving EVs from as little as 10 µL of biofluid. In ovarian cancer models and patient samples, EV sheets revealed spatial heterogeneity in the EV profiles and identified unique miRNA signatures based on their location. This technique detects cancer-associated miRNAs at an early stage before visible symptoms such as ascites develop. Importantly, the miRNA profiles obtained from tumor surfaces differed from those obtained from the surrounding fluids, providing a better understanding of tumor-derived EVs. The EV sheet isolation method has a high EV recovery efficiency and compatibility with small RNA sequencing, demonstrating its potential to advance cancer diagnosis, staging, and treatment planning. EV sheets also offer advantages in EV preservation and transport, making them practical for clinical and research applications. In addition, this method is promising for elucidating EV biology, particularly its role in cancer progression and intercellular communication. Future studies will aim to refine this method for wider clinical use and maximize its potential.

Surface bio-engineering of polymeric nanoparticles (PNPs) represents a pivotal advancement in modern biomedical research, offering transformative potential for diagnostics, therapeutic interventions, and drug delivery. This approach focuses on functionalizing PNP surfaces with bioactive moieties, enabling precise and targeted interactions with biological systems. Despite its promise, several challenges hinder the clinical translation of surface-bioengineered PNPs. These include achieving precise control over surface modifications, maintaining stability within biological environments, and ensuring sustained, targeted interactions with cells and tissues. Furthermore, issues related to scalability, reproducibility, and long-term safety complicate their widespread adoption in medical applications. This review explores recent advancements in the design and application of surface-biofunctionalized PNPs, encompassing their use in biosensing, bioimaging, and targeted therapeutic delivery. Emphasis is placed on the molecular mechanisms driving the attachment of bioactive entities to PNP surfaces and their impact on nanoparticle stability and efficacy in both in vitro and in vivo systems. Current obstacles, such as limited control over functionalization processes and challenges in ensuring consistency across manufacturing scales, are critically analyzed. Finally, potential solutions and future research directions to overcome these barriers are discussed, highlighting the transformative possibilities of surface-bioengineered PNPs in addressing complex biomedical challenges.

Pancreatic ductal adenocarcinoma (PDAC) remains one of the most aggressive cancers with limited treatment options and a poor prognosis. Recent studies have emphasized the pivotal influence of the microbiome, particularly intratumoral and gut microbial systems, in driving PDAC progression and shaping therapeutic responses. Emerging strategies for microbiome modulation, such as fecal microbiota transplantation (FMT), have demonstrated the potential in preclinical models to enhance immune responses and inhibit tumor growth. However, significant challenges including donor variability, microbiome engraftment, and suboptimal delivery methods have hindered the transition of these strategies to clinical settings. Addressing these limitations by optimizing microbiota-based therapies is essential for harnessing their full potential as adjunct treatments for PDAC. This review delves into the intricate relationship between microbiota and PDAC, evaluates the therapeutic efficacy and limitations of FMT, and outlines future research trajectories to advance this emerging field.