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Secondary Neutropenias.

Neutrophils are a critical component of immunity, particularly against bacteria and other pathogens, but also in inflammation and tissue repair. As a consequence, individuals with neutropenia, defined by a reduction in absolute neutrophil counts, exhibit a strong propensity to severe infections that typically present with muted symptoms. Neutropenias encompass a heterogeneous set of disorders, comprising primary neutropenias, in which specific genes are mutated, and the more common secondary neutropenias, which have diverse non-genetic causes. These include hematological and other cancers, inv

Non-Hematologic Toxicity of Bortezomib in Multiple Myeloma: The Neuromuscular and Cardiovascular Adverse Effects.

The overall approach to the treatment of multiple myeloma (MM) has undergone several changes during the past decade. and proteasome inhibitors (PIs) including bortezomib, carfilzomib, and ixazomib have considerably improved the outcomes in affected patients. The first-in-class selective PI bortezomib has been initially approved for the refractory forms of the disease but has now become, in combination with other drugs, the backbone of the frontline therapy for newly diagnosed MM patients, as well as in the maintenance therapy and relapsed/refractory setting. Despite being among the most widely

Молодой мужчина с повреждениями почек в результате генетической митохондриальной болезни: доклад о конкретных случаях и обзор литературы.

Наиболее знакомым синдромом ушей и почек является синдром Алпорта для нефролога. Мутации в митохондриальном гене MT-TL1, который кодирует UUR, может также вызывать почечную дисфункцию и потерю слуха. В этом исследовании мы сообщили о молодом китайском мужчине с белковой и почечной дисфункцией с морфологической презентацией фокусного сегментального гломерулосклероза (FSGS) с m.3243 A > G мутация в митохондрии в митохондриальном гене MT-TL1. Мы провели систематический обзор литературы в целях обобщения ранее сообщенных случаев. 17-year-old Мужчина был доставлен в нашу больницу из-за пенистых моч

Роль воспаления в нейродегенеративных болезнях: болезнь Паркинсона, болезнь Альцгеймера и множественный склероз.

Нейродегенеративные заболевания представляют собой группу заболеваний, которые, как правило, характеризуются постепенным повреждением и дегенерацией нейронов в центральной нервной системе. В эту группу входят болезнь Паркинсона, болезнь Альцгеймера и множественный склероз. В последние годы все больше фактов указывает на важную роль воспаления в патогенезе этих условий. Происхождение воспаления в мозге, которое часто вызывается провоспалительной активацией микроглий или астроцитов, может, следовательно, привести к хронической провокационной реакции, способствующей развитию нейродегенеративных п

Clinical trial challenges, design considerations, and outcome measures in rare CNS tumors.

Clinical research for patients with rare cancers has been very challenging. First and foremost, patient accrual to clinical trials typically requires a network, cooperative group, or even international collaboration in order to achieve the necessary numbers of patients to adequately evaluate a new treatment or intervention. Similar limitations in preclinical models and in the understanding the natural history of the disease or pertinent prognostic factors further impede the development of hypothesis-based, appropriately powered clinical trials. However, despite these challenges, several studie

Skeletal stem cells, a new direction for the treatment of bone and joint diseases.

Skeletal stem cells (SSCs) are tissue-specific stem cells characterized by their capacity for self-renewal and their position at the apex of the differentiation hierarchy. They can generate mature bone cell types essential for bone development, maintenance, and repair. Lineage tracing experiments have demonstrated that SSCs reside in the bone marrow, periosteum, and the resting zone of the growth plate. These findings not only enhance our understanding of bone growth and development mechanisms but also offer novel therapeutic strategies for conditions such as epiphyseal injuries, fractures, os

Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.

CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medic

Circadian clocks and adaptive immune function: from mechanisms to therapeutic applications.

The circadian clock exerts profound regulatory control over adaptive immunity, particularly affecting T and B lymphocyte development, trafficking, activation, and effector function. These immune cells possess autonomous molecular clocks governed by core genes such as Bmal1, Clock, Per1/2, and Cry1/2, which influence transcriptional programs related to receptor expression, cytokine signaling, and metabolism. Circadian regulation shapes the daily oscillation of lymphocyte recirculation through lymphoid organs, modulates antigen responsiveness, and fine-tunes subset differentiation-most notably s

Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence.

On 4 September 2024, the Reagan-Udall Foundation for the FDA (FDA Foundation) in collaboration with the Food and Drug Administration (FDA) and the Gates Foundation hosted a workshop titled "Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence". The event brought together a diverse group of experts, including international regulatory bodies, regulated industries, healthcare professionals, patients, academic researchers and global health advocates, to discuss the rapid advancements in gene therapy and the pressing need for equitable access in low-and middle-

AL Amyloidosis: Unfolding a Complex Disease.

Light chain (AL) amyloidosis is a rare plasma cell dyscrasia. An estimated 12,000 people live with the disease in the United States. AL amyloidosis occurs from the misfolding of proteins that deposit in organs (heart, kidneys, digestive tract, tongue, lungs, and nervous system), leading to progressive organ damage and impairment of quality of life. The treatment of AL amyloidosis has improved greatly over the past several years, with new treatments currently in development. This article will focus on the pathophysiology, diagnosis, and treatment of AL amyloidosis.

Skeletal Metastases of Unknown Primary: Biological Landscape and Clinical Overview.

Skeletal metastases of unknown primary (SMUP) represent a clinical challenge in dealing with patients diagnosed with bone metastases. Management of these patients has improved significantly in the past few years. however, it is fraught with a lack of evidence. While some patients have achieved impressive gains, a more systematic and tailored treatment is required. Nevertheless, in real-life practice, the outlook at the beginning of treatment for SMUP is decidedly somber. An incomplete translational relevance of pathological and clinical data on the mortality and morbidity rate has had unsatisf

Mesenchymal stem cell transplantation plays a role in relieving cancer pain.

Tumors can invade, compress, and damage nerves, leading to persistent pain and seriously affecting the quality of life of patients. However, their treatment is challenging. Sensitization of peripheral receptors, abnormal activity of primary sensory neurons, activation of glial cells, enhanced inflammatory responses, and sensory information transmission contribute towards cancer pain. Therefore, considerable attention has been paid to exploring prospective methods to inhibit the occurrence of these factors and relieve cancer pain. Studies on different types of pains have revealed that the trans

Cannabidiol-from Plant to Human Body: A Promising Bioactive Molecule with Multi-Target Effects in Cancer.

Cannabis sativa L. is a plant long used for its textile fibers, seed oil, and oleoresin with medicinal and psychoactive properties. It is the main source of phytocannabinoids, with over 100 compounds detected so far. In recent years, a lot of attention has been given to the main phytochemicals present in Cannabis sativa L., namely, cannabidiol (CBD) and Δ9-tetrahydrocannabinol (THC). Compared to THC, CBD has non-psychoactive effects, an advantage for clinical applications of anti-tumor benefits. The review is designed to provide an update regarding the multi-target effects of CBD in different

The Role of the CCR5 Receptor in Neuropathic Pain Modulation: Current Insights and Therapeutic Implications.

Neuropathic pain, a chronic condition arising from injury or dysfunction of the somatosensory nervous system, is characterized by persistent hypersensitivity and spontaneous pain. The chemokine receptor CCR5 (C-C motif chemokine receptor 5) has recently been identified as a critical mediator in neuroinflammation and neuropathic pain signaling pathways. Expressed on immune cells and neurons, CCR5 regulates immune cell recruitment and activation, thereby contributing to neuronal sensitization and maintenance of pain states. This review examines the currently characterized molecular mechanisms th

Совпадение в полном тексте источника
… Evaluation of Quality of Life and Satisfaction of Patients with Neuropathic Pain and Breakthrough Pain: Economic Impact Based on Quality of Life. Pain Res. Treat. 2018;2018:5394021. doi: 10.1155/2018/5394021. 11. Cao B., Xu Q., Shi Y., Zhao R., Li H., Zheng J., Liu F., Wan Y., Wei B. …

Drug Repurposing for Cancer Treatment: A Comprehensive Review.

Cancer ranks among the primary contributors to global mortality. In 2022, the global incidence of new cancer cases reached about 20 million, while the number of cancer-related fatalities reached 9.7 million. In Saudi Arabia, there were 13,399 deaths caused by cancer and 28,113 newly diagnosed cases of cancer. Drug repurposing is a drug discovery strategy that has gained special attention and implementation to enhance the process of drug development due to its time- and money-saving effect. It involves repositioning existing medications to new clinical applications. Cancer treatment is a therap

Promoting patient-centered care in CAR-T therapy for hematologic malignancy: a qualitative meta-synthesis.

BackgroundCAR-T therapy has emerged as a potentially effective treatment for individuals diagnosed with hematologic malignancies. Understanding patients' unique experiences with this therapeutic approach is essential. This knowledge will enable the development of tailored nursing interventions that align with the increasing importance of patient-centered care.ObjectiveTo examine and synthesize qualitative data on patients and their family caregivers' experiences during the treatment journey.DesignWe conducted a systematic review and qualitative meta-synthesis. Eligible studies contained adult

PNEUMONIA | Community Acquired Pneumonia, Bacterial and Other Common Pathogens

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Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.

Genome editing has recently evolved from a theoretical concept to a powerful and versatile set of tools. The discovery and implementation of CRISPR-Cas9 technology have propelled the field further into a new era. This RNA-guided system allows for specific modification of target genes, offering high accuracy and efficiency. Encouraging results are being announced in clinical trials employed in conditions like sickle cell disease (SCD) and transfusion-dependent beta-thalassaemia (TDT). The path finally led the way to the recent FDA approval of the first gene therapy drug utilising the CRISPR/Cas

Последние новости и достижения в области диагностики и управления клетками Плазмы.

Плазменные клеточные дискразии (ПХД) охватывают разнородную группу расстройств, начиная от асимптоматических свойств-прекурсоров, таких, как моноклональная гаммопатия неопределенного значения (MGUS) и заморозка множественной миеломы (SMM) и кончая открытой множественной миеломой (MM), первичной плазменной лейкемией и системным амилоидозом. Этот обзор основан на структурированном поиске литературы, опубликованной между PubMed, Scopus, Cochrane Bibrary и Web of Science. 2015 и 2025, дополненные референцной проверкой и основными международными руководящими принципами в отношении миэломы для обесп

The dark side of immunotherapy.

Immunotherapy has broadened the therapeutic scope and response for many cancer patients with drugs that are generally of higher efficacy and less toxicity than prior therapies. Multiple classes of immunotherapies such as targeted antibodies and immune checkpoint inhibitors (ICI), cell-based immunotherapies, immunomodulators, vaccines, and oncolytic viruses have been developed to help the immune system target and destroy malignant tumors. ICI targeting programmed cell death protein-1 (PD-1) or its ligand (PD-L1) are among the most effective immunotherapy agents and are a major focus of current