Establishing Minimal Clinically Important Differences for the Cognitive and Linguistic Scale (CALS) in Pediatric Neurorehabilitation.

The Cognitive and Linguistic Scale (CALS) was developed to serially monitor cognitive recovery of children and young people after severe acquired brain injury (ABI), during inpatient rehabilitation. The CALS can be used to derive Cognitive Ability Estimates (CAE) which are Rasch-propertied (unidimensional, interval-scale) and therefore may be ideally applied for use in research including within the context of clinical trials. Here, we used established statistical distribution-based and expert consensus-based methods to estimate the Minimal Clinically Important Difference (MCID) for CAE derived from the CALS.

Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study.

Background And Purpose: This study was an open-label, dose-escalation, phase 1 clinical trial to determine the safety and dose of EN001 for patients with Duchenne muscular dystrophy (DMD). EN001, developed by ENCell, are allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells that originate at the umbilical cord, with preclinical studies demonstrating their high therapeutic efficacy for DMD.

Methods: This phase 1 clinical trial explored the safety and tolerability of EN001 as a potential treatment option for patients with DMD.

Outcomes and prognostic indicators in daratumumab-refractory multiple myeloma: a multicenter real-world study of elotuzumab, pomalidomide, and dexamethasone in 247 patients.

Background: Daratumumab-refractory multiple myeloma (Dara-R MM) presents a significant treatment challenge. This study aimed to evaluate the efficacy and survival outcomes of elotuzumab, pomalidomide, and dexamethasone (EloPd) in a large, real-world cohort of patients with Dara-R MM, with particular focus on progression-free survival (PFS) and overall survival (OS).

Materials And Methods: This retrospective analysis included 247 Dara-R MM patients treated with EloPd.

Profiling epithelial viral receptor expression in amniotic membrane and nasal epithelial cells at birth.

Introduction: Children with wheeze and asthma present with airway epithelial vulnerabilities, such as impaired responses to viral infection. It is postulated that the in utero environment may contribute to the development of airway epithelial vulnerabilities. The aims of the study were to establish whether the receptors for rhinovirus (RV), respiratory syncytial virus (RSV) and severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) are expressed in the amniotic membrane and whether the pattern of expression is similar to newborn nasal epithelium.

Tissue-Adhesive and Stiffness-Adaptive Neural Electrodes Fabricated Through Laser-Based Direct Patterning.

Recently, implantable devices for treating peripheral nerve disorders have demonstrated significant potential as neuroprosthetics for diagnostics and electrical stimulation. However, the mechanical mismatch between these devices and nerves frequently results in tissue damage and performance degradation. Although advances are made in stretchable electrodes, challenges, including complex patterning techniques and unstable performance, persist.

Registries on transcatheter edge-to-edge repair in heart failure: Current evidence and future perspectives.

Aims: Secondary mitral regurgitation (SMR) and tricuspid regurgitation (TR) are the most common valvular heart diseases in patients with heart failure (HF). Transcatheter edge-to-edge repair (TEER) devices designed for treating MR and TR have been successfully tested in randomized controlled trials, but methodological issues have often challenged their interpretation. This manuscript aimed to provide an overview of TEER registries on SMR and TR in HF, highlighting their key features, describing clinical characteristics and outcomes of patients receiving these devices, and exploring the available data limitations.

Genetically Engineered Hypoimmune Human Muscle Progenitor Cells Can Reduce Immune Rejection.

Cells face two challenges after transplantation: recognition and killing by lymphocytes, and cell apoptosis induced by the transplantation environment. Our hypoimmune cells aim to address these two challenges through editing of immunomodulatory proteins and overexpression of anti-apoptotic proteins.

Diminished DNA binding affinity of DMRT1 caused by heterozygous DM domain mutations is a cause of male infertility.

The most severe form of male infertility is idiopathic non-obstructive azoospermia (NOA), a complete sperm absence in the ejaculate. We performed exome sequencing in the Croatian infertile brothers with NOA and found a variant in DMRT1 (Doublesex and mab-3 related transcription factor 1) gene that was further assessed by the EMSA assay and molecular dynamic simulations. We additionally screened for DMRT1 mutations in 1940 infertile men diagnosed with spermatogenic failure, 644 normozoospermic controls, and 105 females with primary ovarian insufficiency (POI) recruited to the GEnetics of Male INfertility Initiative (GEMINI) or Estonian Andrology (ESTAND) cohorts.

Gut-liver axis in sepsis-associated liver injury: Epidemiology, challenges and clinical practice.

Although the liver has a remarkable regenerative capacity, sepsis-associated liver injury (SLI) is a complication often seen in intensive care units. Due to its role in immune and inflammatory regulation, the liver is particularly vulnerable during severe infections. Understanding the global prevalence, causes, and management of SLI is essential to improve outcomes and reduce healthcare costs.

Artificial intelligence (AI)-powered bibliometric analysis of global trends in mesenchymal stem cells (MSCs)-derived exosome research: 2014-2023.

Introduction: In recent years, significant progress has been made in regenerative medicine, specifically in using mesenchymal stem cells (MSCs) due to their regenerative and differentiating abilities. An exciting development in this area is the utilization of exosomes derived from MSCs, which have shown promise in tissue restoration, immune system modulation, and cancer treatment.

Objectives: This study aims to analyze global research trends and the academic impact of MSCs-derived exosomes from 2014 to 2023, providing a comprehensive overview of this emerging field.

Decellularization of fish tissues for tissue engineering and regenerative medicine applications.

Decellularization is the process of obtaining acellular tissues with low immunogenic cellular components from animals or plants while maximizing the retention of the native extracellular matrix structure, mechanical integrity and bioactivity. The decellularized tissue obtained through the tissue decellularization technique retains the structure and bioactive components of its native tissue; it not only exhibits comparatively strong mechanical properties, low immunogenicity and good biocompatibility but also stimulates neovascularization at the implantation site and regulates the polarization process of recruited macrophages, thereby promoting the regeneration of damaged tissue. Consequently, many commercial products have been developed as promising therapeutic strategies for the treatment of different tissue defects and lesions, such as wounds, dura, bone and cartilage defects, nerve injuries, myocardial infarction, urethral strictures, corneal blindness and other orthopedic applications.

Advances in adhesive hydrogels applied for ophthalmology: An overview focused on the treatment.

Adhesive hydrogels, composed of hydrophilic polymers arranged in a three-dimensional network, have emerged as a pivotal innovation in ophthalmology due to their ability to securely adhere to ocular tissues while providing sustained therapeutic effects. The eye, with its delicate structure and specific needs, presents unique challenges for drug delivery and tissue regeneration. This review explores the transformative potential of adhesive hydrogels in addressing these challenges across a range of ocular conditions, including corneal injuries, cataracts, glaucoma, vitreoretinal disorders, and ocular trauma.

Clinical-grade human dental pulp stem cells improve adult hippocampal neural regeneration and cognitive deficits in Alzheimer's disease.

Disrupted hippocampal functions and progressive neuronal loss represent significant challenges in the treatment of Alzheimer's disease (AD). How to achieve the improvement of pathological progression and effective neural regeneration to ameliorate the intracerebral dysfunctional environment and cognitive impairment is the goal of the current AD therapy. We examined the therapeutic potential of clinical-grade human derived dental pulp stem cells (hDPSCs) in cognitive function and neuropathology in AD.

Monitoring of cancer ferroptosis with [F]hGTS13, a system xc- specific radiotracer.

Glioblastoma multiforme (GBM) is the most common and aggressive primary brain tumor in adults, characterized by resistance to conventional therapies and poor survival. Ferroptosis, a form of regulated cell death driven by lipid peroxidation, has recently emerged as a promising therapeutic target for GBM treatment. However, there are currently no non-invasive imaging techniques to monitor the engagement of pro-ferroptotic compounds with their respective targets, or to monitor the efficacy of ferroptosis-based therapies.

enhancement of CD8+ T cell activity using functionalized hydrogel encapsulating tonsil-derived lymphatic endothelial cells.

This study investigates a method for programming immune cells using a biomaterial-based system, providing an alternative to traditional cell manipulation techniques. It addresses the limitations of engineered adoptive T cell therapies, such as T cell exhaustion, by introducing a gelatin-hyaluronic acid (GH-GMA) hydrogel system. We characterized tonsil mesenchymal stem cells (TMSCs), lymphatic endothelial cells (T-LECs), stimulated T-CD8 T cells (STCs), and GH-GMA biomaterials.

Loss of does not affect bone and lean tissue in zebrafish.

Human genetic studies have nominated cadherin-like and PC-esterase domain-containing 1 () as a candidate target gene mediating bone mineral density (BMD) and fracture risk heritability. Recent efforts to define the role of in bone in mouse and human models have revealed complex alternative splicing and inconsistent results arising from gene targeting, making its function in bone difficult to interpret. To better understand the role of in adult bone mass and morphology, we conducted a comprehensive genetic and phenotypic analysis of in zebrafish, an emerging model for bone and mineral research.

Change in p53 nuclear localization in response to extracellular matrix stiffness.

Chondrocytes are commonly applied in regenerative medicine and tissue engineering. Thus, the discovery of optimal culture conditions to obtain cells with good properties and behavior for transplantation is important. In addition to biochemical cues, physical and biomechanical changes can affect the proliferation and protein expression of chondrocytes.

The Condition of the Meniscus and Cartilage of the Injured Knee on Preoperative Magnetic Resonance Imaging Is a Prognostic Factor Affecting Postoperative Outcomes Following Knee Cartilage Restoration Surgery.

Purpose: To evaluate the relationship between preoperative whole-joint imaging evaluation of the knee with patient-reported outcome (PRO) measures after cartilage restoration surgery (mosaicplasty, osteochondral allograft transplantation, matrix autologous chondrocyte implantation).

Methods: We retrospectively evaluated patients who underwent knee articular cartilage restoration at our institution from 2014 to 2020. The patients' knee magnetic resonance imaging (MRI) was evaluated with the Whole-Organ Magnetic Resonance Imaging Score (WORMS) and semiquantitative synovial inflammation imaging biomarkers of the preoperative MRI.

Non-invasive in vitro NAM for the detection of reversible and irreversible eye damage after chemical exposure for GHS classification purposes (ImAi).

The potential risk of chemicals to the human eye is assessed by adopted test guidelines (TGs) for regulatory purposes to ensure consumer safety. Over the past decade, the Organization for Economic Co-operation and Development (OECD) has approved new approach methodologies (NAMs) to predict chemical eye damage. However, existing NAMs remain associated with limitations: First, no full replacement of the in vivo Draize eye test due to limited predictability of severe/mild damage was reached.

Muscle-specific miRNAs in plasma and skeletal muscle of patients with idiopathic inflammatory myopathy are modulated by disease and training.

Objectives: Objective of this work was to examine myomiR levels in plasma, skeletal muscle, and skeletal muscle cells of patients with idiopathic inflammatory myopathy (IIM), their interrelations with the disease-related clinical phenotypes and with the effects of the disease-modifying 6-month training-intervention.

Methods: Samples of vastus lateralis muscle (n = 12/13) and plasma (n = 20/21) were obtained from IIM patients and healthy controls, respectively. Muscle and plasma were obtained before and after a 6-month training-intervention in 7 patients.

Collagen as a bio-ink for 3D printing: a critical review.

The significance of three-dimensional (3D) bioprinting in the domain of regenerative medicine and tissue engineering is readily apparent. To create a multi-functional bioinspired structure, 3D bioprinting requires high-performance bioinks. Bio-inks refer to substances that encapsulate viable cells and are employed in the printing procedure to construct 3D objects progressive through successive layers.

Integrating microfluidic and bioprinting technologies: advanced strategies for tissue vascularization.

Tissue engineering offers immense potential for addressing the unmet needs in repairing tissue damage and organ failure. Vascularization, the development of intricate blood vessel networks, is crucial for the survival and functions of engineered tissues. Nevertheless, the persistent challenge of ensuring an ample nutrient supply within implanted tissues remains, primarily due to the inadequate formation of blood vessels.

The effect of NLRP3 inflammasome on cardiovascular prognosis in patients with acute coronary syndrome.

The NOD‑like receptor protein domain associated protein 3 (NLRP3) inflammasome is critical in inflammatory responses and may be a valuable prognostic biomarker in acute coronary syndrome (ACS). We aimed to investigate the association between NLRP3 inflammasome levels and short-term outcomes in patients with ACS. We enrolled 295 patients with ACS who were monitored for 6 months for major adverse cardiovascular events (MACEs).

A genome-wide scan of non-coding RNAs and enhancers for refractive error and myopia.

Refractive error (RE) and myopia are complex polygenic conditions with the majority of genome-wide associated genetic variants in non-exonic regions. Given this, and the onset during childhood, gene-regulation is expected to play an important role in its pathogenesis. This prompted us to explore beyond traditional gene finding approaches.

Defining ortholog-specific UHRF1 inhibition by STELLA for cancer therapy.

UHRF1 maintains DNA methylation by recruiting DNA methyltransferases to chromatin. In mouse, these dynamics are potently antagonized by a natural UHRF1 inhibitory protein STELLA, while the comparable effects of its human ortholog are insufficiently characterized, especially in cancer cells. Herein, we demonstrate that human STELLA (hSTELLA) is inadequate, while mouse STELLA (mSTELLA) is fully proficient in inhibiting the abnormal DNA methylation and oncogenic functions of UHRF1 in human cancer cells.