Current pharmaceutical design最新文献

{"title":"Machine Learning-Directed Explainable Artificial Intelligence and Bayesian Predictions of Warfarin Therapeutic Dose for Orthopedic Indications.","authors":"Kannan Sridharan, Gowri Sivaramakrishnan","doi":"10.2174/0113816128507764260910161559","DOIUrl":"https://doi.org/10.2174/0113816128507764260910161559","url":null,"abstract":"<p><strong>Introduction: </strong>Warfarin, a cornerstone anticoagulant for preventing venous thromboembolism following orthopedic procedures such as total hip and knee arthroplasty, is challenged due to a narrow therapeutic window and inter-patient variability. We aimed to apply machine learning (ML) algorithms to predict stable therapeutic warfarin doses in patients with orthopedic indications and to understand the model decision-making using explainable artificial intelligence.</p><p><strong>Materials and methods: </strong>This was an observational study carried out on patients who received warfarin for orthopedic indications. The dataset was partitioned into training (80%) and testing (20%) sets. Five ML algorithms were implemented and compared: elastic net, random forest (RF), support vector machine (SVM), gradient boosting machine, and XGBoost. The following metrics were used for assessing the model performance: mean absolute error (MAE), root mean squared error (RMSE), mean absolute percentage error (MAPE), R², and the percentage of predictions within 20% of the actual dose (P20). The predictions from the best ML model were analyzed using SHAP. Uncertainty intervals were estimated using Bayesian linear regression. Exploratory subgroup analyses were carried out across age groups, gender, genetic, racial, and dose strata.</p><p><strong>Results: </strong>The mean therapeutic warfarin dose was 34.2 ± 18.7 mg/week. Random forest (RF) was observed to have the lowest MAE (5.84 mg/week) and RMSE (7.63 mg/week) with the highest R² (0.86) following bootstrap validation. These findings should be considered exploratory and hypothesis-generating. SHAP analysis identified VKORC1 G/G (positive contribution) and VKORC1 A/A (negative contribution) as the most influential predictors, followed by CYP2C9 variants and anthropometric factors. SHAP analysis revealed age group 30-39 years and CYP2C9 *1/*1 genotype were the main sources of model miscalibration, as observed by their significantly different values between concordant and discordant predictions. Bayesian analysis confirmed positive linear relationships for height and weight with dose requirements but demonstrated inferior predictive performance (R²=0.38, P20=30.77%). Exploratory subgroup analyses revealed better performance in patients aged 50-59 years and African Americans, while the high-dose category (>75th percentile) and patients aged 60-69 years presented the greatest predictive difficulty.</p><p><strong>Discussion: </strong>The ML models demonstrated moderate predictive accuracy for warfarin dosing, with genetic factors outperforming anthropometrics. Varied performance across age and racial groups possibly indicates the need for subgroup-specific refinement rather than a one-size-fits-all approach.</p><p><strong>Conclusion: </strong>Machine learning models incorporating genetic and clinical factors can predict warfarin doses in orthopedic patients with moderate accuracy, with genotype variants ","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149080027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Solid and Nanostructured Lipid Carriers for Plant-Derived Neuroprotective Agents: Advances and Future Perspectives.","authors":"Esra Küpeli, Methiye Mancak, Egemen Cakirli, Hilal Bardakci, Tugba Buse Avci, Samet Ozdemir","doi":"10.2174/0113816128509583260914134550","DOIUrl":"https://doi.org/10.2174/0113816128509583260914134550","url":null,"abstract":"<p><p>Neurodegenerative disorders, particularly Alzheimer's disease (AD) and Parkinson's disease (PD), continue to pose a substantial therapeutic challenge, largely due to progressive neuronal degeneration and the limited effectiveness of currently available treatments. Plant-derived bioactive compounds-including polyphenols, alkaloids, terpenoids, and flavonoids-have shown notable neuroprotective properties. However, their clinical use remains constrained by low bioavailability and limited ability to cross the blood-brain barrier (BBB). Lipid-based nanocarrier systems, especially solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs), have emerged as promising alternatives for improving delivery efficiency. This review evaluates recent advances in SLN and NLC formulations developed for phytochemical delivery in neurodegenerative conditions, focusing on formulation approaches, production methods, and the physicochemical factors that govern system performance. Preclinical findings consistently indicate enhanced solubility, improved stability, and more effective brain targeting with these systems. Even so, several challenges continue to impede their clinical translation. In particular, issues related to formulation reproducibility, scalability of production, and long-term safety remain unresolved. Alternative administration routes such as intranasal delivery offer a potential strategy to bypass the BBB, although their practical feasibility still requires further confirmation. SLN- and NLC-based systems offer a promising approach, although their clinical development is still ongoing. Future efforts should focus on establishing standardized formulation protocols, conducting rigorous in vivo studies, and ensuring alignment with regulatory requirements to facilitate successful clinical translation.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149079960","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emulsomes in Cancer Therapy: Emerging Advances in Nanomedicine.","authors":"Chesta Gupta, Shikha Baghel Chauhan, Indu Singh","doi":"10.2174/0113816128439151260914074133","DOIUrl":"https://doi.org/10.2174/0113816128439151260914074133","url":null,"abstract":"<p><p>Cancer is a leading cause of death worldwide and continues to pose significant challenges in treatment. Poor drug solubility, non-specific distribution, MDR, and systemic toxicity remain some of the major concerns regarding treatment efficacy. Emulsomes represent a promising drug delivery platform that helps overcome some of these limitations, acting as hybrid nanocarrier systems that incorporate the advantages of liposomes and solid lipid nanoparticles. This article provides an overview of the composition, structure, and physicochemical properties of emulsomes, their advantages over conventional nanocarriers, and a detailed discussion of their role in cancer treatment. The authors discuss a variety of medicinal substances, including chemotherapeutic drugs and natural chemicals, that are delivered using emulsomes. Current research highlights the potential application of emulsomes across a wide range of cancers, including colon, brain, lung, prostate, breast, and hematologic malignancies. These features are compared with those of other nanocarriers with respect to composition, size (nm), stability, tumor-targeting capacity, biocompatibility, and toxicity. In addition, this work further highlights the current in vitro and in vivo evaluations of emulsomes, along with a thorough discussion of formulation challenges, scaling issues, and regulatory considerations. Lastly, this study covers recent advancements and potential future applications, including AI-enabled surface modification for targeted distribution, personalized medication, theragnostic applications, and emulsome design optimization. Overall, this study provides a strong foundation for understanding the current and future potential of emulsomes in managing various types of cancer.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149036790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Next-Generation Hybrid Nano-formulations for Polyphenolic Nutraceuticals in Cancer Therapy: An Overview.","authors":"Saloni Bhandari, Bhuvanesh Baniya, Supriya Singh, Deepak Choudhary, C P Jain, Garima Joshi","doi":"10.2174/0113816128501808260903071631","DOIUrl":"https://doi.org/10.2174/0113816128501808260903071631","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Introduction: &lt;/strong&gt;Cancer remains the leading cause of death globally. Conventional cancer treatments are capable of effectively targeting tumor cells; however, they might trigger immunosuppression, multidrug resistance, and harm healthy cells. Polyphenols are nutraceuticals-based naturally occurring phytochemicals with promising nutraceutical, antioxidant, antineoplastic, anti-inflammatory, and free-radical scavenging properties. This review highlights strategies for encapsulating them within hybrid nanostructures to overcome their limitations of poor aqueous solubility, low bioavailability, and lack of target specificity. Moreover, it provides insights into the major molecular signaling pathways underlying the anticancer activity of polyphenols and their hybrid nanoformulations.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A comprehensive literature review was conducted from accredited databases, including Google Scholar, Scopus, PubMed, and Web of Science (1996 to present), on naturally occurring polyphenols and their encapsulation into hybrid formulations. Emphasis was placed on hybrid nanocarriers, with keywords including dendrimers, micelles, polymers, mesoporous silica, gold nanoparticles, and carbon nanotubes, prepared by combining inorganic nanoparticles with organic lipids or polymers.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Polyphenols exhibit promising nutraceutical, antioxidant, antineoplastic, anti-inflammatory, and freeradical- scavenging properties, along with significant potential to modulate oxidative stress, apoptosis, metabolism, proliferation, inflammation, angiogenesis, and tumor signaling pathways, including MAPK, PI3K/Akt/ mTOR, Wnt/β-catenin, and STAT3. Encapsulation of polyphenols into Hybrid Nanoformulations (HNs) yields a synergistic system that significantly improves stability, bioavailability, targeted delivery, and effectiveness in cancer treatment. HNs further demonstrated the potential to integrate the advantages of multiple delivery systems, thereby improving therapeutic outcomes and reducing systemic toxicity.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;The available studies highlight hybrid nanoformulations as a versatile platform to maximize the therapeutic potential of polyphenols in cancer treatment. Integrating the advantages of different nanocarrier systems offers new strategies for developing more effective and targeted cancer therapies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;This review presents a promising approach to cancer therapy by integrating polyphenols into hybrid nanoformulations, addressing key limitations of conventional polyphenol delivery and thereby enhancing their anticancer potential through multiple molecular pathways. However, despite the encouraging benefits, no polyphenol-based nanoformulation has yet been approved by the US FDA for cancer therapy due to limited clinical evidence, challenges associated with large-scale manufacturing, regulatory concerns, and long-term safety assessment. Further preclini","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149015764","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chitin, Chitosan and Chitosan oligosaccharides-based Biomaterials: A Comprehensive Review on Biomedical Applications.","authors":"Kavita Verma, Priyanka, Neelam Yadav","doi":"10.2174/0113816128490186260908051930","DOIUrl":"https://doi.org/10.2174/0113816128490186260908051930","url":null,"abstract":"<p><p>The use of chitin, chitosan, and Chitooligosaccharides (COS) has advanced rapidly in recent years due to their exceptional physicochemical and biological characteristics. Because of their biocompatibility, biodegradability, low toxicity, and functional flexibility, chitin and chitosan have attracted significant interest from the biomedical, pharmaceutical, agrochemical, culinary, and cosmetic sectors. Tissue engineering, wound healing, medication delivery, and the creation of bioactive substances with antibacterial and antiinflammatory properties are among their uses. When compared to their parent polymers, chitooligosaccharides- low-molecular-weight derivatives of chitosan with a degree of polymerization below 20-show better biological activity and water solubility. Numerous pharmacological benefits, including antioxidant, antiinflammatory, anti-obesity, anti-cancer, neuroprotective, cardioprotective, and anti-aging properties, are demonstrated by COS. Although preclinical research presently provides the majority of the data, these bioactivities make COS attractive candidates for treatments in a variety of disorders. Chemical processes that are frequently toxic to the environment and challenging to regulate with respect to polymerization and acetylation levels have historically been used to produce chitosan and COS. In order to produce well-defined products with enhanced functional properties, recent research has concentrated on enzymatic methods utilizing chitinases, chitosanases, and chitin deacetylases. This review emphasizes recent advancements in our understanding of the structural, physicochemical, and bioactive aspects of chitin-based derivatives and discusses both traditional and novel synthesis methods. Additionally, it highlights the necessity for further in vivo and translational research to fully realize the therapeutic potential of chitosan-based bioactive molecules by examining their creative uses in a variety of fields.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149012006","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Smart Cosmetic Formulation: A Systematic Review of AI-Powered Approaches.","authors":"Divyanshu Srivastava, Shikha Baghel Chauhan, Rajan Swami, Indu Singh","doi":"10.2174/0113816128476702260829100829","DOIUrl":"https://doi.org/10.2174/0113816128476702260829100829","url":null,"abstract":"<p><strong>Introduction: </strong>Cosmetics and cosmeceuticals serve both aesthetic and hygienic functions, yet traditional formulation approaches remain resource-intensive, empirical in nature, and limited in their capacity for personalisation. The integration of artificial intelligence (AI) and machine learning (ML) into cosmetic science offers a transformative alternative, enabling faster ingredient screening, predictive safety and efficacy assessment, and individualised product design.</p><p><strong>Methods: </strong>A systematic literature search was conducted across PubMed/MEDLINE, Google Scholar, ScienceDirect, IEEE Xplore, ResearchGate, and Google Patents, covering publications from 2013 to March 2025. Search terms spanned AI-driven formulation design, predictive modelling, ingredient optimisation, toxicity prediction, personalised skincare, and regulatory considerations. Peer-reviewed articles, technical studies, and patents meeting pre-defined inclusion criteria were selected.</p><p><strong>Results: </strong>This review systematically examines AI and ML applications across the cosmetic formulation pipeline from ingredient selection and stability prediction to toxicity screening, safety assessment, and personalised recommendation systems.</p><p><strong>Discussion: </strong>Unlike previous reviews that primarily address diagnostic or imaging applications, this work's particular emphasis is on formulation science: predictive modelling of preservative efficacy, in silico toxicity assessment using quantitative structure-activity relationship (QSAR) and read-across tools, AI-assisted skin analysis, and the algorithmic frameworks underpinning these advances. Comparative discussion of convolutional neural networks (CNN), artificial neural networks (ANN), support vector machines (SVM), random forests, and gradient boosting approaches contextualises their strengths and limitations within cosmetic applications.</p><p><strong>Conclusion: </strong>AI and ML are reshaping cosmetic formulation from an empirical, trial-and-error process into a data-driven, predictive discipline. While challenges related to data quality, algorithmic bias, regulatory harmonisation, and validation remain, the trajectory indicates that AI will become an indispensable partner in designing safer, more effective, and more personalised cosmetic products.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149011965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Yindan-Pinggan Capsules Alleviate Steatotic Liver Disease Through Modulating Multiple Signaling Pathways in Mice.","authors":"Yong Zhang, Bei Li, Junjie Zhang, Hanshi Guo, Guifang Lin, Jianyuan Xie, Chen Zhao, Lijuan Cai, Qihong Deng, Shuhui Cai, Xinyuan Liu, Wei Xi, Pingli Mo, Fei Hong, Shicong Wang, Chundong Yu","doi":"10.2174/0113816128506569260903091707","DOIUrl":"https://doi.org/10.2174/0113816128506569260903091707","url":null,"abstract":"<p><strong>Introduction: </strong>Steatotic Liver Disease (SLD), including Alcohol-Associated Liver Disease (ALD) and Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), is one of the most prevalent chronic liver diseases globally, affecting approximately a quarter of the world's population. It poses a significant burden on global health, but effective treatments are limited. The Yindan-Pinggan (YDPG) formula is a modified traditional Chinese herbal remedy intended to treat liver diseases, but its role in SLD is unclear. This study aimed to explore the effects and mechanisms of YDPG against ALD and MASLD in mice.</p><p><strong>Methods: </strong>The efficacy of YDPG was evaluated by measuring body weight and liver index and assessing hepatic steatosis via H&E and Oil Red O staining. Commercial assay kits were used to detect indicators related to liver injury, lipid metabolism, and glucose metabolism. Intraperitoneal insulin and glucose tolerance tests were performed to assess insulin homeostasis. RT-qPCR, ELISA, and Western blot analysis were applied to measure mRNA or protein expression levels. ChIP-qPCR was performed to assess PPARγ recruitment at the promoters of its target genes.</p><p><strong>Results: </strong>YDPG markedly ameliorated ALD and MASLD in mice, with multiple mechanisms potentially contributing to its protective effects against SLD, which these mechanisms include: (1) inhibiting oxidative stress to mitigate hepatic injury via activation of Nrf2 signaling; (2) promoting bile salt excretion via activation of the hepatic FXR-BSEP axis; (3) suppressing hepatic lipid accumulation through dual activation of the intestinal FXR-FGF15 pathway in the ALD model; and (4) suppressing de novo lipogenesis via PPARγ-inhibition in the MASLD model.</p><p><strong>Discussion: </strong>This research showed that YDPG demonstrated therapeutic potential for SLD, and the underlying mechanism of YDPG against ALD included alleviation of oxidative stress, anti-inflammatory effects, activation of the intestinal FXR-FGF15 axis, and action against MASLD via inhibition of lipogenesis in hepatocytes. These findings strengthened the evidence that YDPG could be a potential treatment strategy for SLD and offered opportunities for therapeutic advances in the field.</p><p><strong>Conclusion: </strong>YDPG holds potential for the treatment of ALD, likely by activating FXR and Nrf2 signaling in hepatocytes, enhancing fatty acid oxidation through the intestinal FXR-FGF15 axis, and inhibiting hepatic PPARγ signaling, which exerts a hepatic protective effect against MASLD.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149012042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Glance at the Recent Nanoparticle-based Therapeutic Approaches Towards Glioblastoma Multiforme.","authors":"Chittathur Midhun Krishna, Rupa Mazumder, Rakhi Mishra, Anjna Rani","doi":"10.2174/0113816128483723260829092032","DOIUrl":"https://doi.org/10.2174/0113816128483723260829092032","url":null,"abstract":"<p><strong>Introduction: </strong>Glioblastoma multiforme, abbreviated as GBM, is a malignant form of brain cancer with no definitive cure or method of prevention and an unknown cellular origin that prevents a comprehensive understanding of its complex pathophysiology. Although still in its early stages, nanotechnology-based therapeutic strategies have emerged as a promising solution for overcoming the challenges of drug delivery to the brain.</p><p><strong>Objective: </strong>This manuscript presents a thorough overview of glioblastoma, its underlying biology, and the drawbacks of current treatment options. It also discusses the new strategies aimed at addressing the challenges and limitations of traditional treatment methods for glioblastoma.</p><p><strong>Methodology: </strong>The literature and data covered in this review were collected from research articles published in the last 5 years, with some selected highly relevant earlier works also. The PubMed, Google Scholar, and ClinicalTrials.gov websites were used for data search using keywords including glioblastoma, nanoparticles, nanomedicine, cancer nanotherapy, drug delivery, and blood-brain barrier (BBB), either individually or in combination, to identify relevant studies.</p><p><strong>Conclusion: </strong>The review integrates recent advancements in nanomedicine-based approaches for glioblastoma, highlights their existing challenges, and outlines the future directions for emerging approaches as a promising and actively investigated strategy for targeted drug delivery and therapeutic efficacy.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149011975","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel Therapeutic Frontiers in Duchenne Muscular Dystrophy: Gene Therapy, Exon Skipping, and Stem Cell Approaches.","authors":"Shahjad Ansari, Mohd Amaan Saifi, Sarfaraz Ahmed, Arun Kumar, Muneer Alam, Deepak Teotia, Sisir Nandi","doi":"10.2174/0113816128486412260828111209","DOIUrl":"https://doi.org/10.2174/0113816128486412260828111209","url":null,"abstract":"<p><p>Duchenne Muscular Dystrophy (DMD) is a chronic X-linked recessive neuromuscular disorder with a prevalence of 1 in 3,500 to 5,000 live male births worldwide. Alterations in the dystrophin gene, one of the biggest known human genes with 2.5 million base pairs and 79 exons, located at Xp21.1, are responsible for the disease. The dystrophin protein, an essential structural component that links the muscle cell cytoskeleton actin to the extracellular matrix through the dystrophin-associated protein complex, is either lacking or dysfunctional due to these mutations. Dystrophin deficiency compromises muscle membrane stability, leading to gradual deterioration of muscle fibers, fibrosis, and replacement by adipose tissue. Numerous disrupted physiological pathways, such as calcium dysregulation, faulty nitric oxide signaling, altered PI3K-Akt-mTOR growth signaling, chronic inflammatory responses, and oxidative stress, are implicated in the pathophysiology of DMD. Affected males clinically demonstrate early childhood muscular weakness; characteristic signs such as Gower's maneuver and calf hypertrophy; increasing ambulation loss by adolescence; and potentially lethal cardiorespiratory problems that generally manifest in the third decade of life. Corticosteroids remain the primary therapeutic modality, significantly improving ambulation and quality of life; nevertheless, extended use entails severe adverse effects. Recent treatment advancements provide renewed optimism via diverse innovative strategies, namely CRISPR-Cas9 gene editing, antisense oligonucleotide-mediated exon skipping (eteplirsen, golodirsen, viltolarsen, and casimersen), microdystrophin gene therapy using Adeno-Associated Viral (AAV) vectors, stop codon read-through therapy, and stem cell-based treatments. But difficulties with cost, immunogenicity, efficacy, and mutant specificity persist. Through multidisciplinary treatment and continuous scientific progress, individualized precision medicine that integrates therapies targeting secondary pathogenic pathways with dystrophin-restoration techniques can finally convert this devastating illness into a tolerable chronic ailment.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148966981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Nanofiber-Based Drug Delivery and Extracellular Matrix-Mimetic Platforms in Cancer Therapy: From Engineering Design to Clinical Translation.","authors":"Md Moidul Islam, Amit Kumar, Moidul Islam Judder, Md Nasiruddin Khan, Isha Dhiman, Gamal Osman Elhasan, Md Ali Mujtaba, Ameeduzzafar Zafar","doi":"10.2174/0113816128483193260824042349","DOIUrl":"https://doi.org/10.2174/0113816128483193260824042349","url":null,"abstract":"<p><p>The nanofiber-based drug delivery systems offer a high surface-area-to-volume ratio, tunable porosity, controllable degradation, and superior drug-loading capacity, making them promising platforms for precision cancer therapy. This review details the engineering design, fabrication methods and biomedical uses of nanofibers for cancer therapeutics, as well as for extracellular matrix (ECM)-mimicking tumor models. The major fabrication techniques such as electrospinning, phase separation, self-assembly, and template synthesis are critically examined, and their impact on the fiber morphology, drug packaging, and drug release are highlighted. Electrospun nanofibers closely replicate the native ECM architecture, which allows for the creation of physiologically relevant 3-D tumor models for the study of tumor progression, metastasis, and therapeutic resistance. Recent studies have shown that nanofibrous platforms can be used to deliver chemotherapeutics, genes, and combination therapies locally and in a stimulus-responsive manner, leading to increased cytotoxicity, extended release times, and decreased systemic toxicity in models of breast cancer, melanoma, pancreatic cancer, and colorectal cancer. The applications of nanofibers in precision oncology are further highlighted by advanced multifunctional systems based on magnetic nanoparticles, pH-responsive carriers, and core-shell architectures. Commercial products, patents, and current clinical trials further highlight the increasing translational importance of nanofiber technologies. However, issues in large-scale manufacturing, residual solvent toxicity, reproducibility, long-term biocompatibility and regulatory standardization remain to be clinically translated. The use of artificial intelligence, computational models and automated manufacturing could further drive the emergence of clinically translatable and scalable nanofiber-based cancer therapeutics.</p>","PeriodicalId":10845,"journal":{"name":"Current pharmaceutical design","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148966997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}