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Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease.

We present an overview of clinical trials involving gene editing using clustered interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), or zinc finger nucleases (ZFNs) and discuss the underlying mechanisms. In cancer immunotherapy, gene editing is applied ex vivo in T cells, transgenic T cell receptor (tTCR)-T cells, or chimeric antigen receptor (CAR)-T cells to improve adoptive cell therapy for multiple cancer types. This involves knockouts of immune checkpoint regulators such as PD-1, components of the endo

Alpha-lipoic acid as a trigger of insulin autoimmune syndrome: Current evidence, a case-based approach, diagnostic pitfalls, and practical management.

Insulin autoimmune syndrome (IAS), also known as Hirata disease, is a rare immune-mediated cause of endogenous hyperinsulinemic hypoglycemia characterized by circulating insulin autoantibodies (IAAs). It typically develops in individuals without prior exposure to exogenous insulin. Although IAS has been associated with viral infections and, less commonly, with autoimmune and hematologic disorders, medications and dietary supplements are considered the most frequent precipitating factors. Sulfhydryl-containing compounds are the most commonly implicated triggers. Methimazole represents the class

Искусственный интеллект основывается на достижениях в области наномедицины для лечения мозговых расстройств: обновленный обзор повествования.

Наномедицина - это наномасштабные, биосопоставимые материалы, которые предлагают перспективные альтернативы традиционным методам лечения расстройств мозга. Технологические достижения в области искусственного интеллекта (АИ), в частности машинного обучения (МЛ) и глубокого обучения (ДЛ), трансформируют область наномедицины путем совершенствования диагностики заболеваний, биомаркировки, прогнозирования и мониторинга заболеваний, целевых поставок наркотиков и терапевтического вмешательства, а также содействия расчетным и методологическим разработкам. Эти успехи могут быть достигнуты за счет анали

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… Many drugs have difficulty crossing the blood–brain barrier (BBB), making it essential to find different dosage forms that enhance drug bioavailability. …

What is new in the pathogenesis and treatment of IgA glomerulonephritis.

Recently, new findings have been clarified concerning both pathogenesis and treatment of IgA nephritis. The four hits theory has been confirmed but several genetic wide association studies have allowed finding several genes connected with the pathogenesis of the disease. All these new genes apply to each of the four hits. Additionally, new discoveries concerning the microbiota and its connection with immune system and IgA generation have allowed finding out the role of the mucosa in IgA nephropathy pathogenesis. The IgA treatment is also changed included the future possibilities. The treatment

Cancer Immunotherapy in Combination with Radiotherapy and/or Chemotherapy: Mechanisms and Clinical Therapy.

Since the United States Food and Drug Administration approved the first immune checkpoint inhibitor ipilimumab for metastatic melanoma in 2011, ICIs have been approved for a range of cancers and significantly improving treatment outcomes. However, the objective response rate of ICI monotherapy remains modest (10-40%), with clinical benefit observed in only 15-20% of patients. The limited efficacy of ICIs in many patients is often attributed to poorly immunogenic ("cold") tumors. Radiotherapy and chemotherapy exhibit immunomodulatory properties that can enhance tumor immunogenicity. These effec

Всеобъемлющая оценка неблагоприятных профилей, связанных с биконкретными антителами в множестве миеломы.

Биотечные антитела (BSAbs) сулили большие надежды в борьбе с повторной/охлажденной множественной миеломой (MM). Несмотря на свою эффективность, этот класс наркотиков связан со значительными токсичными свойствами. В этом исследовании мы провели совместный анализ имеющихся клинических испытаний БСАБ для лечения ММ, включая полные публикации и резюме до апреля 2025. BsAb были отнесены к категории two группы: антигены созревания клеток B (BCMA) и GPRC5D/FcRH5 BsAbs. Тест Welch был проведен для сравнения характеристик безопасности каждого агента. Для кластеризации мы использовали анализ основных ко

Hidrox® Counteracts Cyclophosphamide-Induced Male Infertility through NRF2 Pathways in a Mouse Model.

BACKGROUND: Every year, men use cyclophosphamide to treat various cancers and autoimmune diseases. On the one hand, this chemotherapy often has the beneficial effect of regressing the tumor, but on the other hand, it leads to infertility due to excessive oxidative stress and apoptosis in the testes caused by its metabolite, acrolein. METHODS: The objective of this study was to evaluate the beneficial power of a new compound called Hidrox®, containing 40-50% hydroxytyrosol, in counteracting the damage related to fertility induced by cyclophosphamide. The study was conducted using a single intr

A review of feline infectious peritonitis virus infection.

Feline infectious peritonitis (FIP) is an infectious disease characterized by non-specific laboratory changes and clinical signs. Clinical symptoms include anorexia, jaundice, fever, and weight loss. Moreover, some lesions are found in the digestive and respiratory systems. FIP, whose virulence varies, cannot be distinguished using several diagnostic methods. Moreover, feline coronaviruses (FCoVs) can be classified into two serotypes based on differences in their amino acid sequences, spike (S) protein sequences, and antibody (Ab) neutralization. There are two pathotypes, namely those caused b

Recent Advances in the Development and Utilization of Nanoparticles for the Management of Malignant Solid Tumors.

The purpose of nanotechnology-based drug delivery systems or novel drug delivery systems is to improve the effectiveness of therapy, and their promising properties have led to their increasing significance in the management of cancer. The researchers have primarily focused on designing novel nanocarriers, like nanoparticles (NPs), that can effectively deliver drugs to target cells and respond specifically to conditions particular to cancer. Whether passive or active targeting, these nanocarriers can deliver therapeutic cargoes to the tumor site to release the drug from the drug delivery system

The Role of Vitamin C in Cancer Prevention and Therapy: A Literature Review.

Vitamin C is a water-soluble antioxidant associated with the prevention of the common cold and is also a cofactor of hydrolases that participate in the synthesis of collagen and catecholamines, and in the regulation of gene expression. In cancer, vitamin C is associated with prevention, progression, and treatment, due to its general properties or its role as a pro-oxidant at high concentration. This review explores the role of vitamin C in cancer clinical trials and the aspects to consider in future studies, such as plasmatic vitamin C and metabolite excretion recording, and metabolism and tra

A Deep Dive Into the Newest Avenues of Immunotherapy for Pediatric Osteosarcoma: A Systematic Review.

Osteosarcoma (OS) is the most common primary bone cancer affecting children and young adults, most often occurring at the metaphysis of long bones. At present, treatment with combinations of surgery and chemotherapy for the localized OS has only brought minuscule improvements in prognosis. In comparison, the advanced, metastatic, or recurrent forms of OS are often non-responsive to chemotherapy, adding to the dire need to develop new and efficient therapies. The question of interest investigated in this systematic review is whether immunotherapy can play a meaningful role in improving the clin

Prevention and treatment of oral adverse effects of antiresorptive medications for osteoporosis - A position paper of the Brazilian Society of Endocrinology and Metabolism (SBEM), Brazilian Society of Stomatology and Oral Pathology (Sobep), and Brazilian Association for Bone Evaluation and Osteometabolism (Abrasso).

Antiresorptive therapy is the main form of prevention of osteoporotic or fragility fractures. Medication-related osteonecrosis of the jaw (MRONJ) is a relatively rare but severe adverse reaction to antiresorptive and antiangiogenic drugs. Physicians and dentists caring for patients taking these drugs and requiring invasive procedures face a difficult decision because of the potential risk of MRONJ. The aim of this study was to discuss the risk factors for the development of MRONJ and prevention of this complication in patients with osteoporosis taking antiresorptive drugs and requiring invasiv

Liposomal Drug Delivery Systems and Anticancer Drugs.

Cancer is a life-threatening disease contributing to ~3.4 million deaths worldwide. There are various causes of cancer, such as smoking, being overweight or obese, intake of processed meat, radiation, family history, stress, environmental factors, and chance. The first-line treatment of cancer is the surgical removal of solid tumours, radiation therapy, and chemotherapy. The systemic administration of the free drug is considered to be the main clinical failure of chemotherapy in cancer treatment, as limited drug concentration reaches the tumour site. Most of the active pharmaceutical ingredien

Acute Disseminated Encephalomyelitis (ADEM): Current View into Etiopathogenesis and Clinical Features.

Acute disseminated encephalomyelitis (ADEM) is a rare, immune-mediated demyelinating disorder of the central nervous system (CNS) that predominantly affects children and young adults. ADEM typically follows an infectious or, less commonly, immunization-related trigger, and despite decades of clinical observation, its etiopathogenesis remains only partially understood. Clinically, the diagnosis of ADEM continues to pose significant challenges due to the absence of disease-specific biomarkers and its clinical and radiological overlap with other acquired demyelinating syndromes. This narrative re

Intestinal barrier functions in hematologic and oncologic diseases.

The intestinal barrier is a complex structure that not only regulates the influx of luminal contents into the systemic circulation but is also involved in immune, microbial, and metabolic homeostasis. Evidence implicating disruption in intestinal barrier functions in the development of many systemic diseases, ranging from non-alcoholic steatohepatitis to autism, or systemic complications of intestinal disorders has increased rapidly in recent years, raising the possibility of the intestinal barrier as a potential target for therapeutic intervention to alter the course and mitigate the complica

Cardiotoxicity of Novel Targeted Hematological Therapies.

Chemotherapy-related cardiac dysfunction, also known as cardiotoxicity, is a group of drug-related adverse events negatively affecting myocardial structure and functions in patients who received chemotherapy for cancer treatment. Clinical manifestations can vary from life-threatening arrythmias to chronic conditions, such as heart failure or hypertension, which dramatically reduce quality of life of cancer survivors. Standard chemotherapy exerts its toxic effect mainly by inducing oxidative stress and genomic instability, while new targeted therapies work by interfering with signaling pathways

Epidemiology, Risk Factors, and Outcomes of Neutropenic Enterocolitis in Onco-Hematological Patients According to Chemotherapy Regimen.

BackgroundWhile neutropenic enterocolitis (NEC) is a well-known life-threatening complication during intensive chemotherapy, its incidence, impact, and outcome on specific at-risk populations remain ill defined.MethodsWe report 178 NEC episodes during 1963 myeloablative chemotherapy courses among 1259 adult patients with acute myeloid (AML) or lymphoblastic (ALL) leukemia or receiving autologous hematopoietic stem cell transplantation (auto-HCT) for lymphoma or multiple myeloma. Risk factors were assessed by multivariate logistic regression models.ResultsMost NEC cases (93.3%) occurred during

Contextual analysis of hematopoietic stem cell transplantation in outpatients.

Objectivesto analyze and map the contextual aspects of hematopoietic stem cell transplantation in outpatients in the literature.Methodsa scoping review was conducted from October 2023 to June 2024 in databases, portals, and thesis and dissertation libraries. Data collection and analysis were based on the framework proposed by Hinds, Chaves, and Cypress, according to the contextual layers: metacontext, general, specific, and immediate.Resultsthe final sample consisted of 30 studies. Regarding the contextual layers, metacontext addressed outpatient hematopoietic stem cell transplantation worldwi

Mechanisms of T-cell Depletion in Tumors and Advances in Clinical Research.

T lymphocytes (T cells) are essential components of the adaptive immune system that play a vital role in identifying and eliminating infected and tumor cells. In tumor immunotherapy, T cells have emerged as a promising therapeutic strategy due to their high specificity, potent cytotoxic capability, long-lasting immune memory, and adaptability within immunotherapeutic approaches. However, tumors can evade the immune system by depleting T cells through various mechanisms, such as inhibitory receptor signaling, metabolic exhaustion, and physical barriers within the tumor microenvironment. This re

Uncovering potential targets for antibody-drug conjugates in the treatment of gynecologic malignancies.

BackgroundAntibody-drug conjugates (ADCs) play an important role in the targeted therapy of gynecological malignancies. The purpose of this study was to investigate the expression of targets in gynecologic malignancies in order to predict the selection of targets for the development of antibody-drug conjugates.MethodsIn this article, we identified existing ADCs and their targets through clinical trial databases and public genomic datasets, performed differential analysis of tumor antigen targets (TATs) expression between tumor and normal tissues, and evaluated the necessity of the targets for