Advances in Gene Therapy Clinical Trials for Hemophilia Care.

Gene therapy has revolutionized the therapeutic landscape for hemophilia A and B, offering the prospect for persistent endogenous production of coagulation factors VIII and IX. Recent advances in adeno-associated virus (AAV)-mediated gene transfer, particularly the approvals of valoctocogene roxaparvovec (Roctavian) and etranacogene dezaparvovec (Hemgenix), mark significant milestones in hemophilia care. This mini-review synthesizes emerging clinical data from phase I-III trials published between 2022 and 2025, emphasizing efficacy, durability, and immunogenicity profiles of leading AAV-based therapies.

Alterations in 3D Chromatin Spatial Organisation in Tumourigenesis and Therapy Resistance of Glioblastoma: The Recent Advances in Understanding Molecular Mechanisms, Clinical Implications, and Therapeutic Perspectives.

Glioblastoma (GBM) remains one of the most aggressive and lethal forms of brain cancer, characterised by profound genetic, epigenetic, and phenotypic heterogeneity. Recent advancements in high-resolution genome mapping have unveiled the critical role of three-dimensional (3D) chromatin architecture-encompassing chromatin loops, topologically associating domains, and enhancer-promoter interactions-in driving GBM tumourigenesis and therapy resistance. This review summarises recent insights into the mechanistic contribution of 3D genome reorganisation in sustaining oncogenic transcriptional programs, promoting intratumoural heterogeneity, and facilitating adaptive resistance.

A comprehensive insight into NETosis-extracellular vesicle crosstalk in autoimmune diseases.

Neutrophils are granular and polymorphonuclear cells and one of the main participants of the innate immune system, which have received considerable attention due to the discovery of neutrophil extracellular traps (NETs). Extracellular vesicles (EVs), particularly those released by immune cells such as neutrophils, have been associated with the immunopathogenesis of autoimmune diseases. Besides, studies have reported a fundamental correlation between EVs and NETosis in autoimmune diseases.

Chitosan polyplexes for targeted gene delivery: From mechanisms to clinical applications.

As a diverse natural polymer called Chitosan, it created ground-breaking advancements in nucleic acid therapeutic delivery techniques for handling essential DNA and RNA delivery hurdles. The article investigates how nucleic acids form stable polyplexes with chitosan through electrostatic bonds, as well as explores their chemical and biological properties. The review explores how molecular weight, combined with the degree of deacetylation, combined with advanced functionalization strategies, help enhance delivery results.

Exploring the bioactive constituents from spices targeting N-methyl-d-aspartate receptors: An in silico and in vitro approach to identify druggable leads against neurological disability.

N-methyl-d-aspartate (NMDA) receptors are validated druggable targets for the treatment of Alzheimer's and other associated neurological conditions, particularly in individuals with disabilities. Considering the excitotoxicity associated with NMDA receptors, which leads to neuronal damage, cognitive impairment, and limitations of current therapeutic regimens, better therapeutic candidates are required. One of the validated drug discovery approaches is computer-assisted drug discovery, supplemented by molecular docking, mechanics, and dynamics.

Engineering the Microbiome: a Novel Approach to Managing Autoimmune Diseases.

Autoimmune diseases occur when the immune system mistakenly attacks the body's own tissues, affecting millions of people and often requiring long-term treatment. Current therapies, such as immunosuppressants and biologics, help manage symptoms but can cause serious side effects. A promising new approach involves engineered microbiota-a method that modifies gut bacteria to influence immune function and potentially ease autoimmune conditions.

Optimization of ivacaftor-loaded solid lipid nanoparticles for solubility enhancement.

Background: Cystic fibrosis (CF) is a systemic disease which primarily affects pulmonary system, but also extends to different important organs to cause multitude of associated diseases, leading to rise in rate of morbidity and mortality. The present investigation is focused on the development and optimization of SLN (Solid Lipid Nanoparticles) formulation of IVF (Ivacaftor) for effective treatment of cystic fibrosis.

Methods: IVF-SLN was formulated with the help of homogenization and ultrasonication methods by incorporating Labrasol as liquid lipid, Cetyl palmitate as solid lipid and Polysorbate 20 as the surfactant.

Excellent hydrogen storage capabilities and optoelectronic attributes of XClH (Li, Na, and K) perovskite hydrides for green energy technologies.

The high hydrogen storage capacity, stability, and reversibility of perovskite hydrides make them promising materials for the energy industry. They play a vital role in sustainable energy technologies, including fuel cells and hydrogen storage systems. This work offers a comprehensive understanding of the physical attributes of XClH (X = Li, Na, and K), utilizing the DFT-based Wien2K code.

Polysaccharide- and phenolic-rich Pinus patula extract-mediated TiO₂ nanoparticles for photocatalytic degradation of methylene blue and 4-nitrophenol.

Conventional TiO₂ nanoparticle syntheses rely on high temperatures, toxic reagents and multi-step routes that impede scalability and sustainability. Here, we deliver the first green synthesis of TiO₂ nanoparticles (TiO₂ NPs) using polysaccharide- (42 mg GE g) and phenolic-rich (78 mg GAE g) Pinus patula leaf extract. GC-MS and LC-MS fingerprinting identify terpenoids, flavonoids and phenolic glycosides acting as simultaneous reducing, capping and stabilizing agents.

Hamamelitannin from Hamamelis virginiana Attenuates Ethanol-Induced Oxidative and Inflammatory Responses in Danio rerio Larvae.

Alcoholic liver disorder (ALD) is one of the most prevalent hepatic ailments worldwide, with oxidative stress and inflammation playing a vital role in disease progression. The current study intended to assess the anti-inflammatory nature of Hamamelitannin (HAM), a gallotannin from Hamamelis virginiana barks, which was predicted to possess anti-inflammatory properties based on in-silico docking analysis. To further explore its effects, we examined the therapeutic effect of HAM against ethanol-mediated inflammation using an in-vivo zebrafish larvae model.

The quercetin-serotonin transporter (SERT) connection: a new hope for depression therapy?

Quercetin, a naturally occurring flavonoid, has been found to be a potential agent for the treatment of major depressive disorder (MDD), given the complexity of its interaction with the serotonin transporter (SERT). Clinically, quercetin has direct and indirect modulatory effects as opposed to conventional selective serotonin reuptake inhibitors (SSRI), which act mainly by inhibiting SERT after a time delay and communicate with SERT through possible binding location preferences and allosteric processing, while simultaneously controlling its definite expression through anti-inflammatory and antioxidant pathways, such as the NF-kB, AMPK/SIRT-1, and Nrf2-ARE cascades. These processes assist in modifying serotonergic neurotransmission and minimizing oxidative and inflammatory strains, which are the major contributors to the pathophysiology of depression.

Advancements in Chitosan and Cellulose Nanoparticles for Stem Cell-Based Tissue Engineering.

Stem cell-based tissue engineering offers transformative solutions for regenerating damaged tissues, such as bone, cartilage, and neural tissues. Chitosan and cellulose nanoparticles have emerged as promising biomaterials for enhancing stem cell delivery and scaffold performance due to their biocompatibility, biodegradability, and tunable properties. Chitosan, with its antimicrobial and bioadhesive properties, supports stem cell adhesion and differentiation in soft tissue scaffolds.

Strategies in Tissue Regenerative Engineering for the Treatment of Human Infertility.

Regenerative engineering (RE) is the convergence of advanced stem cell science, material science, physics, clinical translation, and developmental biology to regenerate organ and complex tissue systems. It is a development of tissue engineering, which was first advanced as a method of restoration and repair of human tissue. In recent years, advances in regenerative techniques have shown promise in treating various clinical problems using existing advanced technology to harness the body's therapeutic and regenerative abilities.

Tailoring the biomarkers of Alzheimer's disease using a gut microbiome-centric approach: Preclinical, clinical, and regulatory perspectives.

Alzheimer's disease (AD), a progressive neurodegenerative disorder, poses significant therapeutic challenges due to its complex etiology and limited treatment options. Traditional pharmacotherapies targeting amyloid-β (Aβ) and cholinergic pathways offer modest benefits and are often associated with adverse effects. Emerging evidence implicates gut dysbiosis and the gut-brain axis in the pathogenesis and progression of AD.

Precision diagnosis of citrus leaf diseases using image enhancement and nonlinear fuzzy ranking ensemble approach NLFuRBe.

Citrus fruits, especially lemons, play a vital economic and nutritional role worldwide but are increasingly threatened by a wide range of diseases that diminish yield quality and quantity. Traditional manual and automated methods for disease detection requires domain expert, ample observation time, and is often ineffective during early infection stages. This paper presents a novel automated approach for the symptom based detection and classification of citrus leaf diseases using a nonlinear Fuzzy Rank-Based Ensemble (NL-FuRBE) methodology, enhanced by image quality improvement techniques.

In situ decoration of Pd NPs over methotrexate encapsulated CoFeO as a recyclable catalyst for the reduction of nitroarenes and synthesis of diaryl sulfide derivatives.

This study presents the successful immobilization of a methotrexate-palladium complex onto the surface of magnetic CoFeO nanoparticles through a simple and cost-efficient process. The environmentally friendly heterogeneous catalyst was comprehensively characterized using thermogravimetric analysis (TGA), energy dispersive spectroscopy (EDS), scanning electron microscopy (SEM), X-ray diffraction (XRD), vibrating sample magnetometry (VSM), inductively coupled plasma (ICP) atomic absorption spectroscopy, and Fourier transform infrared (FT-IR) spectroscopy. The catalyst demonstrated outstanding catalytic efficiency, achieving a 98% yield in the reduction of nitroarenes and facilitating the synthesis of diaryl sulfide derivatives under green conditions.

Resveratrol as a naturally occurring inflammasome modulator: Implications for health and disease.

Inflammation plays a crucial role in the pathophysiology of diverse diseases, ranging from chronic disorders to acute inflammatory responses. Inflammasome, a multiprotein complex that controls the activation of pro-inflammatory cytokines and initiates immunological responses, has emerged as a critical regulator of inflammation. Resveratrol, a polyphenolic substance present in grapes, berries, and peanuts, has drawn significant attention due to its potential health benefits.

CRISPR/Cas9 in colorectal cancer: Revolutionizing precision oncology through genome editing and targeted therapeutics.

Colorectal cancer (CRC) remains a significant global health challenge, necessitating advanced molecular therapies to improve outcomes. The CRISPR/Cas9 genome-editing platform has emerged as a transformative tool in CRC research, enabling precise genomic modifications to suppress tumor progression, enhance chemosensitivity, and modulate oncogenic pathways. This review highlights CRISPR/Cas9 applications in CRC models, including MC38 murine and CaCO-2 cell lines, where targeted gene edits demonstrate tumor-suppressive effects.

Targeting the Hippo/YAP Pathway: A Promising Approach for Cancer Therapy and Beyond.

Cancer represents a growing cause of death and a threat to public health worldwide; thus, there is an urgent need to understand its pathological mechanism and design effective therapies. The Hippo pathway regulates diverse cellular processes under physiological conditions; however, its dysregulation is associated with several types of cancer, including lung, pancreatic, colorectal, breast, and prostate cancer. Consequently, compounds targeting deregulated Hippo components represent potential treatments for a broad spectrum of cancers.

Metallic and lipid nanoparticles against multidrug resistant candida: advances and translational hurdles.

Introduction: Among the many ongoing difficulties, Candida infections present significant clinical hurdles due to the rapid development of resistance, recurrent episodes, and the limited effectiveness of conventional therapies. In recent decades, metallic nanoparticles (MNPs) and lipid nanoparticles (LNPs) have shown a specific impact ( > 84% Candida biofilm inhibition in pre-clinical models) by addressing the critical challenges of mitigating drug side effects and multidrug resistance (MDR).

Areas Covered: This paper provides an in-depth overview of synthesis, fabrication, mechanistic insights, preclinical and clinical practices for MNPs and LNPs, discussing and highlighting their therapeutic efficacy against resistant Candida species over traditional methods.

Epigenetic modulation in cancer drug discovery: promising targets and clinical applications.

Epigenetic modulation has emerged as a central strategy that can change the fate of tumour cells to offer more rational and precise approaches by modulating reversible changes in chromatin structure, regulating gene expression without altering DNA sequence. Many reports have indicated the contributions of abnormal epigenetic alterations, particularly DNA methylation and histone modification patterns, as well as their association with non-coding RNA interactions during cancer emergence, development or resistance to standard therapies. Ongoing studies on various inhibitors also demonstrate encouraging preclinical results and potent inhibitory activity.

CRFTS: a cluster-centric and reservation-based fault-tolerant scheduling strategy to enhance QoS in cloud computing.

Cloud systems supply different kinds of on-demand services in accordance with client needs. As the landscape of cloud computing undergoes continuous development, there is a growing imperative for effective utilization of resources, task scheduling, and fault tolerance mechanisms. To decrease the user task execution time (shorten the makespan) with reduced operational expenses, to improve the distribution of load, and to boost utilization of resources, proper mapping of user tasks to the available VMs is necessary.