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    High Prevalence of Preexisting Neutralizing Antibodies Against Adeno-Associated Virus Capsids in Mongolian People with Hemophilia
    Adeno-associated virus (AAV) vector-mediated gene therapy for hemophilia offers the potential for sustained therapeutic benefit after a single administration and is particularly attractive in regions with limited access to standard therapies. However, the prevalence of anti-AAV neutralizing antibodies (NAbs), which can impair therapeutic efficacy, varies geographically and remains poorly characterized in Mongolia. Here, we evaluated the seroprevalence of NAbs against clinically relevant AAV serotypes in Mongolia. NAbs against AAV3B, AAV5, and AAV8 were assessed in serum samples from 23 healthy volunteers and 52 people with hemophilia (PwH) using a validated cell-based transduction inhibition assay. A high seroprevalence of anti-AAV NAbs was observed across all three serotypes in both healthy volunteers (69.6%-87.0%) and PwH (78.9%-84.6%), with no significant differences between groups. NAbs were detected across all age groups, including early childhood, and frequently exhibited neutralization against multiple AAV serotypes, which was associated with higher NAb titers. Taken together, anti-AAV NAbs are highly prevalent and acquired early in life in the Mongolian population, with multiserotype neutralization patterns. These findings identify a substantial population-specific barrier to AAV-mediated gene therapy and highlight the need for alternative therapeutic strategies beyond AAV-based platforms to overcome immunological constraints in this setting.
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    Newborn screening and biobanking for preventable genetic disorders in Mongolia: A developing country perspective
    Newborn screening (NBS) is an important public health program, yet its implementation is highly uneven globally, with developing countries like Mongolia facing significant resource and infrastructure limitations that hinder early detection of preventable genetic disorders. Meanwhile, induced pluripotent stem cell (iPSC) technology, leveraged through biobanking, offers a powerful platform for disease modeling, drug discovery, and regenerative medicine, though its advanced applications are primarily concentrated in wealthier nations. We review the current status of NBS in Mongolia and argue for linking an expanded NBS program to an iPSC biobank. This link would improve diagnosis, create a foundation for Mongolian precision medicine, and strengthen local research capacity. We recognize the practical and ethical hurdles-funding, staffing, and data governance-and outline phased rollout, targeted training, and international partnerships as realistic next steps. Taken together, this approach could prevent avoidable disability in Mongolia while gradually building homegrown capability in genetic healthcare.
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    Scopus© Citations 20
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    Liver knockout of MCU leads to greater dysregulation of lipid metabolism in MAFLD.
    (2024-11)
    Liao, Qichao
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    Zhang, Yurou
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    Pan, Tingli
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    Sun, Yu
    ;
    Liu, Siqi
    Scopus© Citations 6
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    Measurement of atherosclerosis markers in individuals with periodontitis
    PURPOSE: The inflammatory response due to inflammatory cytokines, bacterial pathogens, and the altered lipoprotein metabolism in patients with periodontitis indicates that infection with periodontal anaerobic bacteria may influence atherogenesis in vitro and in vivo. We aimed to explore the effect of periodontitis concerning clinical and ultrasound markers of early atherosclerosis. METHODS: In this case-control study, a total of 30 systemically healthy adults (15 with periodontitis and 15 without periodontitis) over 40 years of age were studied. Periodontitis was determined by measuring the clinical attachment level (CAL) and radiographic bone loss (RBL). Conventional cardiovascular risk factors, including body mass index, serum levels of total cholesterol (TCH), triglycerides (TG), and high-density and low-density lipoprotein (HDL and LDL, respectively) cholesterol were evaluated. Carotid artery intima-media thickness (IMT) was measured using ultrasonography. RESULTS: The mean values of the CAL and carotid IMT were 5.02±0.9 mm and 0.084±0.01 cm vs. 1.6±0.61 mm and 0.072±0.02 cm in the periodontitis and healthy groups, respectively, reflecting statistically significant differences (P=0.001 and P=0.037, respectively). There were statistically significant differences in the serum levels of TCH, TG, and LDL between the 2 groups (P=0.017). The CAL and RBL were positively associated with carotid IMT and serum cholesterol levels, except for HDL, whereas tooth loss was not associated with any markers (P<0.05). Compared to the healthy group, participants with periodontitis exhibited 2.09 times higher odds (95% confidence interval, 1.22-3.59) of having subclinical atherosclerosis. CONCLUSIONS: The presence of periodontitis increased the risk of atherosclerosis.
    Scopus© Citations 4
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    Scopus© Citations 15
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    Analysis of 69 Hepatocellular Carcinoma Cases from the National Center for Pathology in Mongolia : A Comprehensive Study of Samples Collected Nationwide
    (2025)
    Jargalsaikhan, Orgil
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    Shao, Wenhua
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    Ichimura-Shimizu, Mayuko
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    Ishimaru, Soichiro
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    Koma, Takaaki
    The high rate of chronic hepatitis, including hepatitis B, C and D, in Mongolia creates a large health burden of advanced liver disease. This includes liver failure and the highest incidence rate of hepatocellular carcinoma (HCC) worldwide. In the present study, we histopathologically examined 69 recent cases of HCC from the Mongolian National Center for Pathology, which collects specimens from across the country. The background liver histology of HCC exhibited a bimodal distribution, with one peak corresponding to advanced liver fibrosis and another to mild liver fibrosis. The fibrosis severity negatively correlated with age. Additionally, the frequency of poorly differentiated tumors was significantly higher in the HCC with early stage of fibrosis. A comparison of the pathological characteristics of HCC in urban and rural areas showed that poorly differentiated tumors were highly prevalent in urban areas. The characteristics of HCC in Mongolia are different from those in other countries, suggesting that the causes of liver disease are not only related to viruses but also other factors that depend on the region. This study will provide insight into what research is needed next for liver cancer control in Mongolia. J. Med. Invest. 72 : 47-53, February, 2025.
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    PROTAC-mediated FTO protein degradation effectively alleviates diet-induced obesity and hepatic steatosis.
    (2025-01)
    Xiao, Yang
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    Jiang, Tianyu
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    Qi, Xinyi
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    Zhou, Jinfeng
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    Pan, Tingli
    Scopus© Citations 12
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    Aloesin improves metabolic associated fatty liver disease and obesity by targeting TGFBR1
    (2026-04)
    Wu, Jianfeng
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    Liu, Siqi
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    Ma, Zhen
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    Liao, Qichao
    BACKGROUND: Metabolic associated fatty liver disease (MAFLD) is a chronic liver condition with a high global prevalence. Obesity and its associated insulin resistance are among the main risk factors for MAFLD, for which no effective clinical treatments are currently available. Aloesin, a natural chromone compound derived from Aloe vera, has anti-inflammatory, antioxidant, anticancer, and antidiabetes activity. However, its involvement in MAFLD has not been systematically studied. PURPOSE: This study investigated the effectiveness of aloesin against MAFLD and obesity and elucidated its potential molecular mechanism. METHODS: Aloesin was added to the high-fat diet-of an induced mouse model and to oleic acid/palmitic acid-induced HepG2 cells to explore its effect on lipid metabolism. Molecular targets were identified by reverse docking, cellular thermal shift assay (CETSA), and drug affinity responsive target stability (DARTS). RESULTS: Aloesin significantly reduced lipid accumulation in hepatocytes in both the high-fat diet-induced MAFLD mouse model and the oleic acid/palmitic acid-induced HepG2 cells. It also alleviated oxidative stress and energy metabolism disorders and decreased the body fat mass in mice fed the high-fat diet, which ameliorated the pathological features of MAFLD and obesity. Target prediction and validation identified TGFBR1 as a direct target of aloesin, which was confirmed by CETSA and DARTS. Functional experiments demonstrated that overexpression of TGFBR1 increased lipid accumulation and metabolic disturbances, which were reversed by aloesin. CONCLUSION: Aloesin improved lipid deposition and slowed the progression of MAFLD by targeting TGFBR1. The results support its potential application for the prevention and treatment of MAFLD.
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    MRPL44 regulates lipid metabolism in metabolic dysfunction-associated steatotic liver disease through BNIP3-mediated mitophagy.
    (2025)
    Liu, Siqi
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    Xiao, Lianggui
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    Cheng, Qiuchen
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    Liao, Qichao
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    Huang, Yuxin
    Scopus© Citations 4