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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Duchenne muscular dystrophy (DMD), the commonest form of muscular dystrophy, is caused by lack of dystrophin. One of the most promising therapeutic approaches is antisense-mediated elimination of frame-disrupting mutations by exon skipping. However, this approach faces two major hurdles: limited applicability of each individual target exon and uncertain function and stability of each resulting truncated dystrophin. Skipping of exons 45-55 at the mutation hotspot of the DMD gene would address both issues. Theoretically it could rescue more than 60% of patients with deletion mutations. Moreover,

Vaccination with patient-specific tumor-derived antigen in first remission improves disease-free survival in follicular lymphoma.

PURPOSE: Vaccination with hybridoma-derived autologous tumor immunoglobulin (Ig) idiotype (Id) conjugated to keyhole limpet hemocyanin (KLH) and administered with granulocyte-monocyte colony-stimulating factor (GM-CSF) induces follicular lymphoma (FL) -specific immune responses. To determine the clinical benefit of this vaccine, we conducted a double-blind multicenter controlled phase III trial. PATIENTS AND METHODS: Treatment-naive patients with advanced stage FL achieving complete response (CR) or CR unconfirmed (CRu) after chemotherapy were randomly assigned two to one to receive either Id

Closing the loop for Deep Brain Stimulation implants enables personalized healthcare for Parkinson's disease patients.

DEEP brain stimulation implants have improved life quality for more than 70,000 patients world-wide with diseases like Parkinson's, essential tremor, or obsessive-compulsive disorder where pharmaceutical therapies alone could not offer sufficient relief. Still, optimization and monitoring relies heavily on regular clinical visits, putting a burden on patient's comfort and clinicians. Permanent monitoring and combination with other patient health signals could ultimately lead to a personalized closed-loop therapy with remote quality monitoring. This requires technological improvements on the DB

Long-term results of an individualized, multifaceted, and multidisciplinary therapeutic approach to provoked vestibulodynia.

INTRODUCTION: Although it is highly recommended to use a multifaceted approach to treat provoked vestibulodynia (PVD), the large majority of treatment studies on PVD used a one-dimensional approach. AIM: To evaluate the long-term treatment outcome of a multifaceted approach to vulvar pain, sexual functioning, sexually related personal distress, and relational sexual satisfaction in women with PVD. METHODS: Retrospective questionnaire survey 3-7 years after treatment. MAIN OUTCOME MEASURES: Sexual functioning, sexually related personal distress, and relational sexual satisfaction were measur

Bioorthogonal chemistry conjugates: ADCs and beyond.

Bioorthogonal click chemistry has played a transformative role in developing advanced hybrid conjugates. These molecular constructs are formed by chemically linking a targeting component to a functional moiety through a suitable linker. Therapeutic conjugates are designed to deliver a biologically active agent to a specific target to treat, prevent, or modify a disease process. By directing the payload to a defined site of action, these conjugates can improve efficacy while reducing off-target effects and systemic toxicity. Among these, antibody-drug conjugates stand out as the most clinically

Biomarker Integration in the Perioperative Management of Early-Stage Non-Small-Cell Lung Cancer.

The concept of personalized medicine has tremendous appeal-tailoring diagnosis and treatment to an individual, rather than a "one-size-fits-all" approach. The management of non-small-cell lung cancer (NSCLC) has rapidly evolved toward strategies increasingly guided by tumor biology and precision oncology. The safety and significant efficacy of biomarker-directed therapies in metastatic disease have fueled interest in applying precision oncology to early-stage, operable NSCLC. Advances in molecular profiling have revealed that a substantial proportion of resected NSCLC harbor actionable driver

Data-driven subtyping of early Parkinson's disease via mutual cross-attention fusion of EEG and dual-task gait features.

Parkinson's disease (PD) exhibits marked clinical heterogeneity, which poses challenges for diagnosis, prognosis, and therapeutic precision, … yping of PD with prognostic implications for personalized rehabilitation strategies and accelerates precision medicine.

Genetic kidney diseases: Decoding molecular taxonomies to drive precision nephrology and therapeutic innovation.

Genomic advances have transformed the landscape of genetic kidney diseases, enabling a shift from traditional phenotype-based diagnostics to … polycystic kidney disease may lead to inappropriate tolvaptan use. Molecular diagnostics now enable precision medicine by guiding therapy, optimizing transplantation decisions, and facilitating genetic counseling. Meanwhile, innovative therapies targeting the DNA-RNA-protein-epigenetic axis are advancing toward mechanism-driven treatments that deliver genuine patien …

Targeting gut dysbiosis: a new frontier in MASLD management.

Globally, Metabolic dysfunction-associated steatotic liver disease (MASLD) is a prominent leading cause of CLD, which is intricately linked … tively will require moving beyond broad ecosystem resets toward highly tailored, multi-omics-driven precision medicine to accurately target individualized microbial deficits.

Microbes, mood, and metabolism/obesity: Pharmacological insights into the gut-obesity-depression triad.

The global rise in obesity and depression, two highly prevalent and often comorbid disorders has intensified interest in the gut–brain axis … al and psychological dimensions of obesity and depression, marking a major step toward holistic and precision medicine.

Benchmark Development for Fundamental Arthroscopic Skills Using a Simulation-Based Training Program: Observational Study.

BACKGROUND: Surgical education has shifted from the traditional Halstedian apprenticeship model toward incorporating simulation due to work-hour restrictions, increasing case complexity, and economic and liability pressures. Building on the success of the Fundamentals of Laparoscopic Surgery program for general surgery, the Fundamentals of Arthroscopic Surgery Training (FAST) program was developed to establish proficiency benchmarks for orthopedic trainees in basic arthroscopic skills. OBJECTIVE: We aimed to establish benchmarks for 5 FAST workstation modules. METHODS: Sports medicine fellow

Bifunctional Fe-Doped MoS2 Nanozyme for DNA-Engineered Protein Discrimination and Photothermally Enhanced Bacterial Eradication.

The accurate identification of multiple proteins and effective eradication of bacteria hold paramount importance for clinical diagnosis and … ifunctional nanoplatform for biomedical diagnostics and oral therapy, highlighting its potential in precision medicine and infectious disease management.

Highlights from the 2024 annual meeting of the fetal, infant, and Toddler Neuroimaging Group (FIT'NG).

The Fetal, Infant, and Toddler Neuroimaging Group (FIT'NG) is a scientific society advancing research on fetal, perinatal, infant, and early childhood brain and behavioral development through neuroimaging, encompassing theoretical, technical, fundamental, and clinical dimensions. It unites researchers across physics, neuroscience, cognitive science, psychology, computer science, biomedical engineering, and medicine, fostering interdisciplinary collaboration among leading laboratories in the field. The annual FIT'NG conference serves as a key forum for exchanging new ideas and methods, addressi

Modern Era in Personalized Medicine of Dual Antiplatelet Therapy After Myocardial Revascularization.

Dual antiplatelet therapy (DAPT) with aspirin and a P2Y12 inhibitor remains the cornerstone of antithrombotic management after myocardial re … mic clinical factors including age and concomitant medications. We examine the current landscape of precision medicine tools for individualizing DAPT, including platelet function testing, point-of-care genotyping, validated clinical risk scores, and emerging artificial intelligence (AI)-based predictive models. Evidence from landmark trials is synthesized to evaluat …

Pediatric Myocarditis: Challenges in Diagnosis and Treatment.

Pediatric myocarditis is a clinically diverse and often under-recognized inflammatory condition of the myocardium, with presentations that r … n interacts with viral or autoimmune triggers. This paradigm shift has underscored the relevance of precision medicine, emphasizing tailored management based on the underlying etiology and genetic background. While supportive care remains the foundation of therapy, including rest, hemodynamic monitoring, and guideline-directed medical therapy for heart failure, phar …

An updated review on the role of extracellular vesicles in immune system modulation in breast cancer with special emphasis on immune checkpoint regulators.

Breast cancer progression and resistance to therapy are strongly influenced by immune evasion within the tumor microenvironment. Immune chec … mally invasive biomarkers for disease monitoring and as therapeutic targets or delivery systems for precision medicine. Overall, this review highlights current evidence on EV-mediated immune checkpoint regulation in breast cancer, highlighting PD-L1 and CTLA-4associated mechanisms as key drivers of immune evasion and promising targets for precision immunotherapy.

Сигнал Hippo-YAP/TAZ в гематологических злоумышленниках: Молекулярные механизмы, тротуар и терапевтический потенциал.

Сигнал Gippo представляет собой эволюционно сохранившуюся систему регулирования. Она играет центральную роль в контроле за распространением клеток, апоптозом, дифференциацией и гомеостазом тканей. Все больше данных свидетельствует о том, что дисрегулирование этого пути способствует развитию и развитию гематологических заболеваний. К ним относятся лейкемия, лимфома и множественная миелома. В частности, ключевую роль в этом процессе играет аберрантная активация белков, связанных с эффектом ниже по течению (YAP), и транскрипционный ко Активатор с обязательным мотивом PDZ (TAZ). Ненормальный сигна

Совпадение в аннотации оригинала
The Hippo signaling pathway represents an evolutionarily conserved regulatory network. It plays a central role in controlling cell prolifera … f Hippo signaling may provide novel insights into disease biology and accelerate the development of precision medicine approaches for hematological malignancies.

Monogenic and syndromic obesity in children: Clinical recognition, genetics, and precision management.

Obesity is increasingly being recognized as a heterogeneous condition with strong genetic underpinnings. Monogenic obesity, caused by single … ing disparities between health systems. Polygenic risk scores and multi-omics approaches may expand precision medicine beyond rare genetic syndromes to common obesity, highlighting the importance of integrating genetics, psychosocial care, and policy advocacy in the management of pediatric obesity.

Redox signaling in chronic airway diseases: pathogenic mechanisms and therapeutic implications.

Chronic airway diseases, including asthma, chronic obstructive pulmonary disease (COPD), and bronchiectasis, impose a significant global health burden. A central unifying feature of these diseases is redox imbalance, which is characterized by an excess of reactive oxygen and nitrogen species (ROS/RNS) that overwhelms the body's antioxidant defenses, causing cellular dysfunction, inflammation, and tissue damage. Physiological ROS/RNS are essential for immune regulation and transcriptional control, but chronic oxidative stress disrupts these processes, driving disease progression. In asthma, eos

Genetic basis of pancreatic steatosis: a systematic review of comparison between African and non-African populations.

This systematic review compared genetic evidence of pancreatic steatosis across African and non-African populations to illuminate ancestry-s … creas disease (NAFPD)-to-PDAC cascade. These ancestry-differentiated mechanisms position NAFPD as a precision medicine target.