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In a new study, a team of scientists from Peking University in China has developed a cancer therapy that has the potential to make lifesaving treatments accessible to patients everywhere. This new cancer therapy could expand access to advanced treatments. The study was published in the journal Cell.
Most single-gene diagnosed neurodevelopmental disorders are caused by haploinsufficiency, meaning only one of the two copies of a gene remains functional. Haploinsufficiency of the SCN2A gene is one of the most common causes of neurodevelopmental disorders, often manifesting as autism, intellectual disability, and, in some children, intractable epilepsy.
It is known that gut microbes and their metabolites are closely linked to the development and progression of colorectal cancer and the effectiveness of immunotherapy. Gut microbes can convert primary bile acids from the host into various bile acid metabolites, such as 3-oxolithocholic acid (3-oxo-LCA), through reactions such as dehydrogenation and epimerization.
Developing personalized therapies has always been a key goal of medical research. This year, the news of the first patient receiving a personalized CRISPR gene-editing therapy garnered widespread attention worldwide. This marked the first time a gene-editing therapy has been tailored to a single patient. The infant, KJ Muldoon, was diagnosed with a severe genetic disorder, carbamoyl phosphate synthetase-1 (CPS1) deficiency, just days after birth. Researchers developed a customized lipid nanoparticle (LNP)-delivered base editing therapy in just six months and validated its safety and efficacy in monkeys. The therapy successfully corrected the child's disease-causing genetic mutation and resulted in significant clinical improvement. This success has become a model for personalized therapy.
Tuberculosis (primarily pulmonary) is the leading cause of death and disability from infectious diseases worldwide (excluding the COVID-19 period), killing 1.2 million people annually. The burden of TB is particularly high in low- and middle-income countries. TB is the leading cause of morbidity and mortality across all diseases in these countries, and has remained largely unchanged in recent decades.
A new Emory University study reveals why a once-promising experimental amyotrophic lateral sclerosis (ALS) drug, despite successfully reaching its intended target in the central nervous system (CNS)—the brain and spinal cord—failed to help patients.
Specifically, the researchers found that PTDSS1 deficiency not only enhanced tumor cells' responsiveness to interferon-γ (IFN-γ) but also increased antigen presentation (e.g., elevated MHC-I expression), making tumor cells more susceptible to antigen-specific CD8+ T cell recognition and killing. Furthermore, PTDSS1 deficiency improved the tumor microenvironment (TME), increasing the number of tumor-infiltrating CD8+ T cells and the frequency of iNOS-positive myeloid cells. Genetic and pharmacological interventions confirmed that both PTDSS1 knockdown and PTDSS1 inhibitors enhanced the efficacy of PD-1 inhibitors, suppressed tumor burden, and prolonged overall survival in mice. The related research was published in Science Advances.
Chronic pain affects approximately one in five adults, significantly negatively impacting quality of life and leading to severe socioeconomic consequences. In fact, when all pain conditions are considered, chronic pain is the leading cause of disability worldwide. Unfortunately, current treatments for chronic pain are often suboptimal due to poor efficacy and tolerability. The molecular causes of chronic pain are not fully understood, but increased excitability of nociceptors and the central pain pathways to which they project are important factors contributing to this state of pain sensitization.
In the field of medical research, leukemia remains a major threat to human health. Acute myeloid leukemia (AML), a member of the leukemia family, presents a heavy burden on countless patients and their families with its complex and diverse subtypes and aggressive disease progression. Among these, acute megakaryocytic leukemia (AMKL), due to its rarity and highly aggressive nature, remains a pressing challenge for the medical community.
To overcome these challenges, researchers have continuously upgraded and modified CAR-T cells. For example, incorporating different costimulatory signaling domains, such as CD28 or 4-1BB, addresses the short in vivo survival and poor activity of CARs. Simultaneously incorporating two costimulatory molecules into third-generation CARs enhances cytokine secretion. To better target the tumor microenvironment, researchers have developed fourth-generation CAR-T cells—"armored" T cells.
