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Disruption of the dynamic stability of the pro-inflammatory/anti-inflammatory phenotypes of macrophages within plaques significantly affects chronic vascular inflammation and exacerbates atherosclerosis. Reprogramming macrophages from a pro-inflammatory phenotype (M1) to an anti-inflammatory phenotype (M2) can slow the progression of atherosclerosis. However, chronic inflammatory stimulation can cause atherosclerotic macrophages to remain in a chromatin-closed state, inhibiting their phenotype reprogramming.
In recent years, CAR-T cell therapy has achieved remarkable success in treating blood cancers. However, it suffers from a critical weakness: the weeks-long and expensive preparation process, leaving many patients waiting for treatment and missing the optimal treatment window. Even more frustrating is that even when using a patient's own immune cells, these "modified warriors" can sometimes be accidentally attacked by other immune cells in the body, leading to the failure of the treatment.
Although backsplicing of pre-messenger RNA (pre-mRNA) exons is inefficiently processed by the classical spliceosomal machinery, this backsplicing generates circular RNAs (circRNAs) genome-wide. These RNAs are nearly identical in sequence to linear RNAs originating from the same gene locus, differing only at the backsplicing junction (BSJ) site.
The application of CAR-T cell therapy in solid tumors is limited by the suppressive tumor microenvironment (TME), which hinders T cell migration to tumor sites, leads to T cell exhaustion, insufficient T cell persistence, and limited endogenous anti-tumor immune responses. To address these therapeutic challenges, researchers have investigated combination strategies with immune checkpoint inhibitors (such as αPD-1, αCTLA-4, and αPD-L1) and immunomodulatory factors (such as IL-2, IL-7, IL-12, IL-15, and αTGFβ).
Since the late 1960s, when the initial concept of "using exogenous DNA to correct genetic defects in living cells" was proposed, gene therapy has evolved from theoretical exploration to clinical trials at an exponential pace, driven by scientific progress and technological innovation. Current clinical pipelines offer hope by targeting disease roots, achieving curative breakthroughs in some highly challenging complex disorders. In May 2019, the FDA approved Zolgensma-an AAV-9-based SMN1 gene replacement therapy-for treating spinal muscular atrophy (SMA) in infants under two, the leading genetic cause of infant mortality. In December 2023, Casgevy (exagamglogene autotemcel), a lentiviral-modified CD34+ hematopoietic stem/progenitor cell (HSPC) therapy, gained FDA approval for sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT).
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.
