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A recent study published in the journal Science has revealed a cellular mechanism for the transmission of genetic mutations and points to a potential treatment that could reduce the risk of infants developing severe and incurable mitochondrial diseases.
CAR-T cell therapy has shown significant efficacy in treating hematologic malignancies such as leukemia and lymphoma, prompting research into its application in various solid tumors. Among these tumors, glioblastoma (GBM) stands out as a particularly challenging target due to its aggressive nature and the lack of effective treatment options.
Environmental, social, and genetic factors contribute to overeating and reduced physical activity, leading to weight gain. Over time, weight gain is often associated with the development of hypertension, dyslipidemia, and cardiovascular disease. Obesity-related dyslipidemia is characterized by elevated serum triglyceride (TG) levels, elevated low-density lipoprotein (LDL) cholesterol levels, and low high-density lipoprotein (HDL) cholesterol levels. This lipid profile has a pro-atherosclerotic effect. However, the mechanisms by which obesity causes changes in lipid levels remain unclear.
Liver cancer treatment has always been a clinical challenge. Traditional treatments often yield limited effectiveness due to rapid tumor progression and high molecular heterogeneity. Finding targets that simultaneously address tumor metabolism and immune evasion has become crucial for overcoming treatment bottlenecks. Recently, Hepatology published a new study by a team from the University of Hong Kong titled "Targeting sterol O-acyltransferase 1 rewires fatty acid metabolism and uncovers immune vulnerability in hepatocellular carcinoma." They focused on sterol O-acyltransferase 1 (SOAT1), discovering that this protein is not only a metabolic vulnerability point in liver cancer but also regulates the tumor immune microenvironment, bringing a new "two-pronged" strategy to liver cancer treatment.
Hepatocellular carcinoma (HCC) is the fourth most common cancer worldwide and the second leading cause of cancer-related death. Clinically, HCC is often diagnosed at an advanced stage, leaving patients with limited treatment options. Recently, immune checkpoint blockade (ICB) therapy using monoclonal antibodies targeting PD-1, PD-L1, or CTLA-4 has emerged as a promising treatment approach for HCC. However, the unique liver environment significantly reduces HCC's response to ICB therapy, and resistance often develops, resulting in an overall benefit rate of only 15%-20%.
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).
